Dr Steven Pearson Highlights the Challenge of Evaluating New Interventions That Cure Diseases

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In order to start evaluating the economics of new cures, the scope of the evaluation needs to be as broad as possible and be able to wrestle with uncertainty, said Steven Pearson, MD, MSc, founder and president of the Institute for Clinical and Economic Review.

Transcript

How can or should the standard economic evaluation be altered to better evaluate interventions that cure diseases?

When you’re trying to evaluate cures, the first thing is to try to figure out how you’re going to handle uncertainty. Obviously, you can run different economic models with different assumptions about the long-term durability of an effect, but you really have to look deeply into the biomechanics, if you will, of the innovation: how it works, what’s known about the relative chances that it is a true cure, even with short-term data. So, I think the evaluation has to try to take on board a greater focus on how to factor in the uncertainty in the ways that we think about value.

Otherwise, there are challenges in understanding the broader societal effects of cures, because they may extend not just to the patient and the patient’s family, but it could be that the entire infrastructure of care would shift if we have a new treatment and it’s a cure that might lead to changes in screening or the way that even physicians are trained to look for different kinds of diseases, and, if there’s a treatment, refer them on to people who can treat them.

So, I think the evaluation scope just needs to be as broad as possible and wrestle with uncertainty in ways that we don’t usually have to worry about quite as much with many other new treatments.

Do you see any changes in societal willingness to pay for therapies coming to market that are increasingly more expensive and are starting to approach the million-dollar price tag?

I don’t think there’s anything magical about $1 million. There will be, certainly, in the headlines that are generated. No one is going to want to be the first million-dollar treatment. And, yet, what I’m sensing—of course, I hear people from all different walks of life—some people are very worried about the cumulative effects of trying to pay for not just cures but a lot of other treatments for ultrarare diseases.

There’s a sense that even though they are very clinically valuable, that our old assumptions about being able to shoulder very high costs, is running into a real cumulative affordability problem and that we don’t have an easy way out of this given that we do want to continue to incentivize important new treatments.

So, there’s a mixture of excitement and anticipation, and real concern that these costs are going to somehow going to have to be factored in, in some way, through the health insurance system in a way that can remain affordable.