Dr Neil Minkoff on Hopes, Payer Reactions If a Gene Therapy Is Approved for Sickle Cell Disease

Neil Minkoff, MD, chief medical officer and vice president, EmpiraMed, discusses his hopes if the first gene therapy for sickle cell disease is approved and if he has any concerns with payer reactions.

Transcript

What are your hopes if the first gene therapy for sickle cell disease is approved?

Well obviously, the hope is highly effective medication. So, what we’re really looking for is something that is as close to curative as possible with a very long tail, which is to say, you do it once, you really are getting benefit—it’s for 5 years, 10 years, 15 years—because that’s the big open question: What is the length of the effectiveness of any of these therapies? But the hope is that you’ll have something that can be administered once and will provide at least a decade or more of effectiveness.

Do you have any concerns about payer reactions if gene therapy for sickle cell arrives?

I have concerns about payer reactions, as a former payer myself, across all the different types of gene therapy because we’re entering unchartered waters, and we don’t know exactly how we’re going to amortize the cost of very expensive therapies. The therapies that have been approved to date are all 7 figure therapies, and we don’t necessarily have a great mechanism right now of tracking patients over time to see how much of that benefit is accruing and to whom it should accrue. So, we need to figure out as payers different ways to accrue or escrow or find ways to get third parties to manage that interaction.