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Reimbursement With Treatment for Multiple Myeloma


Rafael Fonseca, MD: I have personally been involved with a number of efforts to better understand what the value is for the various interventions we have for multiple myeloma. We often talk about cost; we rarely talk about the whole question, about value. Now, we all should be mindful and should be concerned about the high prices that some of those medications command.

But on the other hand, we’re seeing the benefit, both directly that our patients express by having their disease controlled but also as economic analyses are done and we track with economists. By the out-stream beneficial effects with this, people that avoid hospitalizations, people that avoid blood transfusions, are people that can return to their productive, economic way of life.

So when we talk about the economics of drugs, it’s insufficient to merely talk about cost. Furthermore, the discussions about cost are highly limited because people use the sticker price when they give us analysis, and we have to consider the aspects of the rebates. And in particular for our patients, sure enough, there are some implications for how much the premiums will be as we introduce some of those more expensive drugs. But for the patient in the frontline in the need of treatment, the focus is on the co-pay portion of their responsibility.

We did an analysis regarding the economics of the treatment of multiple myeloma. This was agnostic to the drugs. We’re not trying to prove that a regimen was more effective or not. And we actually compare this over a number of years. This was published in Leukemia & Lymphoma in 2017.

There are many other costs that come into play, including expenses associated with the office visits, hospitalizations, and the administration of these medications. So, we do show that in fact these medicines do contribute in the cost and care of myeloma patients, but they’re hardly the only drug. And, in fact, there are other areas that appear to be driving the costs higher.

So we have to look at this in a holistic way, if you may. There’s some previous analysis on how some of the sages in particular have reduced the health resource utilization because of the prevention of complications, and we’re involved in studies that are trying to look at the economic burden of our recurrence. In fact, we had a poster like that at this particular meeting because from a payer perspective, if a person experiences a relapse because perhaps a less effective regimen is used, there are going to be some costs that are going to be incurred because of the need for the rescue of the complications from that disease progression.

As a clinician, I firmly believe that my only responsibility is to the person in front of me. I realize in the system we have where there’s a third-party payer, we have to work within the boundaries of what that payer may allow us to use or not. In another day, I could take off that hat and I could read a policy, and I could be advising people in what I think is proper or not. But physicians at the bedside need to focus exclusively, 100%, on what’s best for the patient and advocate for that.

I can’t imagine how a conversation would go with someone saying, “Listen, you know there’s this great drug”—whatever that drug is—"but I just feel like we’re spending too much money from the plan”. That is a complete betrayal of what we call the fiduciary responsibility for physicians as they’re dealing with patients. Now, the physician needs to discuss the economic implications of whatever treatment they’re doing with patients, but the realities for that person, it comes to the co-pays. This is simply an offer for those with commercial insurance because there are a number of programs. That needs to be told to the patients. They need to know those programs exist, and it’s quite a bit more complicated than Medicare beneficiaries because of the impossibility of the direct assistance for the co-pays. There’s some discussion going on right now with classes of protected drugs, co-pay accumulators, etc. But that is where the boundary exists for this transfer of patients and the co-pay contribution to their care.

There is a possibility that we will be facing, and, not surprisingly, because of the higher cost of these medications, more hurdles or difficulties in the access of some of these medications. So far, this is mostly manifest in the form of the prior authorizations that we have to obtain all the time. The logic behind them—it’s sometimes not there, but I understand it’s very hard. You know as a payer you want to follow your guidelines. And as an example, just this morning I got a denial for cyclophosphamide, the cheapest agent in the combination. I almost feel like there’s no logic behind that because it was just not on the pathway.

I am personally very concerned with the idea of step therapy, or what’s call failed first. This is a way through which payers can decide that you cannot get a drug unless you try something older and cheaper. Now, I can totally live with that if we’re talking about chronic hypertension. Maybe you try a diuretic, and if that doesn’t work, you’ll go to a beta blocker. That does not work for cancer. It doesn’t work because you’re going to expose patients to ineffective treatments, potentially grade resistance, and certainly the possibility of having the patient experience some toxicity that they did not need to have.

So I think we have to be very careful about this. I’ve explored the topic. There’s a recent white paper by Professor Sharona Hoffman from Case Western Reserve University that looks at the ethical and legal implications of step therapy.

There is a question of how much synchrony there is between payers and physicians. And everyone has their own interests and their own stake, and rightfully so—that’s how the system works. The biggest challenge is, how do you create that synchronization between both parties? And that is becoming very complex.

So after a meeting like this, many of us who devote our lives to multiple myeloma—and we think about nothing else but multiple myeloma—still scratch our heads on what’s the proper next step for therapy. Is it logical, is it proper, is it even fair to assume that a payer is going to be able to understand that? The answer is no. Is there any solution? No. Because a payer is going to be looking at, OK, what are the drugs that are going to be approved, whether we have FDA approval, what does the NCCN [National Comprehensive Cancer Network] say, and then, based on that, we will decide whether we will provide coverage.

For a payer to be able to make medical decisions, you have to be in the room with the patient because every patient is unique, and this is not a clique. This is just the reality of what we see in cancer practice. We cannot create cookie-cutter guidelines for most of the diseases. I can speak with authority about myeloma—it’s my area of expertise—but I can only imagine the same is true for lung cancer and breast cancer, etc. And so it is a very complicated situation right now.

Now we’re at the meeting where we’re seeing CR [complete response] rates in the large fraction of patients. We’re seeing MRD [minimal residual disease] negativity on multiple myeloma, which we could only hope for in the past. So I think that everything is moving in the right direction but with significant tension and friction.

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