by Craig Klugman, Ph.D.
In a surprising move, the Food and Drug Administration approved Exondys, a drug that has not been proven to work, did not have a randomized control trial, and that recommended against by an independent expert panel. The approval was via the accelerated pathway “which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatment. Approval under this pathway can be based on adequate and well-controlled studies showing the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients.” You just need to show a likelihood of benefit, not proof that it works.
The drug targets a genetic mutation that is the cause of 13% of Duchenne muscular dystrophy (DMD) cases. The disease is rare, affecting approximately 20,000 boys in the U.S., aged 5 – 24 years. Most patients die in the 20s or 30s.
Approval was based on a study of 12 boys with the disease who took the drug. There was no control group. The expert advisory board voted against approving the drug, citing a lack of meeting FDA study requirements such as sample size and controls. Usually, the FDA follows the recommendation of the advisory boards.
In this case, approval went against the advisory board recommendation because of pressure from patient and advocacy groups. There simply are not any other good treatments for the disease. The FDA is requiring a future confirmatory trial, after approval. Proof after approval is the opposite of how the FDA usually works.
The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.