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Sickle Cell Gene Therapy Reimbursement: Will Experience Matter? And Which Kind?

Executive Summary

The tandem launches of two gene therapies for sickle cell disease will likely be critical to determining whether and how US reimbursement policies can shape commercial success for hugely expensive, one-time treatments. Bluebird is the smaller, less-experienced company up against Vertex – but argues it has more relevant experience.

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Bluebird’s Fast Lyfgenia Launch Was A Year And A Half In The Making; Medicaid Talks Continuing

When Zynteglo was approved for beta-thalassemia, bluebird said launch would lay groundwork for sickle cell gene therapy Lyfgenia. The strategy is paying off so far with a fast ramp-up. Outcomes-based contracts for Lyfgenia give payers rebate based on hospitalizations over a three-year period.

Bluebird’s Lost Voucher: Lyfgenia Contains Same ‘Active Ingredient’ As Zynteglo, US FDA Says

Surprise denial of rare pediatric disease priority review voucher could prompt calls for the agency to re-examine how it interprets the term ‘active ingredient’ for gene therapies. Bluebird asserts there are significant differences between its gene therapies for treating sickle cell and beta-thalassemia.

Bluebird, Vertex Gene Therapies May Answer $1m Question: Can Competition Reduce Rx Prices?

Medicaid is expected to be the primary payer for the sickle cell treatments. Bluebird bio maintains outcomes-based arrangements are a key part of its access strategy and will overcome what appears to be a significant competitive disadvantage on price compared to the Vertex product.

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