Bluebird’s Zynteglo offers yet another way to pay for gene therapy

Gene therapy companies are certainly becoming more inventive in selling their products' prices to payers. The market now has three recently approved gene therapies, and each has shown how ideas around pricing are evolving. Bluebird Bio was the latest company to come out an attempt to make the thought of paying almost $2m palatable to payers. Its beta-thalassaemia treatment, Zynteglo, was approved in Europe earlier this month, and on Friday Bluebird revealed a staggered pay-for-performance vehicle that appears to put other pricing models in the shade. The group is proposing a one-time €315,000 for the product, and will only receive additional yearly payments of €315,000 if the treatment continues to be effective over five years – essentially putting 80% of the list price on the line if patients do not continue to benefit. In contrast, Novartis's model for Zolgensma is pay-over-time, rather than Bluebird's pay-for-performance idea. So far few have denied the merits of gene therapies developed for rare inherited diseases with few options. The rubber will start to hit the road with the next generation of products, including those for haemophilia, where there are larger populations and, as Biomarin has demonstrated, questions around efficacy duration.