Michael Kolodziej, MD: It’s fascinating that over the last few years, we’ve seen the development of multiple approved pathways within the FDA, the most recent being breakthrough designation. And so, you get breakthrough designation if you’ve got a good story, or you’ve got a marker, or there’s really an unmet need. I would say, as an observer, it seems to me that the hurdle for approval via that mechanism seems to be lower, although when I say that, my colleagues at the FDA take great offense. Let’s just say, for the sake of argument, that we do see drugs getting to market faster. If you don’t think what I’m saying is true, just remember how quickly some of the immunotherapies made their way through the regulatory process—and I’m talking about days, not weeks, years, or months.
The real question is, are we confident that the results of the trials that led to regulatory approval through the FDA are, in fact, representative of what we’re going to see in the real world? And I think it’s got nothing to do with being a payer. I think everybody should be a little bit concerned about that. We’ve seen, for example, that in Europe, where they often hesitate on coverage based on cost, frequently the way those drugs come to market is through some provisional access pending further clinical data. That isn’t such a bad idea. In the old accelerated approval, those postmarketing studies were often a component of the approval and often were not completed, unfortunately.
So, as we look at the breakthrough designation, my personal opinion is we should take a step back and think about a requirement to collect some real-world evidence. We are getting what we thought we were going to get from some of these really exciting therapies. See, the goal should not be to try to limit access to the market for some stuff that really looks exciting. I think accountability is a good thing, and that’s why I feel that way.
The whole question of getting to market with a very precise FDA label, with the hope of expanding your market access based on subsequent approvals, has always been a model that has worked for pharma. I can’t see why it changes today. Because in the absence of action by Congress, it just seems to me that that same pathway to access exists now as it always did.
Now, as we think about this in a more global fashion, I think the real opportunity is to start thinking about indication-specific pricing. From the payer perspective, it just doesn’t make a heck of a lot of sense to pay amount X for a disease like melanoma where there’s a great benefit and pay that same amount X in another malignancy where there isn’t as much benefit. That doesn’t make any sense. I think, as we work our way through reimbursement reform, the concept that indication-specific pricing might eliminate some market access barriers should be a really provocative thought, if I were a developer.
So, we know for a fact that the vast majority of drugs that are being approved by the FDA are being approved on surrogate endpoints as opposed to survival. Everybody says they want survival. Everybody recognizes that survival is a tough bar. Whether one surrogate endpoint is superior to another surrogate endpoint is a difficult question, and frankly, in the current reimbursement universe, isn’t one that the payers have to think about, or face, at all. Because, as we’ve discussed, reimbursement or coverage policy mirrors FDA approval, and then mirrors professional society endorsement, like progression-free survival response rate.
Now, my personal opinion is that as time goes on, as we start thinking about the shifting of risk from payers to providers to oncologists, they will demand a granular understanding because they will be bound by the regulations that the payers are. So, if I’m a manufacturer, I’m thinking, “Well, what makes my therapy attractive, a preferred treatment option, for a provider group that’s ultimately going to be at risk for cost of care?”