Gene therapy, a revolutionary medical breakthrough, offers hope to individuals with rare diseases and genetic conditions. However, its widespread implementation is hindered by the limited understanding of its long-term risks. To strike a delicate balance between innovation and safety, we advocate for an evidence-based approach. This involves developing robust early evidence and implementing cautious early coverage decisions.
The Food and Drug Administration (FDA) has granted approval to a handful of gene therapies targeting rare diseases. Nevertheless, access remains a significant barrier for patients, as highlighted by Senator Bill Cassidy (R-La.) in his address on behalf of the Senate’s Committee on Health, Education, Labor, and Pensions (HELP).
Gene therapies, being relatively novel, pose an evidence dilemma. The scarcity of long-term data on their efficacy and downstream risks, encompassing unforeseen malignancies and infertility, necessitates a measured approach. As we navigate this uncharted territory, it is imperative to prioritize patient safety while fostering ongoing research to expand our knowledge base.
