Welcome to the Age of One-Shot Miracle Cures That Can Cost Millions

June 6, 2019

(Bloomberg) – This is the tantalizing promise of gene therapies, the potential cures for dozens of once-incurable illnesses. The U.S. Food and Drug Administration issued its first approval of a systemic gene therapy, a Novartis AG treatment for spinal muscular atrophy, on May 24 and says it expects to approve 10 to 20 therapies a year starting in 2025. There are more than 800 trials under way, targeting diseases including rare metabolic disorders, sickle cell anemia, hemophilia, and Parkinson’s. As the list grows, such treatments have the potential to fundamentally remake the health-care system at every level. There are two big caveats. First, most studies haven’t run longer than a few years, so it’s impossible to know yet whether the therapies will remain effective for life, help everyone the same, or yield side effects decades in the future.