Paul Melmeyer on Willingness to Pay for High Cost of Rare Disease Therapies Among Stakeholders

There is generally a greater willingness to pay among just about everybody for rare disease therapies, explained Paul Melmeyer, director of Federal Policy, National Organization for Rare Disorders.

Transcript

Have you noticed a greater willingness to pay for high cost of rare disease therapies in recent years?

There is generally a greater willingness to pay in just about everybody for rare disease therapies. There’s a greater willingness to pay we see within the actual HTA [health technology assessment] bodies in that they generally have higher cost per QALY [quality-adjusted life year] thresholds allowing for there to be a higher threshold within rare diseases because there’s an acknowledgement that rare disease can be that much more valuable. Within insurers, we’ll see insurers be more willing to pay for a therapy that is that much for expensive fort a rare disease because, again, perhaps they recognize that to them there’s also a greater willingness to pay.

Also, society in general, as well. We see empirical studies that show that societies are generally much more willing to pay for rare disease therapies, especially in the context of pediatric rare diseases and the context of rare diseases that otherwise don’t have any other therapy, that the greater willingness to pay within society also contributes to the greater willingness to pay within these specific bodies.