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Factors Impacting Formulary Decision-Making in PAH


Derek van Amerongen, MD, MS: When we’re evaluating drugs at the health plan P&T, we want to have as holistic an understanding of the impact of that treatment as possible. Certainly, we want to understand what the primary clinical benefit is. And we talked a moment ago about the use of surrogate endpoints versus more real-world endpoints, which, again, is always a challenge when you’re talking about a rare disease.

But another critical part of that discussion revolves around the safety, the tolerability aspects. Is there a potential for a given treatment to increase adverse events such as a bloodstream infection, such as some type of other adverse outcome that might lead to a worsening of the patient’s condition? Certainly, when you’re talking about something like PAH—and this discussion comes up again in many areas, especially oncology—we’re talking about the use of powerful agents to treat an extremely difficult to manage disease. So, we have to balance the risks and the benefits. We have to understand that every drug will have its adverse events and potential safety issues. At the end of the day, I think a lot of that responsibility really falls upon two stakeholders. One is the FDA, because if the FDA approves a drug, then certainly what the FDA is saying is that that drug can be marketed and can be used across the country by clinicians. But then, the other important player in that discussion is the clinician who needs to decide. Is this drug right for this particular patient?

At the end of the day, I think that the health plan needs to step back and say, “For an FDA-approved drug, as long as the clinician is using it within the context of how that drug is indicated and should be used according to the literature, then it really is a clinician decision.”

One of the challenges that we’ve seen in many conditions over the last few years in PAH and other areas, such as oncology, is the advent of more and more combination therapy. As the data on a lot of these very challenging conditions evolves, we see the role for combination therapy. And PAH has a lot of data that supports the use of double and even triple combinations in terms of achieving the optimal endpoint.

From a managed care perspective, we want the member, the patient, to get the most effective treatment. If that involves combination therapy, then that’s what the patient should receive. The only challenge that we face as a health plan is making sure that the decision on which combinations to use is really evidence based, that if a clinician is requesting a double or triple combination, that patient really matches those criteria as laid out in the literature and by the FDA. And so, we want to validate that combination therapy is being targeted appropriately, and is going to the right individuals. But, as the data matures, and as we learn more about the most effective way to treat conditions like PAH, I believe combination therapy is going to be something we’re going to be seeing more and more in the future.

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