Outcomes Assessment and Combination Therapy in PAH

Derek van Amerongen, MD, MS: One of the important topics of discussion within a health plan, whenever we’re discussing treatment A versus treatment B, especially at the P&T discussions, is, what does this treatment do to maximize the clinical outcome in terms of patient-centric outcomes? What does it do to potentially avoid unnecessary utilization, and hopefully avoid utilization that would occur if the member was not seeing improved clinical outcomes? Because, hopefully, if the individual is doing better, he or she will not need to be hospitalized, end up in the emergency room, or have other invasive or potentially risky therapies being used. So, understanding the offsets, understanding the ability of one drug, one treatment to lead the patient down a better clinical path, if you will, is always extremely important.

We love to see data from articles from the literature that spell out the likelihood or potential for avoiding things like hospitalizations. But, in PAH, our major source to avoid is not only cost, but also, I would say, risks to the patient. Being in the hospital is a challenging time. Hospitals are dangerous places. And the more we can keep people out of the hospital, because they’re performing better clinically and doing better in terms of their treating their PAH, I think that’s going to be a positive thing not only for the patient really, but for the entire care system. So, understanding that data is really important.

I would tell you that part of the challenge that we have at the health plan is that there are frequently very little data that tells us the potential for avoiding things like hospitalizations. If I had one request to make of the researchers, it would be to include those kind of metrics as you go forward. Not only from a cost standpoint, but because if you identify therapies that do a better job of avoiding the need for these types of interventions, that means that in fact the patients are doing better.

As we get more and more data about how to treat conditions like PAH, and as the entire medical system learns more and has more experience in this, one of the things that has been very clear is there is an important role for combination therapy. And we’re seeing that in many clinical conditions, not just PAH. Especially, we’re seeing it more and more nowadays in oncology. I think one of the things that we are always focused on from the managed care side is, are the treatments that are being requested being appropriately targeted? In other words, are we really seeing requests for these types of expensive, and I would also say potentially dangerous therapies, because they have the potential for adverse events and there are certainly very important safety and tolerability concerns with a lot of these drugs? Are they being leveraged for the right patients? And so, our process is to make sure that there’s alignment between the recommendations from the literature, from the FDA, with the request we see.

I absolutely expect to see more combination therapy as we go forward over the next few years as we learn more, as more drugs come to market, and as we have some drugs that are actually going off patent and becoming generic. So, ideally the total cost of care would be lowered. At the end of the day, what we want is the best therapy for the patient. We want the one therapy that is going to lead to the best clinical outcome—and ideally do so at the optimal level of safety and tolerability—and hopefully at a cost that is acceptable.