Full Show: Should Costs be Controlled on the Way to a Cure for Multiple Myeloma? The ICER Report with President Dr. Steven Pearson

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Dr. Steven Pearson, MD, MSc
Institute for Clinical and Economic Review
Interview Date: May 24, 2016

Summary

The ICER Report’s intent is to identify “value-based” treatments for relapsed/refractory multiple myeloma. Identifying the best bang for the buck is good, but with the complexity of multiple myeloma, could this get in the way of a cure? Dr. Pearson explains the objective of the public ICER report and how it will be used. The report has been hotly contested by patient advocacy groups who are concerned that the report will give insurers reasons to limit treatment options. The Myeloma Crowd believes that finding a cure (or the right treatment for the right patient at the right time) is the ultimate goal and that looking at cost cutting as a standalone objective gets in the way of this larger and more important objective. ICER chose to include myeloma in its reporting as the disease had several new drug approvals and uses drugs in combinations, which layer on costs. Myeloma is an incredibly complex disease and patients present with 5 different types of clones on average at diagnosis. Additionally, those clones morph over time. We fundamentally disagree that reallocating money from one disease will provide success in another. Typically, this approach kills innovation. While we understand that combining drugs means increased cost,  we don’t believe that cost cutting measures can be looked at as an isolated issue until we have deeper data about which drug combos work best for which patients. In our opinion, “How can we cut costs?” isn’t the right question. A better question is “How do we collaborate to cure myeloma faster to save life and money at the same time?”

Dr. Steven Pearson on Myeloma Crowd Radio

Full Transcript

Jenny: Welcome to today’s episode of Myeloma Crowd Radio, a show that connects patients with myeloma researchers and the latest in myeloma advances. I’m your host, Jenny Ahlstrom.

Today’s program is our 80th, which is a bit of a departure from what we are usually used to. We’ll be speaking with Dr. Steven Pearson, the Founder and President of the Institute for Clinical and Economic Review or ICER, which is currently formulating recommendations of effectiveness and value of treatments for relapsed and refractory myeloma.

This Thursday, in a couple of days, ICER’s Midwest Comparative Effectiveness Public Advisory Council will hold the meeting in St. Louis to review and vote on recommendations regarding its report on the effectiveness and value of treatment options for relapsed and refractory of myeloma. The final report is scheduled to be released on June 24th.

So Dr. Pearson, welcome.

Dr. Pearson: Thank you very much, Jenny, and I hope you’ll feel comfortable calling me Steve.

Jenny: Well, I understand the effort that goes into becoming a doctor and I know you are one, so I may call you that.

Dr. Pearson: It’s totally fine, whatever makes you most comfortable. Thank you for having me on your call.

Jenny: All right. Before we get started, let me just give a little bio about you.

Dr. Pearson received his degree at the University of California, San Francisco School of Medicine. He completed his residency in Internal Medicine at Brigham and Women’s Hospital in Boston, and obtained a Master of Science Degree in Health Policy and Management at the Harvard School of Public Health. His experience included serving in many advisory and leadership roles in academia and government, including a fellowship with the National Institute for Health and Clinical Excellence, also known as NICE, in the United Kingdom. He has served as Special Advisor to the Centers for Medicare and Medicaid Services. He’s a Senior Fellow at America’s Health Insurance Plans, Vice Chair of the Medicare Evidence Development and Coverage Advisory Committee from 2007 to 2009, and was a Member of the Board of Directors of Health Technology Assessment International from 2010 to 2013.

Now I know this is not a comprehensive CV by any stretch, but I think this gives our audience not just a sense of professional background.

To begin today, I think it would be really helpful to our audience to get a sense of two things. First, can you explain why ICER was created and what its mission is? And secondly, explain some of the highlights of this report on myeloma and what it might mean to newly approved drugs.

Dr. Pearson: Sure. Thanks again. So ICER is the acronym. It stands for Institute for Clinical and Economic Review. I started it about ten years ago. For most of its life, actually, we were an academic research group at Harvard Medical School. And the goal was to try to figure out if we could do a better job of creating reviews of evidence that would do a couple of things: One would be to engage with stakeholders, and I know that word is far overused but that includes the manufacturers, it includes patient groups, it includes the clinical societies. Because up to that point, most of the health technology assessments that were being funded at least by the government had a very, very strict process where they kept these groups at arm’s length. So we wanted to try to do a better job of doing evidence reviews that really engaged with everybody.

Second, and perhaps the most distinctive, everybody looks at the evidence on clinical effectiveness. Part of our mission we felt was to explore ways to bring in information about cost-effectiveness, about potential budget impact, you know broadly speaking, trying to bring in information that could help people make a broader judgment about value, and have that discussed in public.

From the beginning, we’ve had our reports basically debated by public bodies that we convene. We recruit independent clinical experts in methodology. We actually have patient and public representatives on all of our groups. And we have a public hearing where this evidence can be discussed. We can talk about how we wrestle with interpreting evidence on, especially new things, but sometimes we look at old things. And then we do have a public discussion with stakeholders around the table, including patients and insurers and manufacturers around­: “how do we think of working together to move the evidence into the healthcare system? What would it mean for what patient educational materials might be needed that aren’t there already? Are there ways for patients to get involved with research? What are the future research needs?”

We do talk about how might insurers adapt this evidence into their coverage policies—I mean a lot of people don’t really understand how coverage policies are made today. But for a lot of health insurers, they have a small internal team of people trying to keep track of all of the evidence in making coverage decisions. What we try to do is just to bring a more rigorous and transparent process to at least providing information into that process. Again, we don’t shy away from what we feel are some of the key issues, which is, what does the value mean here? What if it looks like it’s a great long-term value but it’s very expensive in the short-term, how will the healthcare system deal with that? What are some of the options? What are the implications?

We have a very technical mission, which is to provide what we hope are very scientifically rigorous evaluations. We have a broader ethical mission to try to bring this information in a way that engages people around a more honest discussion about evidence and value across our entire healthcare system.

So we don’t look at just drugs. Actually, we’ve been pivoting a bit because drug issues around value have been really highlighted over the past few years. But we do reports on things like integrating behavioral health into primary care, bringing community health workers into the healthcare system, we do some devices as well. We did a report recently on palliative care. Again, looking at issues of the effectiveness for patients and the value.

We were academic until about two years ago when we were in Harvard Medical School but we were physically part of a hospital, the Mass General Hospital. And people started to wonder if we were biased because we were in a hospital. So we decided to become an independent non-profit foundation in that point. About 70% of all of our funding comes from non-profit foundations, the lion’s share from a foundation called the Laura and John Arnold Foundation in Texas.

About 30%, which none of the 30% goes to the development of our reports but we have a separate policy summit processed for manufacturers and for health plans so that they can meet once a year, take off their ties, and hopefully, do some serious work together. And we have some evidence webinars. Anyway, that’s about 30% of our funding that comes from a mix from manufacturers and from health plans. But our reports are funded by these non-profit foundations.

That’s ICER, and I’m glad to answer more questions about it. I will say that in the current context, the national landscape issues around drug pricing had become very political. No big surprise to any of you. So ICER is taking a fair amount of scrutiny, which we expect. We try to be as transparent as possible. Everything on our website, hopefully, makes it easy to understand who we are and what we’re trying to do. But there’s been a fair amount of misinformation, actually, that’s coming out, especially recently. And partly, that’s because Medicare cited us as one potential source of information for a future version of payment for injectable drugs. Many of you know that Part B demonstration as it’s called, has been very controversial, so we’ve gotten wrapped into that echo chamber.

So multiple myeloma came up because there has — you know there’ve been some fantastic innovations and several of them were bunched together, some new drugs; elotuzumab and Ninlaro. Sorry, I jump back and forth sometimes between the generic and the trade names. And carfilzomib was out not too long ago. So, there was a lot of interest in a variety of quadrants about what can we know about these new drugs from the evidence so far. Where are the evidence gaps? And again, for us, what do we think about the value? What can we try to estimate about the long-term benefits and costs for these drugs? What does that mean for the prices? Do the prices align well with what we think are the benefits to patients? Are they way off the charts? Are some much more out of alignment, if you will, than others?

So those are the kinds of questions that we try to address in our report. Do you want me to go through just briefly what we found or would that take too much time?

Jenny: No, that would be fine.

Dr. Pearson: Okay. Our executive summary, I know it’s not written for patients. Many of you are very sophisticated and could probably grasp every bit of it. But these are not intended to be the thing that a patient would read before going in to talk to his or her doctor. I hope people don’t get the wrong feeling that that’s what we’re trying to do. We do make patient versions sometimes when we are told that it might be helpful. But I hope you don’t get turned off by the fact that it’s fairly technical.

What we found in large, was that the evidence for the new drugs is adequate to suggest that they do provide a net health benefit. Net health benefit for us means that the benefits in terms of lengthening life for most patients will outweigh the side effects. And you guys are all well aware that none of these drugs come without side effects.

So for carfilzomib, elotuzumab, and ixazomib, that’s Ninlaro, we judged again for both second and third line that the evidence suggested that it was at least a little bit better than existing treatments, and could be a lot better. We had more doubts about some of the evidence around panobinostat, that’s Farydak, and pomali —

Jenny: Pomalidomide?

Dr. Pearson: Pomalidomide. The FDA label doesn’t even include second-line therapy, anyway. But even for third line, we just felt that there were more questions about the patient selection, the comparator treatment in the trials. So our judgment was that the evidence was promising but still inconclusive for the net health benefit for those two. And for daratumumab, that’s the drug that came to the FDA with a relatively small single-arm trial without any comparison, which always makes it very difficult to judge the comparative clinical effectiveness. It doesn’t mean that the FDA was wrong, but we judge that there was insufficient evidence for us to be able to judge its comparative clinical effectiveness.

And as far as the value, when we’re looking at the long-term cost-effectiveness — and we work with an academic group at the University of Washington that had done a lot of work in multiple myeloma before, building a model to simulate long-term clinical outcomes and costs. We found that the drug seemed to be priced higher, at least their list price. For some of the existing drugs, their WAC price, but their list price is certainly higher than a price that would meet what are considered to be standard thresholds in a cost-effectiveness parlance. I can go into more detail about that if you want.

But the bottom line is that we suggested that they come into better alignment with the amount of benefit that they’re bringing to patients, and they are bringing benefit to patients. You would need, for instance for second-line therapy, a 32% discount off the list price of carfilzomib but up to 75% to 80% discount off of elotuzumab and ixazomib. We also raised the policy question that multiple myeloma is not unique but it does have this issue where some of the newer treatments, which are expensive in their own right, get loaded on top of other treatment regimens that are already expensive. Sometimes the drugs like carfilzomib, you take them and at least the label says that you will stop when you get into remission. Others are basically, you just keep treating.

That will always have an impact on the cost-effectiveness. We heard from patients a lot that they also do appreciate time off of treatment. So it’s certainly maybe viewed as a patient benefit as well. But that’s one of the reasons why the percent discount is less for carfilzomib than for the others. And we’ve talked to some drug companies about this. I’d be interested to hear if you’ve had this experience. But you know, the newest trials for myeloma, even beyond the frontier that we’ve looked at, are adding on, again, another layer of more expensive drugs, the PD-1 or PD-L1 drugs.

And the question is, are we really going to just keep adding on costs on top of an existing base, or is there some way for the drug companies to work together to create a package that can be discounted more effectively? Like the way that airlines have worked together so that when you travel from point A to B and B to C, if you get one ticket even if it’s two companies, it’s not as expensive as adding A to B and B to C.

So we provide some ideas that if we were able to get discounts across the board on, for instance, the lenalidomide, that’s a core part of a lot of these regimens. You wouldn’t need as big a discount on the new drug. You could take a smaller discount across all of them.

Jenny: So let me back up just a minute and ask you an overall question because you said you saw a lot of activity and a lot of new drugs being introduced into myeloma. But when you look at lowering costs, which sounds like is your objective—let’s get the biggest bang for the buck, let’s say. Why not choose a different cancer or disease with a larger population, and then therefore, more budget impact?

Dr. Pearson: Well, I mean budget impact isn’t the only thing we look at. We actually keep our ears and eyes open to a variety of sources. What we really want to know is, are there things coming into the healthcare system that are keeping people up at night because they think there is some real question about the evidence, or the value that the health system may not deal with well, whatever that means? But in terms of our portfolio, we will be looking later this year at multiple sclerosis, which is certainly a bigger budget impact than multiple myeloma. Lung cancer, we’re going to be looking at severe psoriasis, which I’m not sure if that is or isn’t.

So our portfolio is certainly not focused in—but we’re also looking at actually an orphan disease, primary biliary cirrhosis, because there’s a new treatment coming in that’s the only treatment for that. And sometimes when you get an only treatment for a disease, it raises again questions about, will the price in some way reflect the added value to patients? Are there questions about whether all patients with the condition should use it or just some? Those kinds of questions.

There’s nothing about the fact that—actually, I don’t even know if we knew what the budget impact of—or had any estimate of what the true budget impact of multiple myeloma was when it made our list of topics that we thought we would take on.

Jenny: How many different disease states does ICER cover in total?

Dr. Pearson: Well, again, it grows. Our new funding from the Laura and John Arnold Foundation, our goal is to ramp up to try to do reports on virtually all “significant new drugs” each year. We’re not there yet, so we have to do more picking and choosing to a certain extent. But in this calendar year, again actually, I can’t add them up. But in terms of the topics, again, we looked at diabetes drugs, asthma drugs, congestive heart failure, cholesterol, again the hepatitis/primary biliary. Obviously, myeloma, lung cancer, multiple sclerosis, psoriasis, and rheumatoid arthritis. That’s this calendar.

Next year’s calendar is still a work in progress. It just depends a little bit on where issues are arising around new drugs and other topics that we think, like palliative care, that deserve or would benefit from our work on it as well.

Jenny: Knowing what I know now about multiple myeloma, when you talk about your executive summary and looking at all these different drugs, you’re looking at them in context of drug pricing, how a new drug is priced, introduced, and how effective it is. From what I’ve heard in talking to all the doctors is that — and I think we should probably talk about this upfront, I was going to cover it later, but I think we’re talking about upfront, is the complexity of myeloma.

I just had a show with Dr. David Siegel at the John Theurer Cancer Center, and he was saying that somebody can become refractory to lenalidomide, or one of the standard core drugs that we’ve had for some time, and something like panobinostat or Vorinostat can bring it back to life and help it work again.

So when you look at these drugs alone, like let’s say panobinostat or an HDAC inhibitor or something, you say, “Well, it might not be that effective of a drug by itself.” Or the same thing with elotuzumab. You could look at that alone and say, “Well, that’s a pretty ineffective drug alone.” But you add something else to it. Because on average, according to some of the doctors, every patient presents with five different clones of myeloma at presentation, at time of diagnosis.

So when you look at these drugs and how effective they are, I think there’s a lot of concern about are you going to knock something out that could bring something back to life again? Or, there’s proven evidence now that three drugs are better than two, by far, and that four might be even better in terms of effectivity, if you’re adding them together. So that’s why when you’re separating them apart, I think there’s cause for alarm on the patient side and different stakeholders that are part of this to say, “If we can’t add one on, and then we get longer overall survival.” The example of Velcade is a good one. It had like three-months better progression-free survival or overall survival when it was first introduced. And now, in combination, much more significant.

Dr. Pearson: Absolutely. So great point. Actually, there are a lot of important points, I think, as part of your comment. So first, yes. I mean, I cared for multiple myeloma patients, but it was literally 30 years ago when I was a resident. And I remember how brutal — there was very little and it was nasty and it was short and it was not good. There’s been tremendously important innovation in a variety of ways, the multiple drugs that (are now available). Also, we spoke to a lot of patient groups, we spoke to a lot of clinical experts to make sure that we tried to get, you know, at least, and we’re never going to be the true clinical experts.

That’s why we always continue to work with them and then we’ll have some at our meetings. We try to make sure that we weren’t clueless and naïve about the variety and the heterogeneity and how these drugs are actually being used. So you’re right, and we’re quite aware, and insurers are aware of this too. And I know that part of the concern here is: will this report or evidence be used to say no to something?

At least as far as all the conversations I’ve had with insurers, not one of them thinks that this is a condition where you force all patients to try one drug and then they have to take the next drug and you can never add them together. There’s a variety of patients, and a part of this is the side effects. Some patients just can’t tolerate a certain level of either renal stress or diarrhea side effects, whereas others can. So there’s no way that the payers, I think, want to try to create a cookbook one size fits all approach here.

That doesn’t mean though that we ought to—I think we’d take a clear look at what the studies, how the studies are being done and what we can learn from them. We’re even, in a sense, giving I think, the drugs the benefit of the doubt, because we are using the trials that existed where they were usually being tested against either Vel/Dex or Revlimid and dex, stuff that people just really wouldn’t use by themselves as a treatment option. But we want to try to capture the progression-free survival increase, factor that into the likelihood that that will lead to overall survival.

Some people still argue a lot about whether progression-free survival does translate to an overall survival. But at the end of the day, all of these drugs, for many patients, as long as they’re healthy enough in some way, most patients will take just about all of these drugs, and we know that. I don’t think that when you look at the evidence—basically, the conclusion of our report is that—we’re having a public meeting so I don’t want to get ahead of the discussion there too much—but people reading our report would probably feel like there’s no evidence to suggest that using one of these drugs over another one is “the right thing for most or all patients.” It has to be individualized.

Jenny: My question, I guess, is how will this report be used? What’s the core value of the report and then how will it be used?

Dr. Pearson: Well, again, it’s fully in the public domain. My hope is it will be used in several ways. One is to suggest where the important evidence gaps are, in particular, around ways to gather that evidence so that patients and doctors, as well as insurers can do a better job of comparing different combinations. Now, again, that’s tricky because as you said, each patient ends up on a different regimen over time depending on what happens. But still, oncologists need, if you talk to clinical experts, they say, “Wow, there’s still a lot that we need to know that we don’t know.” And they will tell you.

We end up with a style. There’s a style at the Mayo Clinic, there’s a style at Sloan Kettering, there’s a style at MD Anderson, and they’re not the same. So the question is, can we figure out a way to compare styles so that at least patients can have more knowledge, and clinicians too, obviously, about whether certain styles… and by style, for instance, one clear differentiation is, some doctors — and I’ve learned this. I haven’t seen it, but again, from talking to patients and doctors, there’s a general approach in which, especially for the “more aggressive forms,” that they will frontload a lot of these drugs into highly multiple regimens.

Other doctors tend to take a slower approach in which they hold some of the medicines for later and see if they can get a period of remission and then add things more slowly. We don’t know whether one is better than the other. There are passionate feelings about that, but no one says that there’s good evidence. So anyway, I hope part of the use of our report is to highlight where the future research can be targeted to try to answer the key questions for patients and doctors, as well as insurers.

In our reports generally, no matter what topic we’re doing, we want people to engage around the question of value and affordability. It is important to recognize that there’s a lot of great innovation that has been created. Partly, that’s because the drug companies view that they will make a handsome reward if they invest, and there’s a lot of risk in trying to develop new drugs for multiple myeloma.

But we also have, in a sense, if you step back, we have the same situation for patients with hepatitis C, where there are now hundreds of billions of dollars that might be spent to help them; Multiple sclerosis, Alzheimer’s disease. To a certain extent, we need to figure out how to share the benefits of medical innovation across the entire spectrum. So one way that I think we might, as a society at least start to talk about, is the way that we are doing pricing and the way that our system is set up, and whether that is allowing us to share the benefits of medical innovation as well as possible. Because as you know, there are real budget crunches that I think are leading to tighter restrictions on new drugs than are necessary clinically, certainly.

And there are real problems with state budgets around education and other things because healthcare is drawing in a lot of money. Now, as a doctor, I have no problem with our society spending a lot of money to help patients with their illness. But I think we have to figure out a way to have at least the conversation around, again, how we share the benefits of medical innovation more broadly, and whether the prices that we have are sometimes really not in alignment with the amount of benefit that patients enjoy from some of these, and whether that needs to be a part of the conversation.

Jenny: I think there are two key points there. When I was diagnosed, what I wanted to see was a system, and you talked about the different styles, was a system that said, “Okay. I want to see all patients that look like me that have my same genetic subtypes or my same cytogenetics. I want to see the treatment options. And then I want to see who had the best outcome because that’s the protocol that I’m going to choose.” What you’re trying to do, it sounds like you’re trying to do it in a similar way to say, “You know, we don’t need to be arguing about these different styles of therapy. Let’s let the data decide.” I think the question is, how we go about doing that? And what’s the most comprehensive? What’s going to give us the most well-educated decision?

I think patients have to be part of that process in a very key way because they will be contributing patient reported outcomes that you can then say, “Okay. Well, patients can say and they can make choices. So I’m going to choose this because it looks like, let’s say I have deletion 17 or I have 11;14 (translocation) or I have 4;14 (translocation) or whatever in my genetics, and then it’s evolved over time and this looks like the right thing for me. I mean they’re going to self-select. So a lot of that data, it doesn’t exist today. I wish it did. And I think that it’s a good objective. It’s just how we go about.

The second thing you talked about is trying to identify the value of doing that. Your care value that you talked about, it looks like it’s made up of four parts, how effective something is clinically, the cost per outcome, other benefits or disadvantages, and then contextual considerations. I think that cost per outcome part is—people are freaking out about it because it seems like it might have been the most controversial because it relies on this World Health Organization standard of cost per quality-adjusted life-years, or called — I don’t know how you say it, QALYs? Somebody might say it’s an arbitrary financial number applied to the value of each year of a person’s life.

Am I right in understanding that the ICER formula is measured—there’s an annual measurement to a year of life? How does that work?

Dr. Pearson: So the whole approach to this was actually developed by American doctors working with health economists about 30 years ago, I guess. It’s used more explicitly by some governmental agencies in Europe and elsewhere, but we’re pretty much the only developed country that doesn’t use it these days. Germany doesn’t use it explicitly but in the background. Because really, if your job is to try to make some general judgment about what’s a reasonable additional cost for a benefit, you need some measure that doesn’t only look at how long it might extend a life, but you have to look at something that also captures side effects or the quality of life.

So for instance, if we’re trying to compare treatments for multiple myeloma with treatments for congestive heart failure, some might increase life longer but might have a very poor quality of life because of really severe side effects. Another one might be a little bit shorter extension of life on average but much better side effects. So that’s what the quality is, it’s basically just the way to combine those two things that we want. We want longer life and we want better quality of life.

When you do that, if you’re going to make a judgment about, again, if the goal is to — in some ways, I guess you can just pose it as a ridiculous question. If each of these treatments for multiple myeloma costs $10 million, would we think that that’s—you know, they extend life but they each cost $10 million. So if you use three drugs, that’s $30 million for the first year. At some point, people would say, “Well, no, that’s not feasible. That’s ridiculous.”

So if you’re willing to say that we can’t spend a completely unlimited amount of money for any benefit, then the tough question becomes, well, how much? What’s a reasonable “threshold?” And that’s where, again, doctors and health economists around the world had worked on this for 20, 30 years. Most of the work ends up linking it to the size of the national economy. So obviously, we can afford more for health in this country than they can in a poor African or Asian country. We can afford more than the English can because our economy is bigger, in general.

So it’s a way to try to do it. There’s a general consensus out there that from the United States, based on our economy and the way that we spend our funds on other things, that $100,000 to $150,000 per QALY is the right general threshold. Now, that doesn’t mean that we are saying that your life is not worth more than $100,000 per year as an individual patient. What it means is that over an entire population, when you’re using additional funds to create better clinical outcomes, it averages out that way. And we all know that some patients will get a lot of benefit from something and some will get much less, et cetera.

So that’s where that idea of a cost-effectiveness threshold comes from. And we don’t believe that there’s one single number that we use. That’s why we use a range, and we tend to tilt it towards the higher end of what health economists and others have said because we recognize in the United States, we’re not a governmental agency. It’s quite clear that I think most Americans don’t, at this point, feel like we want the government to do this. Maybe we should but certainly right now, that’s not been the consensus. We are providing it and we’re using the high-end of standard measures of cost-effectiveness to help suggest if you want to align pricing at least with traditional measures of outcomes and reasonable extra cost for those benefits, this is what it would look like.

Jenny: Okay. I think when you look at it objectively at the disease state per se, I think it’s easier to come to those conclusions. And when you actually have the disease, it’s very frustrating to try to identify a cost per value and things like that on it, because you’re looking at multiple options over time and you just want to keep that flexibility remain as flexible as possible.

Dr. Pearson: I can hear that in the way that you think about it. I mean, you would admit that for patients with different kinds of cancer, if we want to be able to share the benefits of medical innovation — again, to use my extreme ludicrous example, would we as a society want to spend $30 million a year for an extra few months of life? Not that the extra few months of life aren’t important, but if that $30 million means that we can’t then help patients with hepatitis C or breast cancer, the question is whether we can all step back and say, “We want the individual, patient, and doctor to fight as hard as possible to do the best they can. But are there some limits around the pricing, at least, for things that have benefits that we need to share broadly so that some segments of our patient population don’t get taken advantage of?”

Because there’s no one speaking up that loudly until very recently for patients with opioid addiction. They need care. When we looked at palliative care, actually, the extension of life with palliative care and its effects on quality are better than most new cancer drugs. And yet the people can’t find money to hire new palliative care docs. So there are a lot of unmet needs in our healthcare system, and I just think it’s reasonable to try to figure out how we come up with a fair system across all of them to at least start the conversation about where we want to try to set limits on some prices and how do we allocate our resources the best way possible.

Jenny: Well, I understand that question in how you’re asking it. But I guess I’m coming at it from an innovator perspective, and you talked about innovation. How do you take innovation and apply it to be as efficient as possible? My husband does a ton of investing and all entrepreneurs would say, “If you just gave me more money, I could solve this problem.” Basically, it’s the wrong question. It’s, how do you successfully join these different stakeholder groups to come to a most efficient, most inexpensive, most curative way possible?

This is one effort to do that, and I think there are other alternatives that could accomplish the same objective when you look at inefficiencies in the whole process. So you mentioned earlier that drug pricing was one of those, and there are other medical costs that are probably one of those. So I understand your perspective how you’re looking at different diseases and how do we share the wealth, basically, or make sure the other things get taken care of.

But I think it’s fundamentally the wrong question. I think it’s more of a question of, how do we innovate successfully? How do we share data so that we’re coming to the right conclusions faster? How do we encourage patients to help be part of that process to help identify the inefficiencies and help bring them to the clinic? Because I think as a stakeholder group, I’ve seen what they’re capable of doing and adding to the process.

Dr. Pearson: Well, I couldn’t agree more. And I’ve actually been happy that the Patient-Centered Outcomes Research Institute or PCORI has been sending representatives to our meetings because they feel like they can learn ideas for what are the research needs that really address things that patients really feel are important. And a lot of the structural issues that you raised are critically important. How do we increase the efficiency or the ease of patients finding out about clinical trials? How do we structure those clinical trials so that we get the results more quickly and more efficiently, and that the data actually get published and not just buried sometimes, which still happens?

There are a lot of really important questions about how to reconfigure the overall enterprise. I hope that we contribute to that conversation as much as anything else because having a more efficient drug development testing and approval process would be really helpful for everybody. I completely agree.

Jenny: As a myeloma patient who has this disease, we are so grateful for these new treatments. I think it will take some time to understand how they are best used and in what order and timing and staging. Those were some of the comments that were made at ASH by some of the myeloma specialists, that it will take some time. One of the issues in the ICER draft is that the criteria using this first-fail as a standard to affect possibly subsequent reimbursements for medication is I think something that we would like to understand better from you.

So on Myeloma Crowd Radio, like I mentioned, we talked to Dr. David Siegel and he said, “Failing a particular treatment might actually open the door to more effective treatments.” And some of these failed treatments, like I mentioned, with panobinostat could be resensitized at a later stage. I think one of the big concerns is how do the ICER recommendations stay flexible? Because when I talked to Dr. Landgren, he said, “You know, we used to have these European meetings on myeloma every two years. And now, every six months, things are happening in six months incrementally.”

So if you put a framework in place that says, “Okay, this is the best value for relapsed/refractory patients,” how do you stay flexible enough to have these six-month iterations?

Dr. Pearson: Again, great questions. Just to be clear, there’s nothing in our report that suggests, or even addresses the question of fail-first approaches to therapy, nothing in there at all about that. But what I’d like to do at the meeting is actually to ask the insurers because, for instance, when we did that with hepatitis C, I said, “So, this new treatment for hepatitis C is really, really expensive and patients are taking the old treatment, 70% of them were cured. Now it’s 98%.” But you know, 70% of people get cured. I was being devil’s advocate.

And I said, “So, should we do a step therapy where they have to take the old treatment, first fail it, and then get the new treatment?” And the answer was no. We don’t want to do that. The old treatments are too toxic. The new treatment is just that much better than the old treatment that it wouldn’t make any sense for a variety of reasons to do that. And we put that in our report. So basically, we sometimes poke people to explore places where we think that no one wants to go, just to make it entirely clear that—at least at our meeting—the opinions were that no, people don’t want to go that direction.

And again, I don’t want to forecast exactly what’s going to happen at the public meeting, but I wouldn’t be surprised if that’s exactly what happen, because I don’t see anybody viewing the evidence as suggesting that this is a good, much less an ideal place to even contemplate, which is called fail-first or step therapy approaches. And in terms of flexibility, that’s again, if we remember where we’re starting from, ICER doesn’t make coverage recommendations. We don’t decide what Anthem or United Health Care or Aetna do. We put out a report, and basically, they are doing that on an ongoing basis. What they do is they usually have committees of doctors. I actually think they should have more committees that include patients, but anyway, that’s another issue maybe. But they reach out to clinical advisory groups every once in a while at least on an annual basis to have a standing process for revisiting their coverage.

So if that needs to be spread up, again, it’s something that we might want to talk about at our public meeting. I don’t think our report is going to slow that down. It’s not like we say, “This is the way the world is and it won’t change in 6 months or 12 months.” We’re often very clear how rapidly things are changing. Unlike a lot of health technology assessment groups, we actually allow the use of information from abstracts at meetings, particularly, in areas where things are moving so fast. We know that if we wait for it to appear in peer reviewed literature, it’s obsolete.

Jenny: It’s old.

Dr. Pearson: Yes. That opens up some risk because believe me, I’ve seen lots of abstracts that didn’t end up really panning out when it got to the published literature. But we still feel that in situations like multiple myeloma, it’s worth taking the risk and including information, the latest information we can get. And then we say that it’s still obsolete because by the time we get around, we just have to recognize it. As you said, things are moving very fast. And that is one of the reasons that people in insurance companies tend not to put really strict criteria around these kinds of drugs. They tend to use the FDA label and not do too much more.

Jenny: So what kind of follow-up do you do? Let’s say you submit this report and it’s a public report and you make these recommendations. How do you stay on top of what’s happening in myeloma, like let’s say these checkpoint inhibitors come out, or the CAR T-cell treatments come out, and three months down the road, they are now in clinic, and some local oncologist somewhere is looking at some of the recommendations and saying, “Well, it looks like I should use this triple combo.” How do you stay current?

Dr. Pearson: Well, that’s another good question. Health technology says, “We’re not set up like the NCCN where we have groups to revisit it and change the guidelines.” We’re not creating guidelines. We basically say, “This is a snapshot of the evidence.” And will we ever come back and redo it? Probably. I mean if there’s another major new step change in therapy, maybe it is the PD-1s or maybe it’s CAR T or something completely different. Now again, sometimes, the evidence is so obvious. That’s another reason that sometimes we don’t do a report because people say, “No. Everybody knows what to do. We’re going to use this drug, use it for everybody we can, and the price is either immaterial or we think the price will be reasonable or whatever.”

Then we don’t really have much to add to that discussion. It’s more aware, people are swimming around, wondering what to do with either new things or even older things. So we just have to be very humble and say that we try to be clear that our results are not guidelines and that we do not have a formal update process, whereby, every six months, they are updated. There are groups like NCCN that that’s their mission and it’s not ours.

Jenny: Yes. What I’ve heard from some of the doctors, and you’ve probably heard this too, is that some of it is just learning through experience. So having the flexibility and the creativity to say, “I’m going to use these in combination and then maybe I do retrospective study and see whom they worked best for.” Let’s say people with 11;14, or something like that, do best with this triple combination over that. How do the recommendations allow physicians to use this kind of professional judgment? I think we both agree that having different philosophies of treatment is not necessarily that great.

It frustrates patients. You know, it’s frustrating for patients that you’ll go see a myeloma specialist somewhere and you’ll see one across the country and they both have radically different opinions. I think we both agree that having data around it would give us better outcomes. But that’s a lot of data, and the systems don’t exist yet. So a lot of these doctors are saying, “Okay. Well, we’re doing that through clinical trials.” So, how does the reporting influence this learning through experience?

Dr. Pearson: Another great question. I think we’d both agree that there’s a difference between doctors just adlibbing and saying, “What I do is an art and you can never study it.” And in my 10 years or 20 years of experience, I know what I’m doing. That’s not the learning from experience that I think other —

Jenny: No.

Dr. Pearson: So we want them to be organized in clinical trials. And through a clinical trial, they’d have to get approval from an institutional review board to make sure that patients understand that they have other options of treatment—they don’t have to enroll, what are the risks and benefits. And hopefully, there is a system to learn from that. Usually, our report would probably not have much of an interference with that. There are some insurers like Anthem, I think, that have pathways where doctors will get preferentially reimbursed if they use a certain regimen or a set of regimens.

Usually, what they do is they go to NCCN and they take several out of the multiple “appropriate regimens” and they negotiate a price and they decide to set some of the less expensive but “equally effective regimens” as the the preferred regimens. It doesn’t mean that the patients can’t get the others. It just means that the doctors themselves might have a financial incentive to use certain regimens over others.

Actually, with your question, you may know more than I do. I’m not really 100% sure but within a clinical trial, usually, the health plans have an arrangement so that it either supersedes if their patients are in a trial that I think that the payment is not structured the same as for patients who are not in a clinical trial. Basically, your point is well-taken.

Jenny: Yeah, it depends.

Dr. Pearson: We should have systems so that people who were doing formal clinical trials with a goal of expanding the learning in the future, well, sometimes they have to seek funding from the NIH or somewhere to help them gather the data. But in general, we should try to have reimbursement systems that allow for that because we’re never going to get a lot of head to head trials before FDA approval. We know that. So we need to do some good trials afterwards.

Jenny: Yes. And that’s one of the reasons that we created this show is to encourage patients to participate in clinical trials. And there are things, I think, that groups like yours and the insurers could do, which will be one of my following suggestions. But one question that I have before, and I know I want to open this up to caller questions, is how do you go about engaging stakeholders, and especially, patients in this process? Because I have one friend, Yelak Biru, who is coming to the meeting who is — it looks like the only patient representative speaking there.

So, how do you engage these patients who — and there are many that are actually like me that have taken this deep dive into our own disease. We are highly motivated to cure ourselves, and want to do everything as efficiently and as curatively as possible.

Dr. Pearson: Right. So we definitely have pride, and definitely recognize that we need to figure out better ways to do this. So what we currently do is when we are getting ready to announce a topic, we basically do a scan and try to figure out what are the key patient advocacy groups with whom we can connect to learn more about this topic. So for here, here was the Multiple Myeloma Research Foundation and the International Myeloma Foundation. And then we often end up talking to other patients who either — because we put out a press release saying we’re going to do this topic and we welcome public comment.

And then we have several different cycles. The first part of our whole process is what we call the scoping of the review. And that’s where we actually make the final judgment of which drugs or whatever it is we’re going to look at. What’s the real question we’re trying to answer for which patients? What are the comparators going to be? We put that out as a draft after having talked to a lot of clinical experts and patients to try to get our own grounding. But then we put that out for public comment, and again, we get lots of comments from people; doctors, patients, and certainly, the manufacturers. We also engage with them at the very beginning.

And then we start off on our review, basically, once we have set that up. And then we do our review. And at the end of that, we have another public comment. We have press release, public comment on the draft of the evidence review. Actually, through the months we’re doing that, we’re usually trying to figure out how we’re going to put together our public meeting. We always have a public comment phase at every meeting, and I think there are going to be five or six patients who have signed up to give comments. Yelak is one of them.

Anyway, there’s a public comment phase, but we just have lots of phone calls. I don’t know how many phone calls we’ve had with MMRF. What I hope is that over time, people will gain a better understanding of what our intent is and what we do. As I said, I think we need to do a better job of having conversations like this one today where people can have a longer time to get to know us and to ask questions.

But anyway, then at the meeting, we have the public comment and we have, as I mentioned, general public representatives on the C-PACK Council. But as far as what we call our policy roundtable, we have insurers, one or two. Usually, we have a couple of clinical experts, and we have at least one or two. We have two patients for this panel.

So they are part of this discussion and I run it with like Phil Donahue, and that’s where again, I raised questions about:What do patients think of this? What do patients need? What’s missing? What about the future research? What about the value and the coverage issues? I just try to draw that out in a conversation that involves that panel that includes at least one or two patients. So we need to do better —

Jenny: Yeah. I would suggest that a panel, like of eight to ten patients, would give you a very good overview of the different patient experiences. There are a lot of patients that look at it very academically. They’re very studious about their disease. They can give a very deep dive both into the science and into the different approaches. We have several patients that are on our advisory board that help craft clinical trials.

Dr. Pearson: Part of what we think we need to improve is our scanning the horizon. I don’t know how we missed you, Jenny. I really don’t. It would have been great to have talked to you with the beginning of our process like we did with some of the other groups. So we need to do that better. And we’re also going to be working on a template because you know, we think we know how these groups can help us by giving us perspectives like you just talked about. But for some of the patient groups, they’re much less sophisticated and we were finding that we probably need to create a template that they fill out at least as part of the process. For others, we just need to be quiet and let them talk.

We’ve learned a lot about myeloma. Again as I mentioned, it has been a long time since I personally treated patients, and I’m just a single doc, anyway. So we learned a lot about the different kinds of roller coasters that patients that are on, the importance of having oral options that can, for some patients, make a major difference. This used to be a condition for which patients, usually, were told had a very grim prognosis. You had a few months to get your things in order. And now they’re living for years, and that may mean that they have the opportunity to be alive for the next step change in innovation. We’ve heard all of this but I do think we need, and are working on having a more structured approach to making sure that we capture a broader set of patient inputs.

Jenny: I think it would be really helpful for you, because if you look at myeloma patients, we face our mortality every day. And the greatest gift that each of us is given, whether we have a disease or not, is the gift of life. And our second greatest gift is the right to direct that life. So if there is a perception that either one of those is being impacted by an analysis or a report, you’ll get a visceral reaction because you’re impacting those two core gifts.

Overall, I think we have the same objective. Let’s find the very best treatment option for each individual patient. Can I share a couple ideas of how I think your group could help do that?

Dr. Pearson: Oh, absolutely.

Jenny: Well, we talked a little bit about this before we started this show, clinical trial participation. So I would suggest that especially insurers, if they want to accelerate the rate of a cure and they can leverage patients as partners in that effort, if we all as a community, insurers drug companies, patients, doctors, encourage greater participation in clinical trials, and every patient was introduced to this concept as they begin their treatment, we could really achieve results similar to childhood leukemias when they over 80% participation rates, and we, adult cancers have 3-5% participation rates. It’s sad and it’s just pathetic.

So, can we get to a cure faster and save money? Yes, I think we can, by participating in clinical trials. And I think that’s a group effort. I think that’s something you can suggest. I think it’s something we can suggest, and we could definitely work together on that topic because it would speed things up dramatically. It would cut rates in half or more.

Dr. Pearson: Good.

Jenny: Then I think what you said earlier, this is my perception and probably no one else’s, but you’re trying to take data and standardize data to help make clinical decisions about, you know, “we shouldn’t have all these different opinions, we just let the data decide” which is really what patients would like to do too. We need more data. We don’t need opinions, we need data because you go to a local oncologist, they’re going to treat you with what they might be familiar with, and they might be confused about adding in this new drug treatment options. Simply because they just don’t know how to do it.

I think that’s something that we could all do together, but I think as we do it together, then there’s less lack of awareness on the patient side. “What’s happening (with ICER) and am I going to be prevented from getting treatment that I need?” because you’re having that kind of reaction. So if that’s not the objective, let’s involve patients so they can give you that feedback upfront and you’re not getting it on the backend. I think we share the same objective. It’s this:  let’s get to a cure as fast as possible so we’re not treating this as this chronic disease with four drugs for the rest of my life. That’s not something that I want as a patient. So, that would be great.

Dr. Pearson: Absolutely. You don’t want doctors just adlibbing and not really knowing what they’re doing and not paying attention to the process of learning from each patient’s experience. I think that’s really important.

So this is helpful. I mean these are issues that since I’m moderating the policy roundtable at the meeting on Thursday, I can certainly bring up and to have it hammered home there at the meeting as well that these are important issues. But I also hear your message about, again, trying to figure out a way to work proactively earlier in the process, as well with a broader set of patients so that the general reaction is more balanced, I guess. I mean, there are still going to be some people who think that any diminution in the price is somehow going to reduce innovation in that space. I don’t think that’s true but it’s always going to be part of the discussion.

Jenny: Well, I think that’s a concern for patients because there’s been so much innovation in the myeloma space. We’ve had doctors now say the word “cure” for the first time in the last, I would say, two years. And when I started doing the interviews, it was more like, “Well, this is a chronic disease. We’re just going to be able to manage it.” And now, with some of the different treatments and treatment combos, Dr. Siegel, I’ve mentioned him a few times, I have done lots of interviews with lots of different doctors, but he said, “We might even have the cure now because it’s just a matter of which combination is right for which patient.” And we might be able to do that (for example, Is it stem cell transplant upfront or later?) All the efforts are good. They’re just not cohesive, and the data sharing is a major problem that’s preventing us from coming to those conclusions.

Dr. Pearson: Yes, I agree.

Jenny: I want to open it up for our caller questions. So if you don’t mind for a few minutes, I know we’re at our hour but we’ll open up also. Is that okay?

Dr. Pearson: Sure.

Jenny: All right. So we have a caller.

Caller: Yes. This is Garry Peterson. I’m the Editor of myelomasurvival.com, which is a website. Actually, I have had a few blog post centered on just this particular topic. And it’s my concern and my fear. And to tell you the truth, I’ve heard you and you are eloquent. I mean, unbelievably eloquent in the way that you present this. And I’m sure the folks in Europe at NICE are equally as eloquent in how they explain how can you put a value on life.

And from a patient’s perspective, what we see is that — ICER is, in my opinion, very similar to NICE, at least in the way that you are approaching the whole cost per QALY. As a result, what I see or what I have the sense of is that you’re overlaying a nationalized healthcare system of allocation onto the American system of healthcare. And what I mean by that is that what they do in Europe with NICE, which is what I see you guys doing, is finding a way to allocate resources that are very, very limited to the healthcare process.

Now, what that means from your executive summary for multiple myeloma, and I think I’m going to say two things about that. First of which is that our drugs are approved for second and third-line. But generally, what you see happening is that it always, because of our disease, and now we have so many clones upfront, our disease is yearly best treated the most effective when you use multiple drugs early on when the clones haven’t had a chance to morph into more aggressive clones.

So what you find is that our — in our first relapse, it’s half as long as the third relapse, it’s half as long as the fourth relapse. So if you can bring that back further in the process, you have better outcomes. And that doesn’t show up in your analysis, whatsoever. So the fear is, given what you show in your executive summary, is that none of these drugs are effective at their current values. And what Blue Cross Blue Shield of California has already said is that they are going to use this to limit or reject the use of certain drugs, but also to negotiate the price of those drugs. And I’m okay with them negotiating the price of those drugs.

What I’m not okay with is overlaying a system that is used for nationalized healthcare over our system, which is not bad. So that’s my fear. My fear is that you’re going to limit our options just when they’ve exploded on the scene. And we have all these great things in our future, and they’re going to be taken away. So that’s my fear. And I’d tell you what, that’s every patient’s fear, and you’ve done nothing, absolutely nothing to put that fear to sleep.

Dr. Pearson: Oh, I’m sorry you feel that way. As I mentioned, I am unaware of any insurer that is interested in saying no to any of these drugs. I do know that clinical experts, some of them at least, would disagree with your contention that we know that treating patients with all of the drugs or a very large number of them upfront is definitely better than a slower sequential approach.

Caller: Then you are uninformed, unfortunately.

Jenny: Yeah, I would have to agree too, because I’ve heard that multiple times that if you treat and you can eliminate the clone upfront, you’re more likely to stay in a longer remission.

Dr. Pearson: Right. I mean I’ve heard that from some clinical experts and from some who say we actually have no evidence that that’s the case.

Caller: Well, evidence is so overwhelming in that favor.

Dr. Pearson: Well, we could talk about which studies would have demonstrated that, but I’m not aware of any. And again, our job was to try to bring information about the newer drug, in particular, that had just been launched. So obviously, we’re not going to have years of data on them. We will want them but we don’t have it right now. I would say that again, part of the concern about any approach to try to judge value, I hope it can be grounded in where we are now. What I fear, personally, is that health plans that are asked to try to restrain healthcare cost in the short-term will ignore the longer term benefits of drugs like these. And we’ll feel forced in some way or we’ll take options to restrict care not just for multiple myeloma but for lots of other conditions, unless we start to have some discussion around how pricing can align with that value.

Believe me, if the PD-1s end up being the homerun that I hope they are, or a CAR T or whatever, we’re going to want to pay those companies and pay for those drugs extremely handsomely, because they will have brought really important benefits to patients. I guess just part of the question is again, can we at least talk about the idea that if a new drug adds very little, whether it’s myeloma or something else, if it adds very little or no established benefits to patients, should it have a high premium on the price?

There was a famous case of a cancer drug a couple of years ago that Sloan Kettering decided not to use because it was no better but price twice as high. So their question is, can we at least have that conversation? Because in my mind, that’s what’s going to benefit patients as well as continued innovation is making sure that we can price it fairly and make sure that it’s affordable for patients in the healthcare system.

Caller: Yeah. And I think that one of the things that if you look at Mayo’s clinical proceedings, Dr. Rajkumar did an analysis on that. And if you’d seen it, he comes up with some great recommendations on how to reign in some of the issues that we have with regard to the monopoly of pricing of cancer drugs. Like for example, letting Medicare negotiate prices, because we pay twice as much as they do in Europe for the same exact drugs. So why can’t we do that? And when you figure that we have $149 billion for the drugs that we spend every year in that B and D program for Medicare, we can save a ton of money, and we should do that.

There are things that we can do right now within our own system without overlaying something from nationalized healthcare over the top that we can come up with similar savings or even better. And the fact of the matter is if we’re going to have an ICER or a NICE, then I suggest we go to the nationalized healthcare system. Save the 9% that we pay over and above what the Europeans pay, or 19%. Their 9%. And they have better outcomes than we do.

If we’re going to overlay the NICE and ICER system on top of ours, then we might as well have nationalized healthcare because we’re going to limit our options just like they do in Europe. They have the fight tooth and nail for Revlimid. They had to determine that your QALY that you come up with, right, that the QALY for a cancer patient was 1.4 of that for a person who is totally well. They had to come up with that. They had to increase because of that. It’s one of those things where when you look at QALY, poor Stephen Hawking has a negative QALY to convince him that.

Dr. Pearson: No, he doesn’t.

Caller: Yes, he does. He can’t take care of himself. He has a poor quality of life. It’s negative based on the information from NICE.

Dr. Pearson: No. I’m sorry from whomever you got that information, that’s just completely false.

Caller: All you got to do is look at the NICE website.

Dr. Pearson: No, the idea that’s —

Caller: Read your information.

Dr. Pearson: I’m sorry, sir. You’re wrong.

Caller: I beg to differ, because according to the information, and this is the information, I will leave it at that, is that they said that you can go negative if in fact you can’t take care of yourself, that you have a poor quality of life. Your QALY can be negative. So I’ll leave it at that. That’s what they say, so that’s why I said it about Stephen Hawking.

Dr. Pearson: Okay. Just one last quick point about the QALY. Remember, this is an American invention used by countries around the world, and it’s been used by American Health Economics specialists and doctors for 30 years. It’s nothing new or nefarious from overseas. They use it more explicitly as part of a public process in England. Other countries use it in a variety of different ways. But it’s the core approach in trying to judge the long-term value of healthcare innovation. So, how we want to use it and how it’s applied is certainly important. But I just don’t want it to be necessarily mischaracterized as some alien national health system tool.

Caller: Well, that’s where it is right now. I’ll leave it at that. We’ll leave it to the next questioner. I guess my point being is I just want to let you know that this is something that most patients feel is going to limit their ability to obtain care.

Dr. Pearson: Well, I hope that we can assuage that concern, and my hope that they will have greater access to care that will benefit them in part by helping, over the long-term, to align the prices with the value that real patients do get.

Jenny: Okay. Thanks for your questions and for the answer. I appreciate it. Yeah. I think as myeloma patients, we look at myeloma as an international disease. We know specialists. You know, I have interviewed specialists in Spain and Germany, in the UK, in different places. We see places like Australia, and in Australia, they’re crazy. They say you can have one drug at a time, which is insane. So you take dexamethasone until you relapse dex, and then you take Velcade until you relapse Velcade. It’s the most inane way of treating myeloma that I’ve ever seen.

So you look at the UK and people like Gareth Morgan who started Myeloma UK. He’s now at UAMS because of the flexibility of creating clinical trials. And Europeans have had to collaborate together, and most of their patients are in clinical trials because it’s the only way they can get access to these new drugs. We are looking at it from an international perspective and are saying, “You know, what we have right now, the innovation levels right now in the U.S. are just unparalleled.” So I think the concern is that anything that would restrict that level of innovation and speed it up, we feel like, I think patients as a whole have been now told by some of the researchers we are very close to a cure. So I think that’s the major concern.

Okay. Our second question. Go ahead.

Dr. Pearson: Just real quick. I was going to say, I was talking to an old Texas oil man the other day. For some reason, he’s asking me about what I did and I told him some of the issues around how drugs are priced. And he actually told me, he said, “You know what’s interesting in the oil business, we only got real innovation when the prices dropped, when everybody could get whatever they wanted for the next barrel of oil. No one cared about innovation. They just threw money at things and they would get it back.” He said, “The best innovation, the most focused efficient innovation we ever had was when the price of oil drops significantly.”

So every once in a while, I think we’re not talking about shutting off innovation. We’re talking about making it smarter and more focused. And coming to market, honestly, at a price that’s aligned with the value so that — I mean, I don’t feel great about the international situation. There are lots of issues at hand but one of them is, why don’t the drug companies price it so that the countries feel like they can afford it? That’s not the only answer to this situation. But I just have a hard time believing that all of these other countries are evil in some way. It’s ultimately up to the drug company to price it as they feel they can. I know they need to make a profit. Don’t believe in profits, but it’s all a balance.

So the question is, how can we strike the balance in our country that will get us the innovation, but hopefully, not having us pay two to three to six times as much for these drugs as other countries?

Jenny: Well, I think that’s important. We have another caller, Go ahead with your question.

Caller: Yes. I have a question, but based on the conversation that just took place, I’ll have comment before my question. And I think it’s important for everybody out there to realize that NICE is United Kingdom-specific. So when you talk about Europe, it’s a lot of different things, and it’s a lot of different systems. So for example, if you look at countries like France and Germany, and certain countries in Scandinavia, they actually have higher survival rates in myeloma than we do in the United States.

And in France, a lot of that is attributable to the fact that 60% to 80% of myeloma patients take part in clinical trials. So I think we don’t have the greatest system in the world, in the United States, but neither do we have the worst in other parts of the world. There is a balance and there are areas of excellence and that even, Jenny, you even fund crowdfunding research project in Germany. So there’s good stuff going on in other parts of the world, and I think we need to recognize that. And again, I think it’s important to know that NICE is UK-specific. It has nothing to do with anything that happens outside of the UK. So anyway, that’s my comment.

My question comes back to the American point of view. One of the things that concerns me about the ICER report where I don’t see the flexibility and I looked through your report and I saw an indication of specific pricing issues. You even mentioned the idea of how it would affect off-label drug use. And in the United States, for most cancers, most drugs that are prescribed to patients are prescribed off-label, but we have flexibility and we have insurers and so forth that will pay for that. Medicare even pays for off-label indications now on cancer drugs after they did that with thalidomide.

Even in your report on indications of specific pricing, you mentioned that the drugs that would be selected under that system that you propose would have “minimal off-label usage” to quote what I’m looking at right now. So what would your recommendations mean for the off-label prescription of drugs? The other question I have is, wouldn’t your system ultimately lead to more layers of bureaucracy and more layers of reporting? And wouldn’t that counter the idea of what you’re trying to do? I’m just very unclear about that and I’d like to know your point of view about that.

Dr. Pearson: Sure, thanks. Thank you. I’m glad you’re able to navigate our website to find some of these things. We’ve just updated it so it’s actually nice to hear that people are able to find some of these things. So our off-label, we did a policy paper on off-label — sorry, it wasn’t on off-label, it’s on indication-specific pricing, completely disconnected from our evidence reports because as you know, some of the insurers and manufacturers have started to create some contracts around setting up an — by indication-specific pricing means in some way, trying to pay differently for a drug depending on what purpose it’s used for.

So I guess conceptually, if a drug is really, really effective at treating gastric cancer but really not that effective at treating kidney cancer, you would want to pay more for it for gastric cancer and less for kidney. That’s the basic idea. The point our report made about off-label is that just when to do these kinds of arrangements in the U.S. system, manufacturers and health plans have to talk to each other and make up a contract. And if a drug happens to be used a lot off-label, it just makes it not a great candidate for an indication-specific pricing approach because manufacturers aren’t allowed to talk about off-label uses. So they can’t really sit across the table from a health plan and talk about how to pay for an off-label use. The law forbids that.

So that was the only point. It was the cart and horses—if a drug is already used a lot off-label, it’s probably not that good a candidate for indication-specific pricing. So in any area of cancer, for instance, where there’s a lot of off-label use, again, we hope that that’s supported by some good evidence that the compendia or other people will recognize. But if it is that way, then it just makes it really difficult. It’s not impossible area in which to have manufacturers and payers try to contract around indication-specific use.

I hope that was clear. I’m, unfortunately, not really sure what you mean about why our approach would add more layers of bureaucracy necessarily, unless you meant the indications with the pricing piece, specifically. Do you want to clarify that?

Caller: Yeah. Again, I’m just speculating as I look at it and maybe I’m mixing my metaphors here. Let’s go back to the idea that you talked about, about you might pay more for a drug if it’s more effective and less for a drug if it’s less effective. It seems to me that that concept in and of itself would create another layer of bureaucracy because you would have to determine that, you would have to gather data about that. So that was what I meant about that.

And even along those lines, let’s take a specific cancer like myeloma, since that’s what we’re talking about, and you have a cancer that’s first, second, third, fourth-line. It has treatments in those different lines. And if you find that the same drug is more effective on the second-line than it was on the frontline, would, under indication-specific pricing, would that mean that the more effective it becomes, the more expensive that particular drug could become even for the same disease type but you differentiate between progression?

Dr. Pearson: Yes. I guess conceptually. Most people have read our report, which basically came out of some background work but arose from the discussion we had at our meeting between manufacturers and payers. Most people have interpreted our report on education-specific pricing as a cautionary tale that it sounds conceptually easy but it’s actually really hard to put into practice.

Caller: Okay. That’s the conclusion I drew.

Dr. Pearson: Yes, that’s basically what I think our report says. So you’re right, it raises administrative issues around where the data are going to come, who’s going to track them, who’s going to reconcile, which patients used it for this reason versus that reason. We already have an incredibly byzantine process through which drugs get from point A through to the patient. I’m not sensing a huge amount of enthusiasm to do a lot of indication-specific pricing in the U.S. system.

Caller: Okay. So you don’t see indication-specific pricing as being part of your recommendations that you’re going to be coming out with for myeloma.

Dr. Pearson: No. I actually hadn’t even thought if it as something I would raise at the policy roundtable. I guess I could but it’s really just a bump on the landscape at this point. I think some payers and manufacturers want to give it a try for a variety of reasons. But again, there are lots of hurdles to it and to me, multiple myeloma certainly does not present itself as the poster child for a good area to think about it.

Caller: Okay. Thank you so much.

Dr. Pearson: Sure.

Jenny: Dr. Pearson, we are so grateful that you spent time with us today. We know we’re asking you some difficult questions. So I’m grateful for the grace with which you’ve answered them.

Dr. Pearson: I just want to add, I’m grateful that people are honest. I mean this is part of what I hoped ICER would do, which is to generate honest discussion, and that doesn’t always make it easy. So I don’t mind tough questions and I don’t need to try to convince everybody that I’m right. I like the conversation and the broader thinking that this makes all of us do.

Jenny: Well, I think it presents an opportunity. So just in closing, I think I do have this unique opportunity to create a patient-centric model for value, where we invest in the genomic diagnostics that you mentioned earlier to make sure the right drug goes to the right patient at the right time. I think this is an area where we need serious collaboration among all the stakeholders; the insurers, the pharmaceutical industries, groups like yours, clinicians, research, and most of all, bringing patients and their advocates into that conversation because there is much that they can add that’s very valuable.

Dr. Pearson: Thank you, Jenny, that’s very well-said. And since you won’t be on our policy roundtable, I hope Yelak represents these views very strongly, and I will be sure to bring them up as well.

Jenny: Well, Yelak is terrific. You’ll enjoy working with him.

Dr. Pearson: Good.

Jenny: Okay. Thank you so much for joining us today.

Dr. Pearson: Thank you, everybody. I appreciate it.

Jenny: Thanks for listening to another episode of Myeloma Crowd Radio. Join us for our future shows to learn more about the latest in myeloma research and what it means for you.

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About Author

Myeloma survivor, patient advocate, wife, mom of 6. Believer that patients can help accelerate a cure by weighing in and participating in clinical trials. Founder of Myeloma Crowd, Myeloma Crowd Radio and the CrowdCare Foundation.

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