Congress introduced legislation this month allowing seriously ill patients to access select treatments their physicians would prescribe – without FDA approval. That would mark a fundamental departure from the FDA’s total control over every prescription drug in America. That stranglehold is the efficacy mandate – the FDA’s requirement to certify that drugs work before any patient can access them, a federal override of the physician-patient relationship. Six center directors, two commissioners, thousands of departures since January 2025 – yet the FDA’s fundamental problem is not its leadership. The problem is its power.
Safety oversight – ensuring that drugs are not harmful before they reach patients – seems like a valid government role. But the case for the FDA’s drug efficacy authority rests on a myth. Thalidomide caused more than 10,000 birth defects in Europe and was blocked from the U.S. market in 1961 using the FDA’s decades-old safety standard. That tragedy generated public fear, and politicians reacted by adding a new FDA function – certifying that drugs work, as if efficacy was the problem rather than safety – with the Kefauver-Harris Drug Amendments in 1962. For the first time, no drug could reach a patient without explicit FDA approval of its effectiveness.
FDA approval is the gateway to market, but the meaningful efficacy determination – how the drug actually performs in patients, for the conditions that matter – belongs to physicians, not the FDA. Between 20% and 40% of all prescriptions, and more than half in oncology, pediatrics, and several other specialties, are written “off-label,” that is, for a use other than one the FDA approved, purely on physician judgment. Gabapentin, one of the 10 most prescribed drugs in America, is written for neuropathic pain, anxiety, and insomnia 83% of the time – and that’s off-label. Metformin, approved only for type 2 diabetes, is prescribed off-label for polycystic ovaries, prediabetes, and cancer prevention on evidence the FDA has never reviewed. The FDA requirement substitutes a federal agency’s judgment for physician-patient choices.
Waiting for FDA efficacy approval has real costs to patients, but the people who didn’t get the drug may not appear in statistics. Levamisole was proven to cut colon cancer mortality by 33%, but patients waited two years for the FDA to certify it for human use. For rare childhood diseases the mandate is worse: Populations too small for trials mean most rare diseases cannot meet the approval standard. That Congress needed legislation last week just to open a narrow exception proves the efficacy authority is the problem.
The economic toll is just as damning. University of Chicago economist Sam Peltzman alerted us decades ago: “FDA’s proof of efficacy requirement was a public health disaster, promoting much more sickness and death than it prevented.” Tomas Philipson, former acting chairman of the Council of Economic Advisers, estimated a single year’s reduction in the FDA’s effectiveness-review timeline would generate more than $10 trillion – because the FDA has spent 60 years regulating something it was never meant to regulate.
This overreach also produces corruption and industry capture. By 2025, the pharmaceutical industry funded 77% of the FDA’s drug review program with fees; consequently the regulated industry sets performance standards for its own regulation. Scott Gottlieb joined Pfizer’s board three months after leaving the FDA. Stephen Hahn joined Moderna’s founding venture firm five months after authorizing Moderna’s vaccine. Patrizia Cavazzoni ran the FDA’s drug center, left in January 2025, and was back at Pfizer as chief medical officer five weeks later. Curtis Wright approved OxyContin and joined Purdue Pharma a year later. Leadership may be temporary, but the revolving door is permanent.
Still, the obvious objections to paring back FDA authority deserve answers. If not the FDA, who would evaluate whether drugs work? The same institutions that already govern clinical practice: the Cochrane Collaboration, hospital formulary committees, state Medicaid effectiveness programs. These mechanisms are not a replacement for the FDA – they are already operating alongside it, and doing something the FDA does not: comparing drugs against existing alternatives. Who stops false claims? The False Claims Act ($6.8 billion recovered from the health care industry in fiscal year 2025 alone), FTC enforcement, product liability, and state consumer protection law.
A review apparatus so large and so focused on certifying efficacy has diverted the FDA’s institutional attention from its core food safety mission. An independent evaluation commissioned by the FDA in 2022 documented the result: a culture of indecisiveness and inaction in the food program. More than half of foreign supplier inspections find importers non-compliant with U.S. safety requirements.
Every new FDA director inherits the same broken authority. That authority has to change. Patients are being denied drugs their physicians would prescribe, drugs that the FDA has blocked or may never approve. Repeal the efficacy authority – it would reduce the corruption, and restore to free citizens the right to decide their own treatment. Let the FDA do what it was built to do: Keep unsafe drugs off the market and unsafe food out of the supply chain.
Everything else belongs to physicians, patients, and citizens. Not to Washington.
