Sometimes the simplest solution is the best one.
The Food and Drug Administration (FDA) is notoriously slow at approving new drugs, even ones that have been on the market for years in other countries.
This bureaucratic slowness comes at a high cost: billions of dollars wasted and millions of disease sufferers denied access to life-saving therapies.
How to accelerate the FDA approval process is a longstanding debate. Many sensible, incremental reform proposals have been suggested.
But the best of these proposals is also the simplest: Instead of requiring FDA to confirm that a drug is both safe and effective (the current policy), just change FDA’s mission to focus on safety alone.
The Problem
That’s exactly what FDA did from 1938 to 1962. It focused on safety.
Congress added the requirement that drug manufacturers also prove their products are effective as a result of the thalidomide scandal of the late ‘50s and early ‘60s. Thalidomide had been approved in Europe as a sleep aid, and later as a morning sickness treatment, but the U.S. FDA refused to approve it, citing evidence it caused birth defects.
The agency acted properly. But the scare led to the enactment of the 1962 Kefauver-Harris Drug Amendments, which expanded FDA’s role to require proof of efficacy before a drug can be approved for marketing. This reform was ironic, since thalidomide is effective, it just isn’t safe (for pregnant women).
In fact, every major drug disaster in American history has been a failure of safety, not of efficacy.
And as David Henderson and Charles Hooper point out in a recent Wall Street Journal op-ed, “Lack of safety kills. Lack of efficacy [merely] requires trying a different drug.”
Going back to an exclusive focus on safety would dramatically streamline the drug approval process, speed up medical innovation, and save lives.
To establish efficacy, FDA requires drugmakers to conduct large human clinical trials involving hundreds or thousands of individuals. These trials are slow and expensive. And they’re never truly successful. No drug works for everyone. Most drugs work better in some people than in others. Whether a drug works for a particular medical condition in a particular individual can only be verified by letting that individual try it.
In other words, efficacy can only be established through trial and error. The FDA can never fully answer the question; only patients and doctors can do that. And yet the agency spends most of its time and budget trying to answer it, using a blunt instrument (the large-n clinical trial) that needlessly duplicates what must happen post-approval anyway.
Physicians often prescribe drugs “off-label,” meaning for medical conditions not listed on the FDA-approved drug packaging. This is perfectly legal. And it makes perfect sense. Frequently, a drug approved by FDA for one medical condition turns out to work well for a completely different condition that was never considered during the clinical trials. Today, 20 to 40 percent of all U.S. prescriptions are written this way, including more than half of all prescriptions in cancer care and pediatrics. All those millions of experiments are based only on the treating physician’s judgment and clinical evidence.
This is a classic example of a bottom-up discovery process, one that fuels progress throughout society by generating knowledge, innovation, and opportunity, without any need for government permission or top down, centralized control.
The High Cost of the Status Quo
The costs of the efficacy mandate are huge. In 1973, economist Sam Peltzman studied the effects of the 1962 reform. New drug approvals, he found, fell 61 percent below where they would have landed otherwise, with no corresponding gain in either safety or efficacy. His blunt conclusion: The efficacy mandate was “a public health disaster, promoting much more sickness and death than it prevented.”
Why did the 1962 reform fail? Because it tried to address the wrong problem with the wrong tool. It added a costly bureaucratic hurdle that needlessly slowed therapeutic discovery and prolonged human suffering, without any offsetting benefit. Peltzman was right to call it a disaster.
Yet FDA plows on. To take just the latest example, earlier this year the agency rejected RP1, a melanoma drug by Replimmune, not because it was dangerous — it isn’t — but because regulators weren’t convinced it worked well enough.
Sadly, the human cost of this excessive caution is often invisible. As Scott Atlas, M.D., points out at RealClearPolitics:
“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.”
And FDA doesn’t always get it right, and doesn’t always act consistently. For example, it had the evidence for nearly two decades that orally administered phenylephrine, an approved cough and cold medicine, doesn’t work. (As a nasal spray, it does work.) An advisory panel raised doubts in 2007; a second panel in 2023, sixteen years later, called the pill form no better than placebo. Yet the agency did not act on the information until 2025, when it formally proposed to pull the drug’s approval. (It has not yet done so.) Ideally, the agency would just amend the “Drug Facts” information on the package to let consumers know that there is no evidence the product works in pill form.
And then there’s the mandate’s cost in dollars and cents. It’s huge.
While only one-fourth of drug development costs go toward proving safety, a staggering three-fourths go toward proving efficacy — a cost that shows up in every drug on the shelf.
Tufts researchers put the fully loaded cost of bringing one new drug to market at $2.6 billion. The approval process for a new drug takes a decade or more. If we removed the efficacy gauntlet, those figures would presumably fall by 75 percent. More companies could afford to enter the market and compete. Progress would accelerate, and prices would decline. Doctors would have more tools, and disease sufferers would have more options, and more hope.
As Henderson and Hooper conclude, “The cost of the FDA’s efficacy mandate is tremendous, and the value is close to zero.”
Would we be opening the floodgates to quack medicines? No. There’s a robust infrastructure in place to prevent and punish false marketing claims. Manufacturers and marketers are liable to fines and lawsuits.
What happens if an approved drug turns out to be less safe than FDA originally thought? There’s a system in place for that, too. Drugmakers are required to notify the agency about unexpected adverse drug events in real time, and physicians are encouraged to do so. When a serious safety issue arises post-approval, FDA can simply pull the drug off the market.
Why Not “Safe to Try”?
The good news is lawmakers are waking up. In 2018, Congress enacted the Right to Try Act, an exception to the mandate that allows terminally ill patients to experiment with non-FDA-approved therapies under physician supervision. As Dr. Atlas notes, “That Congress needed legislation just to open a narrow exception proves the efficacy authority is the problem.”
A few states have unilaterally taken steps to widen the exception:
- Seventeen states have enacted a “Right to Try 2.0” law that lets a patient try a non-approved drug solely tailored to that individual — a drug for which a clinical trial is pointless.
- Montana has entirely removed the “terminal illness” requirement, allowing any patient with informed consent to seek treatments that have only completed FDA phase I safety trials. Call it “Safe to Try.”
Because these state laws conflict with federal policy, they exist in a legal gray area. But their very existence suggests the federal Right to Try Act is too narrow.
Why not take this movement to its logical conclusion? Why not make the Montana approach national? Why not “Safe to Try”?
FDA would remain firmly in charge of safety, but it would get out of the business of deciding, drug by drug, which drugs Americans are allowed to try.
The benefits would be tremendous:
- Faster cures
- More tools for physicians and more hope for sufferers
- Longer, healthier lives
- Lower drug prices
- Lower government spending and smaller deficits
- A healthier workforce, driving a stronger and more competitive economy
In short, Safe to Try would bring greater health, happiness, and prosperity to all Americans, with no downsides.
Sometimes the simplest solution is the best one.
Dean Clancy is senior health policy fellow, and Sofia Hamilton is senior health policy analyst, at Americans for Prosperity.
