Cleared for Market but Not Proven to Work

Each day, people receive diagnoses of serious illnesses. Often, their doctors prescribe new medications that have just received FDA approval. We assume these drugs have been proven safe and effective.

Well, not so fast.

In an article in the online publication Lever, investigative journalists Jeannie Lenzer and Shannon Brownlee say that the FDA has weakened standards first developed in 1962. Back then, drug companies had to prove with studies that a medication was safe and that it helped people feel better or live longer. But studies take time, so Lenzer told me that companies began seeking shortcuts to bring products to market faster.

“The majority of drugs now reach the market without evidence that they actually work, with the promise that drugmakers will demonstrate effectiveness after approval,” Lenzer says. “That’s a complete reversal of what used to be the case.”

How did we reach this point? Partly through a process called “accelerated approval” — a pathway designed to get drugs to patients faster. But that speed often sacrifices scientific rigor.

Drugs now hit the market not because they’re proven to save lives or improve quality of life, but because they might. Some gain approval after trials lasting only weeks. Others are tested only against sugar pills — placebos — not against the best existing treatments. Sometimes the results are so unclear that experts can’t determine whether the drug causes more harm than good.

And what happens to requested follow-up studies meant to confirm benefits? They’re frequently delayed for years, or they’re never completed.

Consider Elmiron, a drug for bladder pain. It was approved in 1996 without randomized trials until 2015. Then, that study showed it was no better than a placebo. And it’s much easier to get a drug approved than to get it removed. Elmiron remains on the market despite growing evidence linking it to vision damage, eye disease, and colon problems.

“It’s not only still available,” Lenzer notes, “the American Urological Association calls it the only FDA-approved drug for interstitial cystitis. People continue taking it, unaware it could lead to blindness or colitis.”

Another example is Aduhelm, the controversial Alzheimer’s drug approved despite lacking convincing evidence that it slows cognitive decline.

So, what can we do?

Start by asking questions of your health care provider. Ask if the medication you’re being prescribed is new. Has it been tested in large, randomized trials? Has it been shown to improve meaningful person-related outcomes like survival or quality of life?

I should say that, as a rule, I avoid prescribing any new medications for several years until we understand their real risks and benefits.

The challenge is that doctors are often too busy, too trusting of the system, or in denial about how much they are influenced by drug company marketing to know these answers themselves. That’s why public awareness matters. We need watchdogs — not lapdogs — in and out of the FDA. We need transparency. And we need a public that demands more than hype, hope, and headlines.

– Dr. Michael Wilkes with a Second Opinion