Bluebird Bio defends as ‘simple and human’ its pricing of costly gene therapy for beta thalassemia

[J]ust as we spent the past years cracking the code to make gene therapy work, we now need to work just as hard to make sure that gene therapy can be accepted, and paid for, by our health care system.

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The Bluebird [Bio] approach is simple and human:

Calculate a fair value. We offer a lifetime cost-effectiveness approach using well-accepted modeling methodologies that have been pressure tested with external advisors to determine a value based only on direct patient benefit. In the case of Zynteglo for beta thalassemia, this means living longer with a better quality of life than could be expected with existing treatments.

Share risk. After an initial payment, annual milestone payments are made only if the treatment works. That means the company has skin in the game, with up to 80% of the potential total lifetime cost at risk.

Cap the total lifetime cost per patient at five years. Gene therapy might deliver a lifetime of benefit, but we don’t deserve a lifetime of payment for it. We get paid for the first five years if it works and, after that, the savings from costs avoided accrue to the health care system and the patient.

Read full, original post: In pricing our gene therapy, Bluebird weighed value, shared risk, and a lifetime cap