What Is the Cost of Recurrence in Multiple Myeloma?
Keith Stewart, MB, ChB: Let’s turn to some questions about reimbursement. Mary, do you see any differences between using 2, 3, or 4 drugs in the patient population or insurance coverage, from your perspective?
Mary E. DeRome: That’s something that we’re just really beginning to track, so I don’t really have the answer for you there. But certainly insurance coverage is—in some cases, for some patients—a major determinant in which therapies they’ll be taking, depending on cost.
Keith Stewart, MB, ChB: It’s probably easier for them if they are IV [intravenous] or subcutaneous as opposed to…because the oral therapies are where the problem seems to...
Mary E. DeRome: Correct, correct, correct. And many patients watch that very carefully and make sure that what they’re going to be given from their doctor is something that is covered, because they really don’t have any choice, and they don’t want to have to choose between paying other bills and taking their medication.
Keith Stewart, MB, ChB: Rafael, you talked earlier about the value equation of expense over time versus savings downstream. Do you want to just recap that for us again?
Rafael Fonseca, MD: Sure. I think when [we] do analysis of the economic implications of treatment one has to again consider the whole equation: the drugs, office visits, diagnostic tests, hospitalizations, etc. And I think we have a paucity of data in that regard.
I’ve been working on a number of projects. There was recently 1 that actually asked the question, “What is the cost of recurrence?” And what we find actually is that it’s cheaper to stay in remission even if you’re on treatment than to have a recurrence. Now that might say, “Well, wait a minute, what happens long term?” To be incredibly cynical one might say, “Well, the longer you live, the more expenses you incur in a payer.” That’s not always necessarily the case.
So there’s value in disease control. I think we all need to pay attention. At the end of the day in the world resources are sparse and people have to prioritize and there [are] going to be trade-offs. But I think as of now, as I mentioned before, we don’t have the answers on how to best gauge that value. And there [are] all the imponderables that come into the value equation, like being able to be present in a wedding, see a grandkid be born.
Another factor, which is key, which is really fundamental, is what the economists call the “value options.” And this is very important. The ability that you have that by living longer you can realize the benefits of cure and research, and that was best exemplified with the HIV/AIDS epidemic, where people who were able to stay on AZT [azidothymidine] ultimately saw highly active antiretroviral therapy, and many of them are still alive today. Versus those [who] could have or wouldn’t respond, then oftentimes ended up succumbing to their disease.
Keith Stewart, MB, ChB: Of course, [there is] the value to the society if you’re back in the workforce. Some of these groups, ICER [Institute for Clinical and Economic Review] is 1 of the groups in the US [and] NICE [National Institute for Health and Care Excellence] in the UK, are using sort of formal metrics for years saved and for dollar value. What do you think of all that, Andrzej?
Andrzej Jakubowiak, MD, PhD: They’re helpful tools, which potentially can guide us to making our suspense of value of the regimen versus potential gains and losses, financial losses. I look at them as a tool too and collect more information.
Keith Stewart, MB, ChB: Would you do economic analysis prospectively in all trials?
Andrzej Jakubowiak, MD, PhD: I think we should. I’m very absolutely in agreement with it, but I would like maybe to look and hear something [that] I haven’t heard a lot. But it’s with panels, [so] maybe [I could] potentially bounce [it] off you.
I think in the process there is [an] emerging gap, in my opinion, [between] what experts in the field would have done and what the system or payers will allow us to do. There was a time a few years ago, not long ago, that if a peer would ask me, for example, what I would do, I would make my argument [and] it would be usually listened to. That kind of got lost. I think we need to think about that. This field is so quickly emerging, the cost area in particular, that we need to keep more decision making in the hands of people who know more about what potentially can be beneficial as it is emerging with data, and allowing us to do some of those things [that] are eventually exactly in line with what Rafael just said: it is maybe a value to not give more expensive drugs right now, but then you’re going to lose later. And if they hear that from us, then maybe it would be potentially helpful to the patient.
Keith Stewart, MB, ChB: Mary, MMRF [Multiple Myeloma Research Foundation] has got quite involved with Harvard Business School and other people. How much are you looking at the economics of care? Or is that not something you’ve been tackling so far?
Mary E. DeRome: We’ve been really more looking at the direct-to-patient aspect of care, so really making sure that patients know that education and their own education in their disease and the way they interact with their care teams is very important for their outcome. And our particular area of that, the precision medicine initiative, has not been that involved with the reimbursement aspect.
Keith Stewart, MB, ChB: Do you think you should be more?
Mary E. DeRome: I mean, I think that there [are] definitely a lot of questions. It’s a very complicated area, and a lot of people spend a lot of time working on it. It’s a very resource-intensive effort, and in some ways there are some other nonprofit organizations in the myeloma field that are a little bit better equipped to move in that direction than the MMRF.
Keith Stewart, MB, ChB: Some of our colleagues in oncology with solid tumor know increasingly hematology is pretty vocal that they feel the prices are too high and there should be attempts to rein them in somewhat. Any reaction to that, Rafael?
Rafael Fonseca, MD: Yes. Well, let me start by saying, I think we have incredibly smart and passionate colleagues who feel that way. I just don’t think they have the complete assessment of the situation. And you know, we’ve had vocal and open conversations about this. I think the desire, of course, is to make things [as] economical as possible. But it’s not always possible that things are going to be at the aspirational price that would make it available, not only here but in the rest of the world, right? But we live in the real world, and that can happen.
I’m going to give you an example of ICER. I cannot comment broadly about ICER. But I did look very, very carefully at the ICER analysis. In fact, I watched the 5-hour video of the meeting for ICER. And I would suggest that there [are] serious limitations on how some of this value analysis [is] conducted, such that their application for myeloma care, in my mind, would be very short-sighted and not consistent with best practices. In fact, if you look at the report that was done, I believe [it] was a couple of years ago, 1 of their conclusions was that some of this value would come from the use of panobinostat. A drug that is no longer owned by the parent company, has been sold to a secondary company, which we really didn’t use in practice. There’s nothing wrong with the precepts that go into how the analysis was done, but the assumptions and the lack of expertise in the panel was quite evident at the time those conclusions came about. I think we should have panels, we should have groups, and maybe ICER. But it needs to be in a way that also includes a very, very deep part [of the] patient from experts in the field.
Andrzej Jakubowiak, MD, PhD: I agree.
Keith Stewart, MB, ChB: I think [there is] consensus in the United States, though we’ve been very fortunate. Do you feel the payers and physicians are on the same page, or do you think there’s, from the patient’s perspective, too much back and forth? How are patients feeling about that? Maybe they don’t even see it.
Mary E. DeRome: Well, they do. Patients, they do see it. And some patients have discussions with their doctors regarding, you know, I can get this therapy to you for a decreased cost, and this 1 I can’t get [because of] the insurance coverage. And there [are] many doctors [who] are conflicted about that—what kinds of therapies [can] they give to their myeloma patients based on cost? And patients do hear that argument, so they are concerned about it.
Keith Stewart, MB, ChB: Andrzej?
Andrzej Jakubowiak, MD, PhD: Well, I think that my biggest frustration at this point is that [the] decision about to go [or not] go for certain treatment at this point has [become] very much almost administrative—[you] kind of a check the boxes, list of things allowed or not allowed. And we lost this opportunity for an expert to make a case. I don’t have any problems being challenged. Why would you use this? Do you have evidence for that? [What] if I make an argument and I provide data? [What] is sufficient in my opinion—[and] you guys would agree with me and many of my colleagues would agree with me—I don’t think is the right development in what we have. The person who is on the other side would either tell me, “Well, I actually agree with you, but I am not empowered to really do anything about it,” [or] go to the higher level, and then I know how it ends.
Keith Stewart, MB, ChB: The only place I’ve really found challenge is actually in PET [positron emission tomography] scanning. [It] is the 1 I get the most....It always strikes me as rather ridiculous, but we are blessed in the United State with free access to drugs. I think the economic clouds are darkening in terms of, as the costs rise, obviously there’s more scrutiny and more oversight, [and] I think [this] is somewhat inevitable. But I do think we probably would all agree, if we can find cheaper, shorter alternatives, that would be ideal. But for now we’re going to do the best we can for our patients.
Mary E. DeRome: Certainly. I mean, this is not in any way a problem that’s limited to myeloma.
Keith Stewart, MB, ChB: No. But while myeloma has become a bit of a foster child for it because [of] expensive new drugs, right?
Mary E. DeRome: It has because of the cost of some of these therapies.