Walgreens’ Ray Tancredi Weighs in on Expensive Hemophilia Gene Therapies in the Pipeline

Gene therapies in the pipeline to treat hemophilia will be very expensive, but they also seem to be very efficacious, and multiple could hit the market between now and 2024, said Ray Tancredi, RPh, MBA, CSP, divisional vice president, specialty pharmacy development and brand Rx/vaccine purchasing, Walgreens.

Transcript

There are a few gene therapies for hemophilia in the pipeline. What will it mean for the health care system if more than one of these expensive therapies is approved in the next 1 to 2 years?

Yeah, that's an interesting thought. When you think of the cell and gene products, you know, I saw 4 therapies for hemophilia A in the pipeline and 2 therapies for hemophilia B. They all have the potential to come to market between now and 2024. All of these products have similar labels upon approval—use limited to moderate to severe disease—and they're really comparable in safety and efficacy. And not only that, these products will really compete with drugs that are already entrenched in the marketplace, like Hemlibra, which is a long-acting factor replacement product.

So, this may create a market where the payers have to create formularies, maybe even have different patient evaluations for prior authorization. And the primary drivers will be: How do the manufacturer and the payers work together to use these products and ensure that that they're effective? I saw some stats in the New England Journal of Medicine; there was an 84% decrease in bleeds and a 99% decrease in factor use for BioMarin's hemophilia product.

So, I think they're efficacious. I don't know exactly what the costs will be, but you know they will be very expensive, and pharma and the payers and plan sponsors are all going to have to look at these therapies, evaluate their efficacy and the costs, and determine the best patients for these products to be used in.

But very exciting. When you think about hemophilia as a disease state, these products could change patients’ lives. So, it's very, very exciting, disruptive to the market, and it'll be great for patients to maybe reduce their use of factor products.