Pricing gene therapies

The launch of the first gene therapy, Glybera alipogene tiparvovec from uniQure N.V. and partner Chiesi Farmaceutici S.p.A., provides at least one lesson for companies thinking about annuity pricing models: disease outcomes must be simple enough to measure that the model isn't too onerous to implement. Glybera did not tick that box, but uniQure thinks its next-in-line gene therapy for hemophilia will, and therefore could be a good test case for an annuity pricing model.

Glybera is approved in Europe to treat lipoprotein lipase (LPL) deficiency in patients with recurring, acute pancreatitis. On Nov. 26, Chiesi filed a pricing dossier with Germany's Federal Joint Committee (G-BA). Chiesi set an ex-factory price of €43,870 ($54,587) per vial, or about €877,400 ($1.1 million) for a 60 kg patient, who would require 20 vials. The ex-factory price excludes VAT and pharmacy margins, since Glybera will not be distributed through retail pharmacies...