The FDA has approved gene therapy treatments for Sickle Cell Disease. Previously, treatment was performed via marrow transplants which require a donor and have a risk of rejection along with the challenge of finding suitable/matching donors.

As reported, currently gene therapy options are $2.2mil USD, not including the cost of care including hospital stays and chemotherapy.

Also reported (same article linked above): The clinical trial included 46 people in the U.S. and abroad, 30 of whom had at least 18 months of follow-up care after the treatment. Of those, the treatment has been successful in 29.

Another outlet reports: Bluebird said Friday that it’s in “advanced discussions” with the largest commercial health insurance providers in the US, as well as more than 15 Medicaid agencies, representing 80% of people with sickle cell in the country. The company noted that it has designed “outcomes-based” contract options for insurers that tie payment for the therapy to how well it works over time.

The drug will start to be available early next year, bluebird said.

I'm happy to receive feedback on how to define financial and practical accessibility and update resolution criteria for clarity. I will continue to research to establish measurable guidelines and update the market accordingly.

My inclination is to resolve YES when 1,500 treatments have been successfully performed (regardless of successful outcome/cure) and I presume this treatment will be followed and reported on with a lot of interest.