GlaxoSmithKline ($GSK) has struck a Strimvelis deal in Europe. The gene therapy for “bubble boy” disease, only the second of its kind, raised questions about pricing and payment in the up-and-coming field--and GSK’s arrangement offers some potential answers.
According to MIT Technology Review, GSK will offer the one-time treatment with a money-back guarantee. Priced at 594,000 euros ($665,000), it’s among the most expensive therapies in the world. But it’s also a cure for severe immunodeficiency stemming from a lack of adenosine deaminase (ADA-SCID), rather than an ongoing treatment as other rare disease drugs are.
“The drug has to deliver what you say or we don’t pay,” Luca Pani, director general of the Italian Medicines Agency, told the publication. “If it does not work, they will return the money.”
Strimvelis won approval in Europe earlier this year, and at the time, GSK said it would price the drug “significantly less” than the $1 million-plus tag assigned to the world’s first gene therapy, Glybera.
GSK did not respond to FiercePharma’s request for comment before press time, but when Strimvelis was approved, spokeswoman Fiona McMillan told us that the company was considering “different models” on pricing. Those models could include staggered payments--which are part of the Italian deal now--and outcomes-based arrangements, she said.
“[I]f a patient needs to go back onto a different therapy down the line, or if their health declines, we will look at refunding some of the cost,” McMillan said.
The drug will be distributed only in Milan, Italy, which means families have to travel for treatment, and the Italian health authorities’ arrangement with GSK covers all European patients, MIT Tech Review says. Strimvelis was developed in partnership with Italy’s San Raffaele Telethon Institute for Gene Therapy.
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