Gene editing and therapy to replace missing or defective genes is one of the most exciting recent medical developments, offering tremendous hope for people with rare diseases and genetic conditions. With so little known about the long-term risks of gene therapy, we contend that the safest and best value to both patients and society will emerge within a framework of early evidence-development strategies following early coverage decisions.
The Food and Drug Administration has approved several gene therapies for rare diseases through the accelerated pathway. The high costs of treatment, however, continue to be a barrier to access for patients, as described by Sen. Bill Cassidy (R-La.) in his recent request for information on behalf of the Senate’s Committee on Health, Education, Labor and Pensions (HELP).
Now in their early days, gene therapies represent an evidence problem. Because they are just beginning to be used, little long-term data exists on their effectiveness and downstream risks, including unforeseen malignancies and infertility.