Because gene therapies are used to treat diseases affecting small numbers of patients, “they are approved by the [European Medicines Agency (EMA)] and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost,” investigators write.
“As of January 1, 2022, the FDA and EMA authorized 8 and 10 gene therapies, respectively,” the authors report based on available regulatory information. “The FDA and EMA granted orphan designation to all gene therapies except talimogene laherparepvec. Pivotal clinical trials were nonrandomized, open level, uncontrolled, phase I–III, and included a limited number of patients. Study primary outcomes were mainly surrogate endpoints without demonstration of direct patient benefit. The price of gene therapies at market entry ranged from $200,064 to $2,125,000.”