With two new expensive gene therapy approvals, outcomes-based pricing deals grab the spotlight again
Two recent approvals for two expensive gene therapies for blood disorders — BioMarin’s $1.5 million Roctavian in the EU for hemophilia A and bluebird bio’s $2.8 million Zynteglo in the US for transfusion-dependent thalassemia — is shining a spotlight on outcomes-based pricing deals as both companies look to leverage the strong efficacy and durability of their therapies.
In the case of BioMarin’s Roctavian, execs said in an investor call this week that its outcomes-based agreements with EU member states will differ market by market but provide refunds when someone doesn’t respond to therapy. As only 6 of 134 patients resumed standard of care in the late-stage trial, Jeff Ajer, EVP and chief commercial officer of BioMarin said in an investor call that they’re “very enthusiastic” about such high responder rates.
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