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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