On June 2, 2026 in The Wall Street Journal, Charles L. Hooper and David R. Henderson wrote “…Remove efficacy from the FDA’s approval process and focus on safety alone. That would improve doctors’ ability to match patients with the best drugs and help Americans live longer, healthier lives. Medicine is a matchmaking exercise in which potentially beneficial drugs are tested patient by patient, using trial and error” (here, subscription required). Well, that seems to be a big step back to what was in place prior to 1961; the efficacy requirement was added to the Federal Food Drug and Cosmetic (FD&C) Act by the Kefauver–Harris Amendment in 1962… and that amendment came about for a reason, on the heels of the thalidomide tragedy in Europe.
Hey, wait a hot minute! There is already a provision in the Right to Try Act that permits the use of an experimental drug for terminally ill patients or patients suffering from a condition for which there is no approved treatment (see the Right to Try fact sheet here). There are also expanded access programs for as-yet unapproved drugs in clinical trials. In addition, there is an accelerated approval process that the FDA uses for drug products for which there is a reasonable surrogate endpoint. However, under the accelerated program, there have been at least thirty-two drug products or indications for drug products to treat cancer that were withdrawn when efficacy of the products failed to be demonstrated from required post-approval confirmatory clinical studies (see here).
In addition, there are other classes of drugs (for example, Biogen and Eisai’s Aduhelm for the treatment of Alzheimer’s disease) that have been withdrawn from the market for several reasons; the 17-alpha hydroxyprogesterone drug Makena by Amag Pharmaceuticals for pre-term birth was withdrawn in April 2023 after it failed to demonstrate efficacy in confirmatory clinical trials. Because many drugs approved under the accelerated approval process fail confirmatory efficacy trials post approval, questions have been raised as to the benefit of continuing the accelerated approval process. However, this FDA process is in place, and there does not seem to be an FDA appetite to do away with it, so it looks to be a continuing option for the FDA to approve products prior to full confirmatory demonstration of efficacy based on a surrogate endpoint.
There are pros and cons to the argument for the accelerated approval process. Obviously, the biggest pro is that it can bring a promising product to market much more quickly. The major con is that people may be paying a lot of money for a product that ultimately is shown to be ineffective. However, at least under the accelerated approval process, there is some evidence of potential efficacy based on the selection of a reasonable surrogate endpoint upon which to initially base the approval.
To permit the introduction of products that are approved only for safety would take medicine back to before the 1960s, prior to the FDA’s introduction of the efficacy requirement. In this time of significant concern regarding the cost of drugs, relying on safety assessment alone seems like a fool’s errand. Not only could patients be spending thousands, hundreds of thousands, or even (in the case of certain therapies) over a million dollars for a treatment without an evaluation of the products efficacy, they could be wasting their time with an ineffective drug while not taking advantage of other therapies that might help mitigate their disease. There is also the consideration of the uncertainty of insurance coverage for products approved only for safety. Insurance giants and Pharmacy Benefit Managers (PBMs) would likely balk at payment for a drug that the FDA does not know will be efficacious.
Let’s hope that this safety-only concept dies on the vine, and let’s continue to rely on the science that provides the greatest assurance of both safety and efficacy of the drug products that the FDA approves. There are, in my opinion, sufficient options in the current drug review and approval scheme that provide flexibility, such as Right to Try as well as the accelerated approval process. In addition, in the practice of medicine, healthcare professionals often use drug products, which are FDA-approved for safety and efficacy, for off-label treatment.
Let’s hope that we stay within the current bounds of products being approved for both safety and efficacy to best protect patients and so we don’t get to the point of having to tell a patient “There’s some good news and some bad news—the drug you’re taking is safe, but it’s doing nothing to treat you. Oh, and about all that money you just spent, well, I guess that’s just too bad.”
