Spinal muscular atrophy is giving payers a new conundrum, and the indication is poised to become a testing ground with implications for many if not all gene therapies.
The issue is whether, and how, payers will reimburse for both a one-time gene therapy and a chronic therapy for the same setting.
The problem is the lack of long-term data at launch for gene therapies that demonstrates whether or not they provide a cure. The uncertainty means clinicians and patients might seek to add a chronic therapy if a gene therapy isn’t showing results fast enough, to improve the odds of achieving a functional cure. Conversely, they might add a gene therapy to patients already on a chronic therapy for the same reason.
How payers handle reimbursement for follow-on treatment after gene therapy could set the stage for other diseases, with hemophilia or β thalassemia the next settings likely to be confronted with the issue.
The cost paradigm is complex. The most recent market entrant, approved by FDA on May 24, is gene therapy Zolgensma onasemnogene abeparvovec-xioi from the AveXis unit of Novartis AG. The drug