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PAH Treatment Driven by Data From Peer-Reviewed Literature


Derek van Amerongen, MD, MS: Well, there are many trials that have been undertaken in PAH. One of the fundamental functions of the health plan, pharmacy, and therapeutics review committee is to go through those trials and understand what they say and how they support the use of one treatment versus another. We believe that those are really primarily the purviews of the clinicians and the researchers in terms of deciding which of these trials really has the greatest impact. We rely very heavily on the peer review literature to guide us in terms of understanding which trial has really demonstrated benefit and improvement over another trial. I don’t think that selecting one trial and elevating it above another is really a health plan role, or should be a health plan role.

Ultimately, the arbiter of that evaluation is not the health plan. Quite frankly, it’s the peer-reviewed literature. It’s the clinicians, the experts in the field, the KOLs across the country who have looked at this data, and then put down, in the peer-reviewed literature, their assessments and their evaluations of which are going to lead to the optimal outcomes and lead to the best patient endpoint. We want to incorporate that in our evaluations and in our medical policies. But, it’s always a challenge when I’m talking to clinicians and they want to get into a deep discussion about trials. That, again, should be ultimately something that comes to us from the literature.

There’s always an interesting challenge that health plans, and I would say the entire medical community in the United States, has when we’re presented with European data. There’s a lot of tremendous work going on in Europe. I think we need to understand it and read about it. And many of those trials are reported in tier one United States journals such as the New England Journal [of Medicine], JAMA, etc.

There is, to be quite honest, always a hesitation in adopting data from foreign sources, especially if they haven’t been reported in the United States. This is mainly because of the concerns over the discrepancies and the differences in terms of how medical care is structured and delivered in the United States versus other countries, especially in Europe.

At the end of the day, I do believe that foreign trials can have tremendous value, especially from a purely clinical standpoint. We have reviewed many of these over the years in a variety of different areas, including PAH. If the trial supports changes in direction in terms of leading, for example, to more combination therapy, or recommending different combinations based on functional class, etc, we would incorporate that information into our medical policies. Again, this is as long as it is in line with the FDA, the national guidelines, and all of the support we see in the United States literature.

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