ASCO in Action Podcast

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The Impact of Utilization Management on Patients and Practices Apr 16, 2019

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Subscribe to the podcast through iTunes or Google Play. Welcome to this ASCO in Action podcast. This is ASCO's podcast series where we explore policy and practice issues that have impact on oncologists, the entire cancer care delivery team, and most importantly the individuals who care for people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action podcast series. For today's podcast, I am delighted to have as my guest Dr. Jeffrey Ward. He's the chair of ASCO's Government Relations Committee, and a longtime active member in that area. In addition to his important contributions to ASCO over the years, Dr. Ward is a medical oncologist and a hematologist at the Swedish Cancer Institute in Edmonds, Washington. Our conversation today will focus on utilization management in cancer care, or the policies that public and private insurers use to control the use of anticancer drug therapies, such as prior authorization requirements, restrictive formularies, step therapy, or fail first requirements, and specialty specific tiers. These are all new and complex topics for some listeners. For others, there's great familiarity. And we're going to explore all of them in the coming discussion. In September of 2017, ASCO published in the Journal of Oncology Practice a policy statement on the impact of utilization management practices specifically directed at cancer drug therapies. In that statement, ASCO outlined its opposition to payer imposed utilization management policies that restrict patient access to high quality, high value cancer care. The statement also points to high quality clinical pathways as the best first option for ensuring the appropriate utilization of anticancer drugs and the delivery of the highest quality cancer care. So with that as introduction, Dr. Ward, I'm delighted to welcome you to the podcast, and really looking forward to hearing your thoughts on utilization management. Thank you for being here. It's a pleasure. Thanks for inviting me. So to get the conversation started, can you just define first exactly what we mean by the phrase utilization management? Utilization management is classically when insurers or payers put controls over what care a patient may receive. And in our case, that usually means control of the drug therapies that they're going to get. I think that utilization management more strictly could also include when providers themselves use practices that try to strive for the highest quality care at the best price. So that already raises the possibility that there are a number of utilization management policies that payers could employ focusing, for example, on the use of specific prescription medications or other interventions. What are some of the more common utilization management practices that are being used specifically in cancer care? Well, I think the one that we run into every day is specialty care pricing. I was talking to the MedPack folks at Congress just a couple of months ago and began telling them the contortions we go through in a practice, where in my practice, we have seven docs. We have two full-time people who their whole job is to get authorization for drugs, and then to figure out how the people are going to pay for their co-pays that can sometimes be several thousand dollars a month. It's a restriction that I suspect the drug companies actually utilize to try and market their drug by providing co-pay support. At the same time, I think payers build it into the pricing of their insurance. So it's become a big part of what happens. And it's almost a dance between practices and payers. But it takes a tremendous amount of time and effort. And I'm not sure that it accomplishes a whole lot. Wow. I mean, I think there's a lot that we could unpack there. And I think for some of our listeners, it would help to frame this in even more realistic terms. We shouldn't use specific drugs or drug names. But I'm curious if you could provide a more concrete example of what exactly would happen to Mr. Smith in your office when you make a recommendation for a treatment, and what then ensues in terms of this specialty tier pricing. Sure. So Mr. Smith has prostate cancer. And he is appropriately treated with a very expensive oral medication along with his castrate therapy. And in doing so, I write a prescription. I send that prescription to a specialty pharmacy. They then begin doing a preauthorization process, or that preauthorization process may be done from the doctor's own office. They get preauthorization for the drug, but they find out that the patient has a 20% co-pay. This drug may cost $12,000 a year, and so the patient is now responsible for the other 20% of that cost. If it is a commercial payer, then the patient will-- then the specialty pharmacy will go to the drug company, and the drug company will provide co-pay support. That requires often the patient to give them a copy of their last tax return, and there will be some other requirements to show that they don't have the ability to easily pay for that co-pay. If that kind of process wasn't in place, the patient would probably in many circumstances deny the treatment and not get the best care that they deserve. If it's a Medicare patient, then the drug company directly providing co-pay support is called fraud. And so in that circumstance, the pharmacy will then-- or the practice will turn to foundations that help provide co-pay support, and try and get the patient foundation support. You're better apt to do that in January or this time of year in March than you are in November, December, because foundations tend to run out of money. So you run around scrounging up foundation support for your patient until you're able to do it. That puts delays that can sometimes last weeks before you can initiate a treatment. And not to put too fine a point on it, but in this example, you recommended a specific therapy, and ultimately the patient getting it, the delay is really in scrounging up or managing the co-pay through these various channels. Is that a fair assessment of what you've described? Or did I miss something? That's exactly right. And so the patient will almost always get the drug, but there can be considerable delays. OK. And so that answers, in a sense, the next question, that the concern about the overall impact of this kind of approach on individual patients is a delay in terms of needed therapies. And sometimes that might be a critically important one, but other times it might be honestly less critical. But it's also a lot of work. And there's a lot of expense on the side of the practice in navigating this. Is that also a fair assessment? Yes. And none of that is, of course, reimbursed. It's become an expected part of practice, but it is a burden on practices. OK. And just out of curiosity, what do you think the alternative would be? Since all of this seems to boil down to the fact that there is co-pay, and there are multiple ways of mitigating the impact of co-pay on the actual individual patient. What would the alternative to this actually be? Well, I think in a perfect world, the alternative would be that the patient would not have the co-pay, and the drug companies would admit that they're actually lowering their price significantly when they provide co-pay support. And patients would pay less. And we wouldn't have to go through the dance now. That seems, at this point in time, unrealistic. But I think that right now, the payers really have this locked into their cost of their insurance. So it is a dance that's expected. If there was a way to get rid of that, that would be great. One suggestion that has been made is that part D drugs that are cancer drugs actually get moved to part B, and that they get paid for that way for Medicare patients. And then the co-pay issue with part D drugs goes away for the Medicare patients. And then maybe some policy might follow on the commercial side. Maybe not, right? Yeah. That may follow. But the commercial side is a little bit easier, because the commercial side, you can get direct support from the pharmaceutical company for their co-pay. And you can't do that for Medicare patients. OK. So let's-- forgive the pun-- take the next step and talk about step therapy. As I understand it right now, the White House and Congress, along with ASCO and other stakeholders, we are all working-- and I think it's true at this point in a fairly collaborative way to try to talk about and start to address the high cost of prescription drugs here in the US. One part of these efforts from CMS has been to propose expanded use of step therapy. Can you describe what step therapy would look like for a patient with cancer? Sure. I think that some of us prefer the word fail first to step therapy, because that's more descriptive of what it is. Step therapy is the idea that before you can get drug B, you have to use and fail drug A. Most step therapy is based on price of the drug. And we don't think that that applies very well to cancer patients. Most of our patients, there is a best drug for them. And to have to use inferior drugs to get to the best drug is problematic, not just in time and delays, but we know that our disease evolves over time. And if you use an inferior drug first, the superior drug may not work nearly so well as a second line therapy. So in an ideal situation, hypothetically, were there two drugs that on average offered the same response rate, progression free survival, overall survival, and similar toxicities where one costs less than the other, this approach might make sense, assuming there weren't mitigating factors at the individual patient level. And it's fair to point out, I think, that in other domains outside of oncology, classically blood pressure management, step therapy has been accepted and is reasonably successful. If I'm hearing you right though, the problem we see in oncology is that kind of ideal equivalence is rare. And most of the time there really are reasons, good reasons, defensible evidence-based reasons, that oncologists select one treatment or another. Is that a fair summary? Yes, I think it is. An example where someone I think might inappropriately use step therapy would be in renal cell cancer, where we have a number of TKIs that are available. Someone may say that they all have similar response rates. Of course, none of them have been compared head to head. And so that makes it very difficult to say whether they're really the same or not. But they do have different toxicities. And so even if you would say on average their toxicities are similar, for a given patient, one drug may be better tolerated or more appropriate than another. To use those drugs in a step therapy fashion would be inappropriate, and I think would harm patients. There are circumstances where I think step therapy is easy to apply in oncology, but they are few and far between. One example where step therapy I think will be applied-- and I wouldn't have problems with it-- is in the biosimilar products. And my understanding then is that the Medicare Advantage, at least, some of the plans began allowing step therapy just at the beginning of this year, 2019. Have you begun to see or hear about any impact on patients yet as a result of that or is it still too early? I have seen payers doing some step therapy in, for example, the biosimilar realm, or even requiring you to use a filgrastim instead of pegfilgrastim. But I have not yet seen the part D payers using step therapy. I don't know whether ASCO and its state affiliate counsel has begun gathering examples across the country otherwise. The filgrastim-pegfilgrastim issue that you just mentioned, I think, is a good one for highlighting some of the subtle challenges here. It's probably fair to say that the impact on hard measures, like infection or admission to the hospital, will be indistinguishable. But in one case there are multiple either office visits or health care professional interactions or a burden on the part of the family members or patient for regular administration, whereas with the other choice, of course, there's one dose per cycle. So the price, if you will, of that convenience, I think, is another issue that will eventually bubble to the surface. Right? Yes. And the ironic thing about that step therapy is that if a patient decides that they want to self-administer the drug at home, they have to pay co-pays for the privilege of giving themselves a shot. Right. Well, we could probably fill the whole podcast with perverse incentives and bizarre quirks of the system, I'm sure, in terms of drug administration, right? I think that that's one of our problems. So look, we've identified these challenges. Of course, our job here is both to highlight them so that our members and listeners are aware, but even more importantly is to do something about that. So can you start to describe how ASCO is working with Congress and the administration to address some of these issues to encourage policies that would be better for people with cancer? Well, I think one of the first things that we've done is we've collaborated with our state affiliate societies in individual states where there has been legislation regarding some of these utilization management activities that don't make a lot of sense. There are a number of bills last year in just step therapy itself. In a number of states, I think there were a total of about 90-some odd bills that addressed some kind of utilization management in different states across the country last year. We have a similar bill that's in the legislature here in Washington state right now that is looking at step therapies, and to put some curbs on it, and more particularly, to make the processes transparent so that when you do have a step therapy in place, you know it. You understand it. And you know what literature has been used so that you have an ability to appeal it if it's appropriate for your patient. So those kind of processes ASCO is facilitating. The big picture, I think, is that we need to somehow develop a reimbursement system that actually incentivizes oncologists and payers to work together instead of being at odds over this. And that would involve developing pathways that are value based, but in our mind, very distinct from step therapy. Well, I mean, that's a natural segue to the next topic, which is, in fact, ASCO's 2017 policy statement, which addressed utilization management, and pointed to high quality clinical pathways as payers best first option for ensuring appropriate utilization of anticancer drugs. Can you talk a little bit about why we at ASCO have turned to focus on that as the tool to drive the delivery of high quality care? Well, I think there are several reasons. One, we've staked out a position that we believe that as oncologists we should be responsible for the way we use drugs, and the management of our patients' care, and that part of that responsibility should be to be cognizant of the cost and the value of the drugs we give. But we don't believe that we should be responsible for the prices that those drugs carry. A system that we have right now in buy and bill makes us responsible for that. And many of the reimbursement reforms that have been done, the oncology care model being one of them, for example, actually makes oncologists responsible for the cost of the drugs in the reimbursement scheme. We think that that is a mistake, and that there are better ways to try and leverage the cost of drugs than making us responsible for them. Right. I mean, one of the things we say is that we don't set the launch price, and we're not…

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Joint Assessment of ASCO, ESMO Value Frameworks in Focus on ASCO in Action Podcast Mar 26, 2019

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Subscribe to the podcast through iTunes and Google Play. Dr. Clifford A. Hudis (CH): Welcome to this ASCO in Action Podcast. This is ASCO's podcast series where we explore policy and practice issues that impact oncologists, the entire cancer care delivery team, and the individuals we care for-- people with cancer. My name is Cliff Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action Podcast series. For today's podcast, I am really delighted to have Dr. Lowell Schnipper, chair of ASCO's Value of Cancer Care Task Force as my guest today. In addition to his extensive service to ASCO, Dr. Schnipper is the Theodore W. And Evelyn G. Berenson Professor of Medicine at Harvard Medical School, Chief of the Division of Hematology/Oncology at the Beth Israel Deaconess Medical Center, and Clinical Director of the Beth Israel Deaconess Cancer Center. He is also an Associate Director of the Dana-Farber Harvard Cancer Center and a member of the Cancer Center's Executive Committee. Now, to provide some background for our listeners, I do want to highlight a couple of related points. First, as early as 2007, ASCO, led by Dr. Schnipper, was already focusing on rising drug and health care costs. And two, as detailed in our five-year strategic plan, almost everything we do at ASCO is aimed at helping our members and all of society achieve high-quality, high-value care for all people with cancer. Examples of the latter include our Patient-Centered Oncology Payment model, our participation in the Choosing Wisely campaign, our entire CancerLinQ project, and ASCO's Quality Oncology Practice Initiative, or QOPI, which is now available internationally. So our conversation today will focus on one longstanding project that's part of all of this effort. And this is one that's been a critical component of our efforts going back really, starting in 2007, ASCO's Value Framework. Here, I want to note that both ASCO and ESMO have developed algorithmic scales that are designed to evaluate the benefit of new cancer therapies. Again, ASCO's is called the Value Framework. And it was developed primarily as a physician-guided tool to facilitate shared decision making by patients and their oncologists as they select among high-value treatment options for individual patients. In our framework, the clinical benefit of a specific treatment and its known toxicity are combined, and they produce a score that we call the Net Health Benefit. So after years of building, testing, and refinement, we recently conducted an analysis that compared the output of ASCO's Value Framework against the European Society for Medical Oncology, or ESMO's tool, which they call the Magnitude of Clinical Benefit Scale. Dr. Schnipper, you co-authored the assessment that we're going to discuss today. And that's why I'm so delighted to welcome you to the podcast. I want to thank you, in advance, for sharing your insights with us here today. Thanks for joining us. Dr. Lowell Schnipper (LS): It's my pleasure to join you. This is a terrific opportunity to explain a bit about what ASCO is doing and put it in the context of what other groups are doing, in particular, a group like ESMO. We started this effort, as you were pointing out, approximately a decade ago, not the Value effort, but our emphasis, as a society, on the increasing cost of cancer care for our patients. And we wrote several manuscripts detailing the importance of doctors being sensitive to cost and, of course, providing the patients with the best opportunity for high-value care. As the years evolve, and some of the initiatives that you've already mentioned, Cliff, we felt it really important to develop a formulaic way of approaching how we, as an oncology community, might assess the clinical value of the drugs we use to treat patients with cancer and convey that, then, to our patients in the context of shared decision making. That's really the background of this effort. And the Value Framework itself has actually evolved over about three or four years in a number of iterations. And I'm hoping that as the discussion ensues, we'll be able to get into that in more detail. That's great. So let's actually start with the main topic today, which is the comparison between ASCO's work and ESMO's. Why, exactly, did you decide to pursue this comparison and then publish it? We became aware that ESMO was undertaking a very similar initiative, namely an attempt at developing an algorithm with which to assess the value, the clinical value, of oncologic therapies representing all of the European nations. That's about 27 nations. In parallel, but actually quite independently, we at ASCO were developing our own Value Framework. And as one can see when reading about either of them, in many ways, they're quite different. But they have overwhelming similarities, specifically based on assessments of how much good a given therapy does for the patient when compared with a control treatment and, of course, how negative any of the effects of our therapies are to that patient population. And we integrated that, at ASCO, in our Value Framework in a concept that you rightly referred to as the Net Health Benefit. Well, ESMO, we came to learn as we were readying to publish our framework, that they had worked on something very similar, looking at clinical benefit and toxicity somewhat differently. And they, of course, published theirs in the Annals of Oncology. And we published our framework in the Journal of Clinical Oncology. These frameworks were met with an enormous amount of interest by every component of the oncologic community. And by that I mean patients, of course, are exceedingly interested in this. And we can get, I hope, to the patient perspective in a little while. But in addition, manufacturers were quite interested in how we were going about assessing the products that they bring forth and we put into clinical trial. And of course, ultimately, the oncologist, him or herself, is the deciding factor in recommending or not recommending a particular therapy. So we elicited tremendous interest. And I didn't even mention the payer community which, of course, was very, very interested because of the rising cost of oncologic therapies. In fact, I will say that one of the motivating factors for the ASCO community, and I think for the ESMO community as well, is the awareness that oncologic therapies are very, very costly, sometimes eliciting huge out-of-pocket payments for our patients. And now, I speak for the American health consumer, not so much those in Europe. And this becomes a major factor in the patient's and the patient's family's well-being, as in this risk we know for financial toxicity. So we felt it very important to establish points of overlap and similarity, as well as points of difference with the ESMO framework in order to understand and improve each other's work. And so while we don't view this necessarily as a planned collaboration with which we'll modify each of ours after arguing about different points in a scholarly way, what we really wanted to do was see how each framework performs when looked at through the same lens of a number of trials. And so that led to this comparison of, well, we started out with over 100 trials and focused on 97 that were being done in a prospective randomized clinical trial context for palliative purposes, meaning for non-curative situations. And that really is the background of how it came about. CH: So before we dive into that aspect of the results, I think it might help to clarify, for just a moment more, the comparative effort on two planes. On the one plane, which you touched on, there is the actual design of the tool, what it takes into consideration, and what it weights. And on the other plane, there is how it actually evaluates specific treatment options. So can we just circle back, for a second, at a high level, what is it about the two tools that you would say is similar to overlapping? And what is it that is, perhaps, different, if anything, about the two tools methodologically? LS: Both tools are premised on using a prospective clinical trial in which a comparator is compared in a specific clinical disease setting, clinical cancer disease setting, with a test agent, or a test regimen. And so both of them look at that particular subset. And, in fact, that is the case for these 97 trials that I just alluded to. The ASCO clinical benefit, when comparing a test regimen to a control, is literally measuring the difference between the two in the hazard ratio, or the overall survival. So, for example, if the overall survival is measured in months, but no hazard ratio were given, if the overall survival for the control was 10 months, and the overall survival for the test agent was 20 months, in fact, we would register basically a doubling, or a very high degree of clinical benefit. And if the hazard ratios are such that there's a very low hazard ratio for death, meaning a high likelihood of survival, then the ASCO Clinical Benefit Score would register a large number, accordingly. Our attempt at finding, numerically, the difference between a clinical comparator and a test regimen differs from ESMO where they have reduced categorical differences. In other words, they ultimately arrive at a score of 1, 2, 3, 4, 5 rather than measuring the quantifiable difference between the comparator and the test agent. So we actually differ in how we go about defining the clinical benefit. We are trying to be a little bit more precise. And ESMO, I think, feels that that attempt at precision may not always be completely valid, because studies vary in terms of sample size and that could influence the variability around the mean or the median. So there's some technical differences, or biostatistical differences. But basically, we each are trying to develop a magnitude of clinical benefit and have done so in our frameworks. We differ from ESMO in the sense that we have developed a rather elaborate toxicity scoring system. And that's largely because we have, in developing the framework, interviewed many, many patients or leaders of advocacy groups. And they impressed upon us that it's not just grades three and four, or heaven forbid, grade five toxicity that worries them. It's often low level of chronic toxicity that also impacts on the quality of their lives. And we felt we needed to develop a tool that reflected the patient's perspective as it was explained to us. And so we have a much more elaborate, and I will confess probably more difficult to pull out of the literature and score, than the ESMO toxicity assessment in which they more or less say, this is highly toxic, not so toxic, or very minimally toxic. So we feel that we needed to do that and that it makes sense because it reflects our patient's input. And again, this is a tool we keep coming back to that's to help patients and docs make decisions. So those are a couple of the ways in which we actually differ appreciably. In addition, ESMO practically ignores progression-free survival, which, as you know, many, many prospective randomized trials use as a primary endpoint. I think we, at ASCO, completely agree it's not the very best surrogate endpoint. And as such, if [INAUDIBLE] namely, the better endpoint, is not the primary endpoint in a given trial, we will utilize the progression-free survival in our value assessment, but we actually downgrade its value. So, in other words, if the value of a score for overall survival as opposed to the value of a score for progression-free survival differs, we indicate it by about 20% less for progression-free survival. So the differences in the toxicity parameters we felt were important to maintain and the difference in the progression-free survival measurement, or I should say downgrading its importance, that, too, was also difficult to at least-- well, I should say progression-free survival turned out to be a less valuable endpoint than overall survival. And we felt quite legitimized in reducing it. ESMO agrees, but they basically give very little credit for progression-free survival unless the improvement in progression-free survival is associated with virtually no toxicity. So we differ a little bit in that. But again, we're circling around the same sets of variables, but coming at them somewhat differently. CH: All right. So let's talk about the results for a moment. That background, I think, is really helpful to audiences who are trying to put these in context. But a couple of points that I would make, and then I have a question, or maybe one point I would make is, there have been a number of prior studies that have been conducted by external researchers. They've looked at and compared the two frameworks. And they reported lower levels of agreement between ASCO and the ESMO frameworks than you and your colleagues are reporting now. What's fundamentally changed? I mean, how was your analysis performed? And why does it show a greater degree of concordance than earlier external investigators found? LS: I'm glad you asked that. That's a key question. And of course, we were initially kind of dismayed when we saw published results in which there was some degree of disagreement between the ASCO and the ESMO scales. Well, it turns out that there was one, out of about four or five that were in the literature, that actually found a high degree of concordance, although it looked at a relatively small number of trials within the same disease area-- lung cancer. So what we realized was that these are complicated frameworks to work with, that they're not trivial. And the reason I arrive at that is because we are blessed with a very, very bright, talented staff within ASCO who embraced the task of applying our Value Framework to these 103 trials. What we learned is that for people who weren't involved in generating the framework, but were called upon to apply it, they literally had to get onto a learning curve in order to produce consistent assessments after culling out the relevant data from a given paper. So we are pretty sure that the low levels of concordance really had to do with people misapplying the scales in many, many cases. And the proof of the pudding, I think, is in the eating because while we didn't find perfect concordance, and that gives us additional food for thought, we actually did pretty well, because 65% to 70% of the trials actually came out with an agreeable scoring, meaning that what ESMO concluded was reasonable to recommend incorporating into a European nations Pharmacopoeia for cancer drugs, represented the upper half above, let's say, a Net Health Benefit score of 45 for ASCO. So those 65%, 70% of trials actually scored pretty much concurrent with one another in terms of identifying drug regimens as useful. The outliers, those that differ, were actually another cause for our scratching our heads and, ultimately, teasing apart the differences. And we do understand some of the reasons why the scales are different, which isn't to say one is better than the other, but it probably just rests on bedrock assumptions in terms of incorporating various variables into the Value Framework. CH: So that's great. A couple of follow-up questions, I suppose. And I'm just curious, either from the ASCO side or, if you know, from the ESMO side, based on this effort to compare them, is there any plan to actually tweak them to increase their alignment? Or is everybody happy that they're each fulfilling their planned functions and this little bit of discordance is OK? LS: That's a great question. I can say that, at the very start, this goes back a couple of years, I had meetings with counterparts at our ann…

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Exclusive Interview: NCI Director Talks Big Data, Clinical Trials, the Cancer Research Workforce—and Why He Lives to Conquer Cancer Mar 06, 2019

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Subscribe to the podcast through iTunes and Google Play. Dr. Clifford A. Hudis: Welcome to this ASCO in Action podcast. This is ASCO's podcast series where we explore policy and practice issues that impact oncologists, the entire cancer care delivery team, and the individuals we care for-- people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO as well as the host of the ASCO in Action podcast series. For today's podcast, I am delighted to have as my guest Dr. Ned Sharpless, the director of the National Cancer Institute. The NCI is the largest funder of cancer research in the world, and it has helped to drive many of the major prevention and treatment advances we've seen over the past 50 years. This includes things like HPV vaccination and the identification of the link between HER2 status and breast cancer outcomes and treatment, as well as new discoveries that have dramatically improved outcomes for childhood cancer. Dr. Sharpless, welcome, and thank you for joining me today. Now, we really have a whole lot to discuss, but before we get to our planned topics, I have to jump ahead and start with the president's State of the Union address, when President Trump mentioned that he wants to see $500 million appropriated for childhood cancers over the next decade. Can you talk a little bit about how you expect that, specifically, to play out? What will the NCI be able to do with those new specified funds for pediatric research? Dr. Ned Sharpless: Sure. I think childhood cancer-- childhood cancer is an area where the National Cancer Institute has had a long interest and a robust portfolio of research. And I think it is an area where we've made some progress, in terms of mortality, over the last few decades. But you have to say two things about childhood cancer. While progress has been good, and we're making-- more kids are surviving cancer therapy today than ever-- there's still a long way to go. Too many kids dying of cancer in the United States, and even the kids that we're able to cure have these significant lifelong survivorship challenges, in some cases. So the therapy that is curative may leave patients with side effects of surgery and chemotherapy and radiation for the rest of their lives. So better treatments for kids and less toxic treatments for kids are what we are really looking for. And with that amount of money, I think a good-- the thing that it appears to me that one could do to most quickly move the needle in childhood cancer-- which, as you know, is a collection of less common cancers, even rare cancers-- is really a more intentional effort at aggregating and using and linking clinical data with molecular data and other sorts of patient data, so that we can really learn from every child with cancer in the United States, so that we can really figure out what's working in certain populations and then disseminate that information as rapidly as possible-- without having, in all cases, to rely on slower clinical trial structures that are challenged for certain populations where accrual can be difficult. So I think that is the vision for the president's initiative, is to, with additional funding, allow for very aggressive, intentional, and organized data linkages and data aggregation so that we can learn from every trial and therefore treat every child's cancer in a better, more effective way. Dr. Clifford A. Hudis: You know, I think that's great. And that actually provides two different segues-- one I'm going to pick up right now, and one I want to come back to. The first is about data-- big data, specifically. We'll come back to that. The second is about the way the pediatric oncology community for years has really led in designing studies that could accrue the majority of children diagnosed with various specific diseases. And that leads me, that idea of eligibility and the structure of research, to ask about the way that you're thinking about modernizing clinical trials. This is something I know you wrote about in JAMA Viewpoint in the last couple of months. You addressed financial pressures, the need to increase overall rates of accrual to the trials, especially representing patients from underserved populations. Can you expand a little bit on that effort and what kind of progress you see as possible in the coming months and years? Dr. Ned Sharpless: Yeah, I think everything we do successfully in cancer today is in some ways the results of a clinical trial. And this is clearly one of the most important things the NCI does, in terms of moving basic science into patient care through experimental clinical trials. And it's an area where we-- frankly, a lot's changed in the last couple of decades. When I was a wee fellow, the clinical trials apparatus was very different from the way it is 20 years later today. And we need to make sure that we modernize the clinical trials process to keep up with the changes in our understanding of the biology and the new kinds of therapy we have for cancer. So that brings up a bunch of items that are areas where the NCI is really doing a lot of things. So, for example, one of the first problems I noticed when coming to the National Cancer Institute was that the clinical trials infrastructure, the big networks that we have for doing these kinds of trials, were under-resourced, that they had a funding problem. And they were becoming non-competitive with the trials sponsored by industry. And this showed itself in many ways, in accrual fees for patients, or the wait times to get the trial open, or the slow accrual once the trial was open. And so they were laboring under a number of problems. And so we decided we had to invest in the Clinical Trials Network and have been doing that and will be continuing to doing that in a number of ways-- through direct funding to attempt something like the National Clinical Trials Network or the NCORP, for example, the NCORP organization, but also by additional funding for biobanks and data aggregation initiatives, targeted clinical trials, et cetera. I think we've also-- there are some structural problems with the clinical trials that you alluded to. For example, eligibility criteria, I think, hadn't really kept pace with modern clinical trials. And I think ASCO and other groups have played a really important leadership role in identifying what are good eligibility criteria and which ones are not as necessary anymore. And then, do we have to have the same criteria in all the trials, and be more thoughtful about how those are used as a way to enhance accrual, because often we have a-- superfluous eligibility criteria can limit accrual. And increasing accrual by a variety of measures is really important. And we've thought a lot about how to do this through novel ways of clinical [? house ?] matching. I think one of the more successful efforts we've had in clinical trials accrual recently has been the MATCH trial, the NCI MATCH trial, which was able to accrue 6,000 patients at 1,100 sites in the United States, filling a targeted accrual two years ahead of schedule. It's the fastest-accruing trial in the history of the NCI. And I think one of the things MATCH teaches you is that if you have an interesting trial that's written in a nimble way that is open in the community-- that patients don't have to drive six hours to a cancer center, but can go to a local NCORP site, for example-- then those trials will accrue. We can accrue quickly, and we can accrue underserved populations, and we can accrue rare cancers. And that framework is more nimble than, say, the large phase III randomized trial run only at cancer centers that we had 10 years ago. There is still a role for large, randomized, phase III trials. The NCI is not backing away from that, or where we will support those. But I think, as we discussed in the JAMA piece, we really have to be thoughtful about where the NCI needs to be involved with those kinds of trials, compared to which of those should be supported by industry, for example. Dr. Clifford A. Hudis: It sounds like you're alluding to something I think you and I discussed even when you first got into your current role, which is the identification of those trials that industry should run, essentially, itself, and those trials that the NCI should support as complementary to industry trials. Can you expand a little bit on how you see that distinction and where you draw that line? Dr. Ned Sharpless: Yeah, the thing to know about clinical trials in oncology in the United States right now is most are actually paid for by industry. There's a huge pharmaceutical industry spend on clinical trials, and from my point of view, that's great. The fact that industry is paying for trials to develop therapies for cancer patients-- that's less money the NCI has to spend on those same questions. So we think that's a wonderful development and healthy for cancer research. But if that's the way it's going to be, then the NCI has to ask itself-- for the precious moneys that we have to spend on clinical trials, we need to use those in a way that's maximally effective and, in particular, not duplicative with what industry sponsors are doing. It's important to say, we do a lot of work with industry. So it's not just us either-or. Many of our trials, through these agreement processes called CRADAs, allow us to do trials with pharma sponsors and use their compounds in our trials. And that's a real boon to our research effort, as well. But there are certain kinds of trials that are very important where we really want to know the answer, but they're a bad fit for what industry is going to fund. For example, a de-escalation trial-- that's a trial where there's a standard of care that's pretty good, but the therapy is toxic. And so we'd like to see if we can get the same good outcome in a population using less aggressive therapy. A very important example of this was the TAILORx trial recently, where we showed that based on a genetic risk score, an RNA-based risk score of the breast cancer, women with estrogen receptor positive breast cancer-- many of them could forego cytotoxic chemotherapy and just take anti-hormonal agents and have the same good outcome in terms of their long-term survival. So that's a trial that is not going to be industry-led, for a variety of reasons. But I think it is the kind of question that's really important for patients. It's important, also, to say that de-escalation trials are hard to do. They require a lot of thought. They don't always work. And so they require these comprehensive thoughtfulness and infrastructure that the National Clinical Trials Network can provide. So an additional example is these multi-modality trials we have, where maybe two different agents come from two different pharmaceutical companies, and then there's some surgery and some radiation. There's very complex, multi-integrated care. And those can be very hard for a single sponsor to run, but, again, can be a very good fit for the NCI. And there are many other examples like this. But I think the real question we have to ask is, if our budget is limited and finite, what are the trials that the NCI really should do and lead on? Dr. Clifford A. Hudis: Yeah. And I think one of the points there is you need to conduct-- we need to conduct-- trials as efficiently as possible, getting the most so-called bang for the buck. You alluded to the fact that the NCI, along with ASCO, has been working on making trials essentially more efficient by making them more representative of the actual cancer population we end up treating. And a specific area of focus for us at ASCO, in this collaboration and also in our TAPUR trial, has been driving the eligibility age down below 18. My understanding is that this is something that you're adopting as a recommendation across the NCI, as well. I guess my question is, how broad and how quickly do you expect to see this implemented? Dr. Ned Sharpless: We have a number of efforts related to these barriers to accrual. You mentioned age as one of them and other sorts of exclusion criteria. And we've looked deeply and thought about this sort of care across the continuum of life-- both age limits on the less than 18 side, but also at the greater than 65-year-old side, where we see, often, eligibility criteria structured around a maximum age that don't often make a lot of sense. So that is one of several topics that we are addressing. As you know, we have a variety of networks and programs, and we fund a variety of kinds of trials. Some are led predominantly by the academic institution. Some are led through NCI networks. And so we are rolling out these policies, not in a one shot fits all way, but across these networks at different scales. They often require scientific buy-in from the other participants, and you know how that process works. I think this is an area, fortunately, where there is a lot of buy-in, where we're not having lengthy debates about whether or not we should do this. Really, the question is how we operationalize it and make it happen as quickly as possible. Dr. Clifford A. Hudis: That's great. And you know how strongly supportive we are, on lots of levels, for this effort and the related ones, in terms of barriers to accrual. I want to pivot, though, back to something that you introduced earlier about the big data. And my understanding is, in the annual plan and your bypass budget for 2020, you specifically called out the need to harness big data to speed up all of our work across the cancer research enterprise. And there are many companies, organizations-- we ourselves at ASCO have CancerLinQ-- that are involved in trying to collect data, share it, analyze it, and advance science and clinical care. But what exactly do you see as the NCI's role in facilitating this, and what do you think is our biggest challenge going forward? Dr. Ned Sharpless: Yeah, it's an interesting topic. I think the-- it's maybe two things to say off the top about big data in cancer research. The first is the NCI already has one very important example of how big data can transform a field, and that's The Cancer Genome Atlas, which later became the Genomic Data Commons. This is petabytes of genomic data that we make available in the cloud now to any researcher, basically, who is interested in cancer. And that set of data has led to thousands of papers and just a fundamental reorganization of how we think about cancer biology in many ways. And it's been a huge success, I would argue, and well worth the investment of the NCI to do it. And the data has been used in ways we never envisioned. We never thought of some of the papers and applications that would come out of the analysis of the Cancer Genome Atlas, for example. But the problem, then, one quickly sees, is that while that data set is great, it's limited. It doesn't have the clinical data, it doesn't have radiology and histology, it doesn't have-- we don't really have a way of binning big epidemiologic cohort data, for example. So the GDC, the TCGA, the Genomic Data Commons, proves how useful these kinds of data aggregation efforts can be, but also makes very clear what the shortcomings of our modern efforts are. The second thing to say is that this is a problem where the NCI is well-poised to be a leader, right? There are a number of issues around data sharing and data aggregation that really benefit from a Switzerland-like federal entity, a non-conflicted, dispassionate entity like the NCI that just wants to create the data structure in a way that's maximally beneficial for everyone, so that there are-- this is an area where the imprimatur of the federal go…

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ASCO Chief Medical Officer Highlights Top Clinical Advances and Nine Research Priorities to Accelerate Progress in New Podcast Feb 12, 2019

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Subscribe through iTunes and Google Play. Welcome to this ASCO in Action podcast. This is ASCO's podcast series where we explore policy and practice issues that can impact oncologists, the entire cancer care delivery team, and those individuals we care for, people with cancer. My name is Clifford Hudis, and I'm the CEO of the American Society of Clinical Oncology. I serve as the host of the ASCO in Action podcast series. And today, I am really pleased to have as my guest Dr. Richard Schilsky. He is ASCO's senior vice president. And chief medical officer, and Rich is here to talk about our new clinical cancer advances report, which was just released. In clinical cancer advances, ASCO identifies the most important clinical research advances of the past year across the full range of cancers, and from prevention to screening to treatment and survivorship. The report also announces what ASCO has identified as our advance of the year. And for the first time in this year's report, we will debut what we believe are the research priorities with greatest potential to advance progress against cancer. Rich, welcome and thank you for joining me today. Thanks, Cliff. Great to be back. now let's start with what we always do. Every year, we announced the advance of the year, the one area of clinical cancer research that has demonstrated the most significant progress in a year's time. And we've seen tremendous progress in the treatment of rare cancers, earning it the 2019 advance of the year recognition. Rich, can you talk about why this area was chosen? Why is this particular line of research so important for individuals with cancer? Well, first, let me start with a definition of what we mean by rare cancers. And generally, what we're talking about are cancers that are diagnosed with a frequency of less than six cases per 100,000 cancer diagnoses each year. Collectively, though, because there are many kinds of rare cancers, overall rare cancers comprise about 20% of all new cancer diagnoses every year. But those numbers may not even tell the full story, because as we learn more and more about the molecular subtyping of cancer, what we're learning, of course, is that there are many very, very rare mutations and fusions and other genomic alterations that occur in a very small proportion of even common cancer diagnoses. So the patient with lung cancer, who has a RET fusion that occurs in about 1% of all lung cancer cases, that begins to become a very rare subset, even though it's overall a common disease. So we're going to be dealing more and more with this general area of rare cancers. But the reason that it's so important to single this area out, of course, is because historically, we haven't been able to learn very much about these rare cancers, simply because they are rare. There aren't very many of them that occur each year. Therefore, they're difficult to study. It's difficult to complete clinical trials. It's difficult to find patient samples to be able to understand the underlying biology of these diseases. And yet, many of them are refractory to standard treatments. Many of them have a very aggressive clinical course. And for patients who are affected by one of these rare cancers, they desperately need new treatments. And this year, we're seeing, for the first time, some real progress being made in a number of these rare cancers. I'll give you some specific examples that we called out in the report. So for example, although thyroid cancer is a very common form of cancer, anaplastic thyroid cancer represents only about 2% of all thyroid cancers. And of those anaplastic thyroid cancers, about 16%-- so now we're talking 16% of 2% have a BRAF mutation. But it's clear now that treatment with BRAF directed therapy produces a very high response rate in this rare group of individuals who have BRAF mutant anaplastic thyroid cancer. Another example, take the drug we're all familiar with, trastuzumab. We know now, of course, that trastuzumab is effective not only in breast cancer, but also in gastroesophageal adenocarcinoma that's HER2 amplified. And there's emerging data that HER2 directed therapy may be active in other tumor types, where the HER2 gene is amplified. And one of those is uterine serous carcinoma. So uterine serous carcinoma is a rare subtype of endometrial cancer. And about 20% or 30% of those patients have a HER2 amplified gene driving their tumor. And trastuzumab has been shown to be an effective therapy in those patients as well. Last example I'll give you right now. A tumor that most oncologists probably will never confront in their practice, tenosynovial giant cell tumor. This is a very rare soft tissue tumor that occurs in the joints, typically of young adults, typically is refractory to all standard known cancer treatments. And yet, this year, we saw very promising results reported for a new class of anticancer drug, a CSF 1 inhibitor, called pexidartinib, that produced a 40% objective response rate in patients with these advanced tumors, compared to a placebo treated control group. So we're starting to make real progress in treating these rare cancers, particularly when we can begin to understand their underlying biology, and develop a therapy that's directed at the key drivers. It sounds to me-- I mean, in listening to that wonderful list of successes, that we've rolled up a process and an approach to drug development and science into a category that is appropriately called rare cancers. Because when you think about the way you presented it, which I think is lovely, first, rare cancers, as a group, aren't rare is what you said. Two, rare cancers cross from the rare histologies to include some of the common histologies. But the underlying theme, if I think about the way we present this, is a deeper understanding of the driver mutations, allowing us to move a little bit off of histology towards genomics to define these diseases. And that's not to say that genomics is the only way we're going to make progress. But the unifying theme in these cancers is probably that shared trait of an alteration and a driver mutation and an available drug. And that's the advance that's helping us with rare cancers. Is that a fair roll up of that? I think that is very fair. And you can think about it in terms of a common histology, like lung adenocarcinoma, having a large number of rare genomic subtypes, each of which comprises a rare cancer, if you will. Alternatively, you could think about it as in the trastuzumab example, of saying, well, if you look at the universe of HER2 driven tumors, those HER2 driven tumors comprise a whole bunch of different histologies. But they all are responsive to HER2 directed therapy. And so you know, as we understand the underlying biology of cancer much more clearly, it's moving us away from the long held view that the way you diagnose cancer is looking under the microscope. And if you see something that you see only very rarely, you say it's a rare cancer, to we're not going to interrogate the cancer if we see a rare genomic alteration that occurs infrequently in the population of cancer patients. That's what we're going to call a rare cancer. Yeah, I just say think it's almost like an introduction or a preview of an interesting future where more and more of the cancers we treat may be selected on this basis, rather than their conventional light microscopy appearance, right? To be sure. I mean, we know still that context is important. Not all of these molecular drivers behave in the same way in every tumor type. We already have examples where not all the targeted therapies work equally well against the same alteration, again, in every tumor type. But slowly, but surely, I think the science is moving us toward a day where we will be identifying cancers, primarily, if not exclusively, by their genomic profile. It may not be a single driver. It may be a signature. But that is ultimately is what we use to direct therapy. So to some degree, this is a fulfillment of a multi-year view that we've had about where to invest in cancer research, and the fruits, I think, are obvious. But this wasn't the only advance that we reported on last year. It's the one we named as the advance of the year. But what were some of the other advances that we called out for recognition? So as in the last several years, where we named some aspect of immunotherapy as the advance of the year, this year we continue to see progress in immunotherapy of cancer, particularly with extending the range of indications for many of the immune checkpoint inhibitor drugs, as well as new indications for CAR T cell therapies. So that continues to be a rapidly emerging area where there's a lot of progress continuing to be made. We continue to make progress how with the introduction of second and third generation targeted therapies. We've come to understand, of course, that many targeted therapies although they work well for a period of time, cancers ultimately develop resistance, patients need additional treatment options after the first line of targeted therapy. And of course, the science has responded by giving us the insight as to the mechanisms of resistance, which has led to the development of second and third generation inhibitors that can effectively overcome the treatment resistance. We're seeing this particularly in lung cancer, particularly with drugs recently introduced like osimertinib, which is effective against the T790M mutation, the common resistance mutation in EGFR mutated non small cell lung cancer. And interestingly, some of these drugs are also now showing much greater effectiveness in treating or even preventing the onset of brain metastases in lung cancers that commonly spread to the brain. So this has opened up actually a whole new area of research on effectively treating and preventing brain metastases in those tumor types, where there's a high propensity for such metastases to occur. The last thing I'll mention as another area of continuing progress is the continuing development of new biomarker strategies to help us refine the way in which we select patients to receive treatment. And certainly, this last year, the big news were the results of the so-called TLRX trial in breast cancer, a test, a gene expression profiling test, that clearly indicates that there is a substantial proportion of women with hormone receptor positive early stage breast cancer who can safely forego treatment with adjuvant chemotherapy with no detrimental effect. And this type of test I think is now going to really move us even further down the road of precision medicine, because it's allowing us to identify those patients who are most likely to benefit from adjuvant chemotherapy. They should get treated, and they will certainly benefit. But it also is allowing us to identify those patients for whom adjuvant chemotherapy is unnecessary, and who can be spared both the physical toxicity and the financial toxicity of adjuvant treatment. The more of those tests that we can develop, going forward, the better we'll be able to refine prognosis, the better we'll be able to apply adjuvant therapy in the future. I think one of the subtleties here is this highlights something we almost touched on before, which is precision medicine doesn't have to be only about gene rearrangements. There are multiple paths towards some degree of precision in treatment selection for individual patients. And this is, I think, a good example of that. It also is a good example of the fact that precision medicine is not actually just about treatment selection. It's about risk assessment, risk stratification, assessment of prognosis, identifying early recurrence, as well as directing patients to the right therapy at the right time, based on the biological characteristics of their cancer. So one of the things that we've done this year, and it's a first for us, is to announce a set of research priorities. These represent areas that our leading volunteers and others have identified as needing urgent attention. They are areas where the progress is promising, but not fulfilled completely. Can you talk a little bit about the motivation for creating this kind of a research agenda, as well as a criteria for actually selecting the specific research priorities? So obviously, you know, our field is advancing very rapidly. But there are still very many unmet medical needs. There are many clinical conundrums that oncologists face every day in practice. And we felt that given all the potential directions that research could take, ASCO is in a strong position to be able to at least begin to describe those areas, where we thought the potential benefits in patient care would be greatest, and could be realized soonest. ASCO, because we are the physicians who treat patients with cancer, we have a pretty good sense as to what the unmet medical needs of our patients are, what the lack of evidence is that our doctors struggle with every day in making clinical decisions with patients, where the field needs to continue to grow and to develop new information, to help fill those evidence gaps. So we felt that we could take a stab at setting a research agenda, and putting out there where we thought the unmet needs, where we thought the opportunities were ripe for investment in research, and trying to articulate how, if we were successful in fulfilling those research needs and priorities, the field would ultimately be transformed. So that's what we've done with the nine research priorities that we are offering this year. So the nine priorities that's important for readers in a moment, if they go look at this or pick up our publication to recognize, they're not rank ordered. They just happen to be nine. Maybe next year, there'll be fewer or more. And the second thing is in no particular order, as I understand it, we've divided them into a couple or maybe three big buckets. One is essentially the issue of who really benefits from IO, the advance that you already talked about, as a multi-year call out from us. The second is really a little bit about health care disparities and precision medicine all rolled up in the concept of special populations. And related to that is access to research itself. And then the third is something which we always worry about, but have, I think struggled with as a field for decades, and that is reducing cancer risk, along with screening, which is surprisingly still controversial in many settings. We'll talk a little bit more about some of the specifics, but I would just remind everybody listening that you can find a list of these nine research priorities if you go to our website asco.org/cca. So Rich, as you think about the nine areas that are rolled up in those three broad areas, can you talk a little bit about how specific research would potentially transform patient care? And you've set a relatively short timeline for results in introducing this. And what kind of resources might these projects need? If you take the first area, for example, of essentially getting the right treatment to the right patient at the right time, you know, we've touched on some of these themes already. Look at the results so far with immune therapy for cancer. It's remarkable that a significant, although still small fraction of patients across multiple tumor types, who receive an immune checkpoint inhibitor, will have prolonged disease control, 20% or so of patients apparently surviving, without disease progression, or even disease free for many, many years in melanoma and diseases that previously were death sentences for patients. The question is why is it only…

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Exclusive Interview: FDA Commissioner Talks Drug Pricing, Expanded Access, and Tobacco Jan 29, 2019

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Subscribe to the ASCO in Action Podcast through iTunes and Google Play. "Welcome to this ASCO In Action podcast. This is ASCO's monthly podcast series where we explore policy and practice issues that impact oncologists, the entire cancer care delivery team, and the individuals we take care of, people with cancer. My name is Clifford Hudis, and I am the CEO of ASCO as well as the host of the ASCO In Action podcast series. For today's podcast, I am delighted to have as my guest Dr. Scott Gottlieb, the commissioner of the United States Food and Drug Administration. The FDA of course plays a critical role in the delivery of high quality cancer care by reviewing and approving cancer treatments. This continues to generate discussions about the pace of scientific advances, and indeed the regulatory role of the FDA. Given that, we are really lucky to be able to talk today with Dr. Gottlieb about the FDA's efforts to increase overall efficiency by updating or modernizing aspects of our clinical trials conduct and expediting the end to end drug development process. I will admit that I'm going to take advantage of this opportunity to also ask how his agency is tackling the issue of tobacco control for the next generation of tobacco products, another area of deep concern from our community and others. Dr. Gottlieb, welcome and thank you for joining me today. Thanks for having me here. Great. Now as a professional society, we are very focused on the intersection of science and society. Given that, and noting that you've been at the FDA for more than a year following a long career in public service and in private industry, I have to start by asking, for you, has your current experience changed or evolved your view of the FDA's role or functions, and if so, how. I've been at the agency three previous times. I had a good sense of what the agency's public health mandate was and what its mission was. I think that the nature of the market and the science that we're grappling with has certainly forced or compelled the agency's mandate to evolve. I think what we're seeing right now is, I feel like we're at the inflection point with respect to a lot of new opportunities from technology. We look at things like gene therapy and cell-based regenerative medicine. Those fields largely didn't exist last time I was at the agency, and now we're an inflection point where we're going to see gene therapies approved to the market, and we saw three CAR-T's approved already, that are going to fundamentally transform the treatment of disease. When I was last here, we were talking about the ability to advance genomically derived drugs and have more targeted approach to the treatment of patients where you can get the right drug to the right patient at the right time with sort of a drug diagnostic system, yet seeing some early examples of that. But now that is a much more routine development pathway. With respect even to tobacco, you mentioned tobacco at the beginning, we're at a point right now where we have the opportunity to use new technology potentially to help currently addicted adult smokers transition away from combustible tobacco products onto products that we presume don't have all the same risks associated with them. And so using new authorities we have to regulate tobacco, including regulate nicotine levels in combustible cigarettes to render them minimally and not addictive, and allowing for a regulatory pathway that puts some of the new technologies like e-cigarettes through an appropriate series of regulatory gates, we have the opportunity to transition adult smokers off of combustible tobacco with all their morbidity and mortality associated with combustible tobacco use more rapidly than we did in the past. And so across the board, I think we're seeing technological inflection points. Digital health tools are another example that are not only creating significant new opportunities, but I think compelling the agency to rethink its traditional approach to regulation in order to accommodate the opportunities that these new technology platforms create. So is it fair to sum all that up as, the agency is recognizing that technology in many domains is defining and driving the need for new regulatory frameworks, and in turn, we have to educate Congress and others to make sure that the agency actually has the appropriate authorities. Is that the virtuous cycle you're describing, you think? I think that's exactly right. I think that there are areas of profound technological change where the traditional approach to regulation doesn't apply well. Digital health tools, probably a very obvious example where you can have sort of practical incentives, where you have a digital health tool, you might evolve it almost on a daily if not weekly basis in the marketplace. It's a medical product that's a digital health tool, like a medical lab, for example. And a regulatory process that requires you to come in and file for premarket approval every time you want to make a modification or have to file a 510(k) supplement, that is antithetical to the rapid cycle of innovation and evolution that those kinds of products undergo. So we had to think of a new regulatory paradigm for how we would treat these products, and that's where we move towards the Pre-Cert model, where effectively what we're doing is validating the underlying architecture of the platform, of the software platform, and validating the SOPs, how good is the company at certifying its own software and validating its own software products, and then we would allow them after an initial approval to come to market with modifications as well as subsequent approvals without having to seek premarket clearance from the FDA every time. And we would shift toward the postmarket regulatory regime for subsequent products. So basically, instead of regulating the individual products, in essence we're regulating the firm and taking a firm based approach. That's an example of where we've had to rethink our regulatory model in order to accommodate a much different approach to innovation. Another example is with cell-based regenerative medicine, where we have very clearly said that we think that there's a lot of opportunity with cell-based regenerative medicine, but we also see a lot of clinics promulgating products based on what we think is incomplete if not poor science and creating substantial risk of patients using cell-based products intrathecally, or for injections into the eye, where they're creating substantial risk and don't have really a scientific basis to argue that there's convincing evidence of a benefit. And these products are clearly subject to FDA regulation. They cross the line between what is and isn't regulated by FDA, but subject to enforcement discretion of the agency over many, many years. Prior to when I came in here, the agency didn't actively regulate these products. We have said very clearly, we're going to actively step in to regulate this field. In fact, it's going to take a number of enforcement actions, and we'll be taking others. At the same time, we also recognize that a lot of these technologies are being promulgated by small developers, and there's a lot of promise here. And so we've had to again come up with a more accommodative approach. But how do we regulate a field where a lot of the really interesting innovations are being brought forward, for example, by academic investigators working in small clinics. And so what we've said in that case is that we'll allow investigators to pool their data so long as they follow common manufacturing protocols that are doing similar things with cells. And it can file a common BLA, common Biologics License Application. And then we'll give individual licenses to the individual institutions or individual investigators. That's a much different approach to regulation than what we've traditionally done where you think of, we regulate companies, we regulate a biotech or a pharmaceutical company. We had to ask ourselves, how do we regulate small clinics or even institutions, academic investigators in institutions, who want to promulgate these technologies. And we've come up with an approach to do that. So at the same time that we've said we're going to be taking more enforcement action to make sure patients aren't being put under duress, we're also going to take a more accommodative approach to allow for regulatory approval for products that are being, in many cases, promulgated by smaller entities and individuals. So that actually lets us pivot, I think, to an area of traditional focus for at least a large group of our members at ASCO. And that would be drug development. And it's clear that you've made it a priority to streamline how new drugs specifically are reviewed and approved. And I think as part of that effort, recently you announced a new office. It's the office of Drug Evaluation Science. And my understanding is the goal is to centralize performance metrics like biomarkers, patient reported outcomes. How do you see this new office specifically helping to support this goal of a more standardized and ultimately more efficient and faster review process? Well, the Office of Drug Development Science, the goal there is to create an infrastructure here that will help better validate scientific tools that are being used to help advance drug development, like patient reported outcomes, like bioinformatics. What we've seen is that these tools now, there's a lot of hard science behind these tools. And they've become much more commonly used in drug development programs. And so we need an infrastructure here that not only provides for a more standardized approach to assessing these modalities when they're incorporated into applications, but also helping to advance the science of how to use these tools. I compare it to what happened in 2003 with modeling and simulation. And I was here during the time period. What we were seeing over that time period was we were seeing more drug developers starting to use modeling and simulation as a component of their overall drug development programs. And we saw modeling being included in applications. Early on, it was often used for dose finding because you wanted to give the dose finding trials. But then you wanted to use the data that you derived from the dose finding trials to simulate what would happen if you picked a dose in between the two doses that you might have tested. And so we said to ourselves, well, this is very interesting. This could really help inform drug review better, give us more information about safety and benefit. We need some standard approach to how we're going to both evaluate these tools as well as help develop them into a harder science so it could be more rigorously used in drug development. We created a Modeling and Simulation Office when I was here. Mark McClellan was involved in doing it. He helped recruit the guy who stood it up, him and Janet Woodcock. It was Larry Lesko. And it started as a two-man office. Now, we've got probably 30 or 40 people in the Modeling and Simulation Group in Cedar. And well more than 95% of all applications that we get for new drugs contain a component of modeling and simulation. It's now a routine part of drug development. And we have a rigorous approach to evaluating these components of the applications as well as helping to evolve the field through multiple guidance documents that we've put out. I see the Drug Science Office, this new office that we created within the Office of New Drugs, working in a similar way where it's going to be a holding office, if you will, for new areas of science that can help improve tools used to inform us about the risks and benefits of new products. So I want to pivot from that to one of the big societal issues. And you and I have been discussing this informally, I think, for more than a year-- drug price. The Trump administration has made it a priority to address the cost of drugs, price specifically. And I guess the question from many will be how confident can we be that ultimately a faster and more effective drug development process will itself actually and favorably impact the costs of drugs. What's your feeling about that, knowing everything that you're doing that's supportive in that way? Well, I see my role in the drug price debate to be focused around creating product competition. You have price competition. But you can't price competition without product variety and product competition. And we're focused on creating the product competition by facilitating entry into the market of generic drugs, but also, facilitating a pathway that allows for follow on innovation within categories. And what we've seen-- and we've analyzed this. We're going to be publishing this data soon. But what we've seen over time is that second to market innovation within a category is coming to market much more slowly. We looked at a cohort of approvals from the early 2000s. And then we looked at a more recent cohort of approvals over like a five-year period. And it's very clear that when you look at areas of unmet medical needs, orphan drugs, first in class drugs and oncologies, the second to market innovation is taking much longer to come to market and more categories of drugs are remaining sole source drugs in perpetuity. So to the extent that you're not getting that second to market innovation for new drugs, that is thwarting the opportunity for price competition within those categories. Because you do see price reductions. Oftentimes those price reductions come in the form of rebates that aren't transparent to the consumer. But there are price reductions or discounting nonetheless when you have a second and third to market drug within a category. And the hepatitis C category was the best example of that. We saw a very dramatic price reductions in negotiations once you had multiple entrants in that category. So we're focused on that. And we're asking questions about why it's become harder to bring second to market innovation to patients. And I think that there are some very specific reasons and there's things we can do to help address that, to make it less costly to bring second to market innovation in an area of unmet medical need. If the trials are onerous or very costly to bring that second to market drug to the market, sometimes the economic opportunity might not be robust enough after this one entrant, especially when you're talking about very narrow niche categories of unmet medical need, they're might not be enough economic opportunity to incentivize that second to market innovation. I think where-- just to sort of close, I think where this might be most evident is in some of these inherited diseases. You've seen this play out in gene therapy, for example, where once you come to market with a treatment and you treat the prevalent population, the people who already have the disease, the incidence population, the number of people who are going to get it on an annualized basis, that might not be a big enough market to support a second entrant that's going to split the market with the first in class product. And so I am quite literally seeing investors pull out of these opportunities which we see what we think are applications being slowed down. We see people pull out if they think they're going to be third to market. And that's ultimately bad for patients. Because it's not just robbing patients of product competition, but it's also robbing them of potential product variety. And we know no…

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ASCO Podcast Coming Soon: Exclusive Interview with FDA Commissioner Scott Gottlieb Jan 25, 2019

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Don't Miss: Exclusive interview with FDA Commissioner Dr. Scott Gottlieb by ASCO CEO Dr. Clifford A. Hudis on the next ASCO in Action Podcast, coming January 29.

The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience and conclusions. The mention of any product, service, organization, activity or therapy should not be construed as an ASCO endorsement.

This is Cliff Hudis, CEO of the American Society of Clinical Oncology and host of the ASCO in Action podcast series. For our next podcast I will be speaking with Dr. Scott Gottlieb, Commissioner of the United States Food and Drug Administration. The FDA plays a critical role in delivery of high-quality cancer care by reviewing and approving new cancer treatments. This discussion is sure to be very interesting. It's going to highlight what the FDA is doing to modernize clinical trials and expedite drug development, and with all of that I urge you to tune in for upcoming information about this special AiA podcast with Dr. Scott Gottlieb.

What You Need to Know About the Final 2019 Medicare Physician Fee Schedule and Quality Payment Program Rule Jan 08, 2019

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Welcome to this ASCO in Action podcast. This is ASCO's podcast series where we explore policy and practice issues that have an impact on oncologists, the entire cancer care delivery team, and most importantly, the individuals we care for-- people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action podcast series.

For today's podcast, I'm going to give our listeners a quick update on an important announcement from the Centers for Medicare and Medicaid Services. In an August podcast, I outlined the proposed Medicare Physician Fee Schedule and the Quality Payment Program Rule for 2019. This is commonly referred to as the Physician Fee Schedule. Today, I'm going to provide an update on where we are with this for next year. I have to say in passing, it's probably a good day for me not to have a guest, because I'm here with a terrible cold.

So what is the 2019 Medicare Physician Fee Schedule? This is a fee schedule which consists of a complete listing of all of the fees that Medicare uses to pay doctors or other providers and suppliers. It's a comprehensive listing of the maximum fees. And it's updated each year and then used to provide reimbursement to physicians and other providers working on a fee-for-service basis.

Now at ASCO, we, every year, review this rule very closely. And we try to determine and predict the impact that it will have on our members, and of course, on our patients. There are three provisions in particular that we want to highlight today. The first of these is related to care provided in calendar year 2019. And CMS estimates that there will be, overall, a 1% reimbursement cut for hematology and oncology, as well as radiation oncology specialties. It is important to note, however, that the actual impact on any individual physician or physician practice will depend on their mix of services-- that is, what it is they exactly provide and bill.

Now the administration has publicly stated its aim to reduce the growing administrative burden that we've all been noting and complaining about for the last few years. And the second item we want to point out is there is some evidence of their sensitivity to this issue in the 2019 fee schedule. They intend to reduce the documentation required for evaluation and management services, frequently referred to as E/M.

What CMS did is finalize provisions that consolidate E/M payments. And ASCO had expressed concerns about this previously, which the agency acknowledged, along with other stakeholders, by revising the proposal. And, if fully implemented, they believe that the impact will be delayed-- that is, it will not impact providers until 2021.

But by that time, CMS plans to consolidate what has historically been Levels 2, 3, and 4 into a single billing level, and then to pay for Level 5 E/M services separately. So overall, this represents a simplification. And it fulfills one of their stated aims, again, of reducing some of the administrative burden that practitioners face.

Finally, the third area that I want to highlight is a new rule starting in 2019 that refers to the amount of reimbursement you will receive for new Medicare Part B drugs. Currently, those drugs in Part B are reimbursed at wholesale acquisition cost plus 6%. They will, going forward, be reimbursed at wholesale acquisition cost plus 3%. It's critically important to emphasize that this relates only to those new drugs that are introduced into the supply chain this year.

This new provision will also apply to drugs that have not yet reported an average sales price. But the point is it will not apply to drugs that have already been in use. So it only applies to new drugs, meaning that its reach is going to be relatively limited. However, what you can imagine going forward with each new year and new drugs being introduced is that the percentage over wholesale acquisition cost will translate into more and more absolute dollars. And therefore, this may be a growing concern for practices.

I want to switch our attention and talk about the Quality Payment Program, or QPP. In the final rule, there is an update to QPP for 2019. The final 2019 payment adjustment for Merit-based Incentive Payment System, or MIPS, practices and providers will become plus or minus 7%. And it will have adjustments to maintain budget neutrality, as well as to reward exceptional performance.

Other noteworthy changes will include an increase in the MIPS performance threshold from 15 points, which is where we were in 2018, up to 30 points for 2019. CMS also finalized two new optional opioid-related measures that MIPS providers can use to report on under the Promoting Interoperability category. These measures will give providers an opportunity to earn bonus points and therefore potentially boost their overall MIPS score.

These are the two measures specifically. One allows for checking a prescription drug monitoring program, or PDMP, prior to submitting an electronic opioid prescription for any individual patient. And the second is an attempt to verify an existing opioid treatment agreement with the patient receiving the prescription.

So I hope that this summary of the updates to the Physician Fee Schedule for 2019 is helpful to our listeners. Ultimately, our goal is to make sure that oncologists can provide the right treatment to the right patient at the right time. And we aim to help CMS implement policies that will advance that goal. ASCO will continue to work closely with the administration to ensure that CMS understands the needs of the oncology community and the full impact that the rule is likely to have.

I would encourage you, if you need more information on the Medicare Physician Reimbursement Plan for 2019, to visit ASCO in Action's website. That's at ASCO.org/ASCOaction. And ASCOaction is written as one word. We have a link to the final rule there. And we also have a helpful, I think, webinar that explains the final rule schedule and QPP rule in greater detail. So hoping this is helpful. Until next time, I want to thank you all for listening to this ASCO in Action podcast and hope you don't catch my cold.

How Can Financial Barriers to Patient Participation in Clinical Trials Be Addressed? Dec 04, 2018

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Subscribe through iTunes and Google Play. Welcome to this ASCO in Action Podcast. This is ASCO's monthly podcast series where we explore policy and practice issues that impact on oncologists, the entire cancer care delivery team, and the individuals we care for, people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action Podcast series. For today's podcast, I'm really delighted to have Dr. Beverly Moy joining us. Dr. Moy is a medical oncologist at the Massachusetts General Hospital who specializes in breast cancer care. She's also an Associate Professor of Medicine at the Harvard Medical School and a longstanding and dedicated ASCO volunteer. She led the ASCO roundtable on addressing financial barriers to clinical trials, and she was one of the authors ASCO's policy statement providing recommendations to address this important issue. So our conversation today will focus on the existing financial barriers to patient participation in clinical trials and how policymakers, trial sponsors, institutional review boards, and other stakeholders can help remove and overcome those barriers Dr. Moy, welcome and thank you for joining me today. I'm so glad to be here. By way of background for our audience, only a small percentage of patients with cancer ever participate in clinical research. And yet, we know that certain groups, especially people from low socioeconomic status are underrepresented even in those trials. To give a little bit of shape to that, no more than 3% or 4% of adults with solid tumors participate in research studies. And even then, that number over-represents certain higher socioeconomic groups and underrepresents others. Can you tell us why this issue is important to be addressed, and, furthermore, how we might improve the diversity of clinical trial participation? So I think it's incredibly important that to improve clinical trial participation among underserved groups. When I think about how we can provide the highest quality cancer care, we really can deliver three basic things-- the best possible standard cancer therapy, the best possible supportive and palliative care, and the best possible novel or experimental therapy. So we know that clinical trials are crucial to the advancement in cancer care. And in the current genomic era, sometimes these trials may even represent the best possible treatment option for some of our patients with cancer. We also know from the medical literature that patients who participate in clinical trials tend to do better and sometimes even live longer. So when access to the best possible experimental therapy is threatened, optimal cancer care becomes impossible due to financial barriers. And this is an example of social injustice, where poor or underserved patients are being deprived of quality cancer care. So I think that improving the diversity of clinical trial participants is also critically important. When we determine a new treatment's effectiveness, the cancer clinical trial participants really should reflect the general population of patients with cancer. So as you already said, just a small percentage-- no more than 5%-- of all adult cancer patients in the United States participate in clinical trials, most of whom are white and are from a higher socioeconomic class. That means that the results of our clinical trials are less generalizable, and we need to do better. So I think that's a longstanding issue, and I know that many listeners are appreciative the efforts to address it. But it raises a question about the underlying reason for this. And one of them, I think, is financial, that is financial barriers to participation, financial barriers to care, and what is now called financial toxicity. As an aside, we recently conducted a national cancer opinion survey-- we do this every year-- and one of the striking observations this year was that 57% of Americans say that if they received a cancer diagnosis, they would be most concerned about financial impact or paying for treatment. And that was compared to 54% who said they'd be most concerned about dying or suffering from the diagnosis. I think it's a remarkable statement that at least a large proportion of people think first and most profoundly about the financial implications of a cancer diagnosis as think about the health consequences. So if we think about that and then turn back to the clinical trial situation, it is, I think, true that most clinical trial participants will have to face even more additional costs that may prevent them from participating in trials. Can you provide us with an overview of what some of those additional costs are that participants face when they consider a clinical research setting? So Cliff, I think you're absolutely right and. You know, you're talking about the general financial burden that any cancer patient faces is incredibly high. And I actually would reference listeners to listen to your podcast that you did after the ASCO Quality Symposium, where you actually talked about a few studies looking at really the high rates of financial burden that patients just generally diagnosed with cancer faces. When you add the complexity of participating in a clinical trial, not only do you have that general financial burden that a cancer patient has, but you add additional potential financial costs that become prohibitive for our poor and underserved patients. So I think of these additional costs related to clinical trials falling into really two basic categories. One is gaps in insurance coverage, and then the second is medical out-of-pocket costs. So picking the gaps in insurance coverage policies category, we know the Affordable Care Act was passed in 2010, and it does require coverage of routine health care costs for patients participating in clinical trials. But these protections do not extend to patients with Medicare or Medicaid. It's only for patients with private insurers, and there are even restrictions there, which we can go into later. That means our poorer or older patients are more vulnerable to not having these protections. These potential costs could consist of things like investigational care costs, such as this specific therapeutic drug under investigation, or more likely the cost of additional services that would not have been required if the patient was receiving standard therapy. These services could be things like extra blood draws for safety data or imaging studies that fall outside of the routine staging exams. So these extra services have the danger of either being billed to the patient or forcing the patient to pay more towards their insurance premium due to policies that increase cost sharing to patients in the modern era. A second important cost category, which is just as important, if not more important, would consist of non-medical out-of-pocket costs resulting from clinical trial participation. So these costs include travel and lodging expenses, or costs due to lost wages because they have clinical trial visits, or the need to pay child care because they have to come into the hospital or the cancer center more often. So patients participating in clinical trials often have far more frequent visits than standard therapy, perhaps at centers that are farther away from their home. If they participate in early phase first in human trials, they could require longer days of things like pharmacokinetic testing that might even require overnight stays in hotels. So these out-of-pocket costs can be considerable, and prohibitive, and make it impossible for poorer patients to enroll into clinical trials. I know that-- and I'm sure many of our listeners know that-- you're actually a day-to-day clinician. You see breast cancer patients, as well as do research at MGH. And I'm interested in the practical ways that you might have seen these costs directly affect your patients. Do patients ask about trial costs when you're talking to studies with them, talking about studies with them? Have you ever had patients hear about a study and then declined to enroll because they thought it would be too expensive? So I think that's a really interesting question, Cliff. And I think, in the past, when we've presented clinical trials to patients, the idea of costs never really came up maybe because it was something that patients didn't feel comfortable bringing up to their physicians. But now, either costs are becoming more considerable, or it's being raised into awareness that I think that patients are much more comfortable talking about it, maybe not at first, but maybe towards the end of the process. So in my own practice, I've seen people bringing it up more, and more, and more, usually even during the consenting procedure when you're talking about all the extra visits and studies that the patient may need to have to undergo the clinical trial. I've also seen patients who've actually enrolled in trials, and they're on study, and they consider even coming off trial because they didn't anticipate some of the costs that might happen. So, for example, recently, I treated a young woman with high risk breast cancer on an adjuvant CDK4/6 inhibitor trial. And this trial requires more frequent medical visits and blood testing because of potential toxicities. And she found that the extra co-pays and financial costs toward her insurance deductible were completely prohibitive. And despite conversations, she actually prematurely dropped out of the trial after only about nine months of therapy. And this is a single mother of two young kids. That's really not acceptable that a financial reason was the reason why she couldn't continue on the trial. So I think patients are asking more and more for financial guarantees even before signing consent for clinical trial screening, and I'm afraid we really can't provide financial guarantees because that's really not possible for us to do. So one of the reasons that we bring together volunteer groups like you've participated in and then publish results at ASCO is not merely to identify, name, and describe the problem, but in fact to offer solutions or at least potential solutions. So I want to turn and talk a little bit about the recommendations that you've made in the policy statement and through your work. The most recent policy statement on financial barriers to clinical trial participation focused, as I understand, on three key objectives. One was transparency in terms of clinical trial costs and these gaps in coverage that you've been describing. The second is reducing concerns about inducement, that is making sure that whatever we do does itself in an unreasonable way lead to the perception or reality of induction onto the trial. And finally, improving data in the course of participation in trials. That is studying this scientifically like we do other aspects of care. So let's start with the first objective, transparency. What are some of the ways that lack of transparency has affected participation in trials? And how can clinical trial sponsors, or sites, or investigators help us address the issue of transparency? So I think that transparency is a very, very critical issue here. And one of the potential problems is that lack of transparency about who is responsible for the specific costs of clinical trial participation can lead to uncertainty from the patient's part about what he or she might be responsible for paying in the end. So often, insurance payers and research sponsors, whether it be industry or other, they might disagree about who is responsible for which costs. They might argue about what really constitute truly safety assessments, what's really routine costs, so generally the research sponsor covers the costs of additional or more frequent services that the insurance payer may disagree about what services are truly additional or more frequent. You know, what is really standard? So if a health plan denies coverage for the entire trial or individual services within a trial the sponsor considers routine, this could be problematic. So without transparency or protections the patients run the risk of being billed directly for these services. So these costs need to be addressed specifically so that the financial burden doesn't land on the patients in the end. One of the reasons for the costs being a barrier, of course, is that clinical research is often very expensive, and at least some of the recommendations focused on clinical trial design is a way to reduce costs. What changes do you think sponsors might make to clinical trials that could directly bring down the costs for patients? And I would add, especially with regard to those traditionally facing greater barriers, the populations we're talking about today. So clinical trial design is really important because we have to be very cognizant that what this means to the patient and how we're burdening them. So clinical trials really need to be more aware of the financial implications of their study design. So, for example, excessive follow up medical visits, or additional laboratory draws, throwing on more imaging studies, or other procedures that are not absolutely critical to the study really ought to be eliminated because these costs add up. And if they're not critical, they really shouldn't be done. There are costs associated with every test or visit performed. So we really need to reduce the excessiveness of what's required of patients on clinical trials. So in addition to that-- and I mentioned this earlier-- one of the concerns on the other side is about what happens ethically if we pay patients directly to defray these out of pocket costs? This is referred to as an inducement. And around clinical trials, there's really a high degree of, I think, appropriate concern for inducing patients on to studies. What are some of the concerns? Do you think that they're well-founded or overblown? Is there anything that we can do to remove or address those concerns while defraying the course of participation? So I could talk about the ethics about this issue all day long, but I'll try not to. But I think the theoretical concern that I've heard often is that financial compensation or reimbursement of clinical trial expenses could represent a form of inducement or coercion to enter into a clinical trial. And this probably stems from FDA and OHRP regulations that clinical trialists should minimize the possibility of coercion or undue influence on patients. There may also be a potential hurdle from CMS that the Social Security Act specifies criminal and civil penalties for offering financial remuneration to a Medicare or Medicaid beneficiary that influences the selection of their medical provider. So I'm not a lawyer, but I think these concerns are fairly ludicrous because first, coercion, as a principle, is completely irrelevant here. From an ethical perspective, coercion involves a threat that makes a certain choice irresistible, and that is not relevant in the case of cancer clinical trials. Undue inducement is also irrelevant because we have multiple examples in the medical literature that large payments do not disproportionately affect patients' willingness to do medical tasks, for example even donating a kidney. There was actually even a recent article published in JAMA Oncology, written by some bioethicists from UPenn, that argues that the worry that offering inducements to participate in research is inherently wrong. The authors even go so far as to discuss paying patients for cancer clinical trial participation to make participation more attractive to a wider population of patients. And that's really important for both social justice and trial completion issues. So the…

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ASCO CEO Discusses Striking Findings from National Cancer Opinion Survey Nov 13, 2018

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Hello, and welcome to this edition of ASCO in Action. This is ASCO's podcast series where we explore policy and practice issues that are important to oncologists, the entire cancer care delivery team and, most importantly, the patients we care for, people with cancer. My name is Clifford Hudis, and I serve as the CEO of ASCO and the host of the ASCO in Action podcast series.

For today's podcast, I do not have a guest. Instead, I am personally going to share key findings from ASCO's 2018 National Cancer Opinion Survey. We conduct this survey yearly, and we always hope to find interesting information that can help us as we talk to patients, policymakers, and all of the stakeholders in cancer care. This year was no different. Perhaps, the most striking and concerning finding for us this year was this.

Nearly 4 in 10 Americans believe that cancer can be cured solely through alternative therapies. Over the next few moments, I'm going to explore this observation in a bit more detail and then consider some other notable findings from this year's survey. Thanks for listening in. Now, by way of background, ASCO just recently released the results of our second annual National Cancer Opinion Survey.

We conducted the survey in collaboration with the Harris Poll to help us better understand the views held by the US public regarding cancer research and cancer care. One reason that ASCO established this annual survey was our view that by tracking the American public's perceptions of cancer, over time we might be able to better identify opportunities to add useful information and insights that could positively influence public policy.

Therefore, the survey is designed to collect high quality objective data that can be used to understand what the public does and does not know about cancer. We then use this research to help guide ASCO's educational policy and advocacy efforts. This year's survey was conducted online for a one month period. This was between July and August of 2018. Nearly 5,000 US adults over the age of 18 responded to our survey.

This included about 1,000 individuals who currently have cancer or have had cancer in the past. And, as I noted, it always amazes us that we find interesting tidbits in these kinds of surveys, and this year was no exception. I'm going to highlight three areas in particular. These include the role of alternative therapies, access to pain management, and the continued financial burden of care. ASCO's core values, as many of you will know, include evidence, care, and impact. Given that, we start with evidence, and one of the most surprising findings for us is that nearly 4 in 10 Americans believe that cancer can be cured solely through alternative therapies. And I note that given that research has shown that the sole use of alternative therapies for cancer is actually associated with a much higher mortality rate when compared against patients treated with standard evidence based approaches.

Now for clarity, I want to point out that when we say alternative medicines, what we mean specifically are interventions like acupuncture, diet, so-called enzyme therapy, massage, medical marijuana, meditation, vitamins, herbs, and other supplements. All of that and more, perhaps, in this context, we're referring to them as the sole therapy for cancer, not as complementary treatments where they have arguably different roles and different impact. It's this potential reliance on them as the sole therapy for cancer that is so concerning. Now even with direct experience with cancer, respondents don't report a very different perspective. For example, even among those who have had cancer, either themselves or have been close caregivers as a family member, for example, a sizable proportion expressed the belief that cancer can be cured solely through alternative medicine.

Younger people, those age 18 to 37, and, to a slightly lesser extent, those aged 38 to 53, are most likely to believe that cancer can be cured through alternative therapies. Clearly, given this, we have a great opportunity in front of us to help our patients, families, legislators, and everyone understand the real limits and the reality of scientifically valid evidence based treatments. And we have an obligation, given the association with improved overall outcomes, to highlight this.

We cannot ignore this widespread belief. Now I want to turn to another issue that's been getting a lot of attention these days, the opioid crisis, and explore what Americans are thinking about the use of opioids for pain management in cancer care. Our survey found that nearly 75% of Americans do not agree that there should be limits on access to opioids for people with cancer. Specifically, most Americans believe that cancer patients should not have their access to opioids limited at all.

Yet the survey shows that accessing opioids right now for cancer pain is difficult for many patients. From the small survey sample that we had of patients with direct experience, 40% of those with cancer who had used opioids within the past year to manage pain or other symptoms reported trouble accessing them. If opioids are an important part of maintaining quality of life and palliating cancer symptoms, then this obviously is an active and real problem. Highlighting the importance of providing optimal palliative care, we also learned that most Americans support alternative methods of managing pain. And, again, I want to emphasize, in this case we're talking about alternative therapies as complements not as the sole approach. 83% of respondents supported the use of medical marijuana among people with cancer, for example. Here again, however, there is an issue with access.

In a small sample of patients who have reported using medical marijuana within the past 12 months, nearly half, 48%, reported difficulty obtaining it. For those patients with cancer or who have had cancer, who are interested in using medical marijuana, almost 60% of them wish that there was more information available about its benefits for symptom relief. This is clearly a research opportunity for our community. We believe these views are likely to be heard both federally and at the level of state houses. And we think, therefore, it's important for you, our listeners, to be aware of this widespread point of view. Finally, I want to turn to one of the biggest and longest standing issues, as well as fastest growing issues in health care in general in cancer care specifically, and that is finance. We find that the financial burden is a specific worry for Americans confronting cancer. On a percentage basis, our survey respondents said they are just as worried about the financial impact of cancer as they are of dying of cancer. Now this doesn't mean that the depth or level of an individuals anxiety is the same, but it does mean that it's on the minds as about as many people. Probing this a little more, if faced with a cancer diagnosis, 57% of Americans say they would be most concerned about the financial impact on their families or about paying for treatment.

And that compares to 54% who said they'd be most concerned about dying or about suffering with pain. So I think this just highlights how central worries about financial toxicity and cost of care have become. Among people who have had cancer, or who have survived it, more than 40% say that they've had barriers. They've experienced barriers accessing care because of health insurance, with deductibles and co-pays specifically being their biggest hurdles.

And what's interesting, as well, is that patients bear a significant burden here, but so too do family caregivers. In fact, among caregivers responsible for paying for cancer care, 3/4 say that they are concerned about affording it. More than half of caregivers say that they or another relative have had to take some kind of an extreme step to help pay for their loved one's care. Examples include working extra hours, postponing their retirement, or taking on an additional job, even, in some cases, selling family heirlooms. Clearly these findings are in line with other research that has shown that financial toxicity is a growing and very real concern for people with cancer and for their families. And, again, we think it's important for our listeners to be aware of these issues since our sense is that some of these don't necessarily come up, at least not overtly, during routine clinical encounters. The bottom line is, more of our patients are suffering degrees of financial distress than we may recognize during our busy clinical days, and we need to be both aware of this and help take steps to address it. So with that, I want to thank you for listening in today. As the world's leading organization for oncology care professionals, ASCO believes that it is critical to understand what the public, including individuals with cancer, think of, expect, and need from the nation's cancer care delivery system.

As I mentioned earlier, this year's findings will help inform ASCO's future educational, policy, and advocacy efforts. And we need all of our members to help, as well. Keeping informed is one critical first step, of course. Looking ahead, please know that ASCO's National Cancer Opinion Survey is scheduled to be conducted again next year. And this will give us one more opportunity to drill down even deeper into findings from this year and explore other emerging issues while tracking potential changes in the focus and concerns of the general public we serve.

If you would like more information about the survey, please visit ASCO's website at asco.org and search for National Cancer Opinion Survey. Until next time, thanks again for listening to this ASCO in Action podcast.

ASCO CEO Discusses New Studies on Patient Financial Toxicity and Opioid Use Risks Oct 23, 2018

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Welcome to this ASCO In Action podcast. This is ASCO's podcast series where we explore policy and practice issues that may impact oncologists, the entire cancer care delivery team, and the quality of care they provide. But most importantly, of course, the patients we care for. My name is Clifford Hudis. And I'm the CEO of ASCO and the host of this ASCO In Action podcast series. For today's podcast, I'm going to share with you some highlights from the research that was featured at this year's recent Quality Care Symposium.

So ASCO is the host and sponsor of the Quality Care Symposium, an annual meeting that brings together oncology leaders, and all of the members of the cancer care team, to share strategies and methods for improving the measurement and the implementation of quality and safety activities in oncology. The recently held 2018 symposium presented a wide range of scientific abstracts, focused on initiatives that aim to improve the quality of care for patients with cancer, and, also, new research in this field.

Today, I want to highlight five of these abstracts from the Quality Care Symposium. These are abstracts that deal with issues I know are of particular concern to ASCO members, financial toxicity for the one and opioid use for the other.

Now, turning first to financial toxicity. As everybody knows, oncologists see the impact of high treatment costs on our patients, many of whom are not taking all of their prescribed medication because of cost. They are drawing down their savings, when they have savings. And they're often not paying their other household bills or taking other drastic measures because the cancer treatment that they have to receive has become so expensive. This distress is now broadly defined as financial toxicity.

Three different studies were presented at the Quality Care Symposium that put a spotlight on this issue. In the first study, by Wheeler, et al, we saw results of a national survey of more than 1,000 patients with metastatic breast cancer drawn from 41 states. What we saw here, was that in these individuals, especially those who are uninsured, there really was significant financial distress. A full third of these patients were uninsured and more often they reported refusing or delaying treatment because of the cost of care. They also reported that they were contacted by collection agencies because of unpaid bills, again, for their cancer care.

The study also found that insured respondents were not immune to financial toxicity. Those with health insurance reported having higher cost-related emotional distress, being stressed, themselves, because they weren't sure about the cost of their treatment, as well as spending a lot of time-- as well as having, I'm sorry, a lot of financial stress placed on their families because of cancer.

The second research study by Arastu, et al, related to the cost of care. And this showed that nearly one in five older patients-- and these patients were defined as age 70 and above-- who had advanced cancer, were experiencing financial difficulties, again, due to the costs of their treatment. They noted that these difficulties negatively affected their care, their quality of life, and mental health. In this study, patients experiencing financial toxicity had a prior higher

prevalence of severe anxiety and depression, poor measured quality of life, than patients who did not report financial hardship.

And finally, there was a third study by Greenup, et al, in which 600 women with a history of breast cancer were surveyed. The majority of them said that they would prefer to discuss the cost of care before beginning treatment, but few of them recalled having such conversations about treatment costs with their cancer care teams. In this survey, fully 79% of these women said that they preferred to have a full understanding of the costs of care prior to starting, but 78% of them said they never actually discussed costs with their cancer care team.

The findings of these three studies, I think, are important. They're a reminder that financial toxicity is real, that it represents a particularly harsh reality for many of our patients, and that we, as oncologists, are expected to initiate and guide conversations about the cost of care with our patients, although it doesn't happen very much yet.

A very good starting point for this seems to be to direct our patients to resources and help our patients prepare for these discussions. So ASCO offers an array of these materials on its patient information website. You can look that up at cancer.net.

Now, the other big area of focus, and, again, one that's been covered in the late news extensively in the last years, is, of course, the opioid crisis. At the Quality Care Symposium there were several aspects of the opioid issue that were addressed. I'll point out in background that although there's evidence clearly available that shows that patients with cancer may be at lower risk for abusing opioids than the general population, we also are aware of the fact that opioids are a controlled substance and they can be addictive. And patients with cancer are not immune to addiction either.

So understanding the size of the problem within the arena of cancer care, and then learning what the best practices are to help control the use of opioids to the most appropriate usages, especially after surgery, are very important matters for us. There were two studies presented during the symposium that provide some important insights on both of these matters.

The first by Chino, et al, was a retrospective study conducted over a 10-year period that comprehensively explored the risks associated with opioid use among cancer patients and compared that to the general population. They analyzed death certificates. And they found that deaths attributed to opioids, in cancer patients, were about 10 times less than in the general population. Although the incidence of opioid deaths had increased significantly in the general population, that increase, again, attributed to the opioids among patients with cancer was much, much smaller. Deaths from opioid use were highest in patients with lung, GI, head and neck, hematologic and GU cancers.

The second study by Stevenson, et al, looked at how oncology care team members could use a two-prong approach to achieve a reduction in opioid use-- in this case, it was 46%-- among cancer patients who underwent a variety of urologic surgical procedures. And they accomplished this without increasing pain or anxiety.

The first part of their strategy involved developing new processes for post-operative pain control that focused on non-opioid medications and therapies. These were interventions provided first line as pain management. Patients could still be prescribed opioids, but when they got these prescriptions, they were at lower doses and dose escalation was only performed if necessary.

The second prong of their two-pronged attack involved post-operative conversations with patients. Talking to them. Rather than having nurses routinely ask patients whether they wanted any pain medication or not-- and this was often a direct reference to opioid medications, specifically-- the nurses, instead, discussed current non-opioid medications that the patients were receiving for pain, along with their frequency and dosage, asked whether these medications were sufficient and discussed their potential side effects, along with the side effects of opioids. The opioids were never withheld, but they were no longer the reflexive standard, thanks in part to this two-pronged approach.

Now, from an ASCO point of view, we are really supportive of efforts to address opioid misuse and abuse. And we counsel our members to discuss the benefits and the risks of opioids with their patients. And then, of course, to prescribe pain treatment for patients responsibly and especially paying attention to those patients who have risk of addiction. At the same time, we keep our primary concern on adequate pain control for cancer patients. This has been a long-term issue for us. And it's an important one. So taking all this into account, we are working continuously with policymakers to ensure that both federal and state initiatives that are implemented do not impede cancer patients access to essential pain medication.

Now in closing, I want to say, again, thanks to all of you for spending some time with me today to learn about ASCO's Quality Care Symposium. The meeting provides a great forum for the entire cancer care delivery team to learn about evidence-based strategies and methods for evaluating and reporting on patient outcomes, provider efficiency, and quality and safety in cancer care. And the abstracts that we've highlighted today point that out nicely, I think.

In the end, all of this is about evaluating which approaches work best and how to continuously improve them so that we can do our best to ensure that every individual patient with cancer continues to receive the highest quality care that's possible. If you would like more information on the research that was presented at this year's Quality Care Symposium, please come to our website, abstracts.asco.org. And until next time, thanks again for listening to this ASCO In Action podcast.

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