The FDA Approved Hundreds of Drugs Without Proof They Work

• The FDA relied on four essential standards to assess a drug’s effectiveness and safety — According to the authors of the report, while these criteria don’t confirm “sound scientific evidence,” they do provide the minimum standard that determines drug manufacturers have given “substantial” evidence to back up their claims. These include:

However, after an intensive analysis, the reporters found that the majority of the FDA-approved medications failed to meet these basic criteria.

• Nearly 3 out of 4 new drug approvals did not comply with these criteria — Among all the drugs approved by the FDA between 2013 and 2022, 73% failed to meet these foundational standards that prove the drugs work as expected.⁴ “Fifty-five of the approved drugs met only one of those four standards, and 39 met none of them,” the CHD notes.

• Drugs were green-lit despite insufficient data presented by manufacturers — The Lever found that more than half of approvals were based on preliminary data — without considering sound evidence, such as if the patient experienced fewer symptoms, had improved function, or lived.

• Particularly concerning are cancer drugs — Based on their findings, of the 123 FDA-approved cancer medications, only 2.4% met all four of the FDA’s scientific criteria. On the other hand, 29 of these drugs tested (23%) did not meet a single criteria.

• AIDS — the worldwide crisis that changed it all — When AIDS became a pandemic during the 1980s, killing 46,000 people and leaving 37,000 with a novel condition, activist groups turned to the FDA, seeking access to new drugs. These groups partnered with drug makers, who were only too eager to sell their products.

“Together, the activists and companies argued before the FDA that the compassionate, lifesaving strategy would be to loosen scientific standards for establishing the efficacy of drugs,” the report said.

• The agency caved in to immense pressure — Aside from a lack of reviewers to verify new drugs that are coming in, the FDA knows that drug trials can take years to complete. To provide a solution to the growing need for AIDS drugs, the agency created an “accelerated pathway” in 1992. This allowed manufacturers to provide preliminary evidence that their AIDS drugs were effective.

• Companies were allowed to use “surrogates” to track patient outcomes — Through the new guidelines set by the FDA, manufacturers only needed to provide laboratory tests, or imaging studies like CT scans. These evidence “don’t themselves track quality or quantity of life, but are hypothesized to be reasonably likely to predict a so-called ‘clinical benefit.’”

• Relying on surrogate outcomes allowed companies to get approval — Drugs were fast-tracked despite having shorter and cheaper studies. The expectation is that the companies would provide more substantive evidence of the drugs’ effectiveness after they were allowed to be sold on the market. However, not only do surrogates fail to correlate whether the drug truly improves a patient’s quality of life, but they also don’t reflect if a drug is causing harm.

• AZT (zidovudine) is one example of how surrogate outcomes are unreliable — When it received approval in 1987, this AIDS drug was expected to be a “raging success,” according to results that measured T-cells — these are disease-fighting cells that the virus attacks.

However, less than two years later, researchers who studied 365 patients treated with AZT reported disappointing results;⁷ not only did the patients’ T-cell levels return to pretreatment levels, but they also became prone to infections, malignancies, and even death.

• The dangers of FDA-approved Alzheimer’s drugs — Published in January, the first part of the report tells the story of Genevieve Lane, a senior with advancing Alzheimer’s disease who partook in the study trials for the experimental drug Leqembi. Lane received a placebo during the trial, but then began receiving the actual drug as part of the study’s extension phase — she was dead in six weeks. The cause? Severe cerebral inflammation, likely a result of taking Leqembi.¹⁰

• Lane’s case was not an isolated case — Others were also harmed while taking Leqembi. Out of 714 participants during the study’s extension phase, four deaths occurred. During the main trial, two patients were left disabled after using Leqembi, while 22% developed brain hemorrhage or swelling — more than double of those on the placebo (10%).

I’ve also previously written about the dangers of Leqembi — read this article for more information “FDA Gives Accelerated Approval for Risky Alzheimer’s Pill.”

• Alzheimer’s drugs were developed based on the amyloid hypothesis — The theory was that the disease occurs due to a buildup of amyloid beta protein in the brain. But although amyloid deposits are present in the brains of patients with this disease, some people with amyloid never become demented. What’s more, there’s no definitive proof that this protein causes the disease rather than being a byproduct of it.

• In fact, a “landmark” Alzheimer’s study has been recently retracted — An Alzheimer’s study that pointed to a specific form of amyloid beta protein as a major driver of memory has been retracted due to manipulated images, as proven by forensic analysis. Read more about it in “Landmark Alzheimer’s Study Retracted After Evidence of Data Manipulation.”

• A prescription drug for your bladder is leading to blindness — In the second part of the report, the researchers told the story of how a pharmaceutical drug is causing blindness. Dr. Nieraj Jain noticed the symptoms in his patients — their vision was becoming blurry and it was becoming difficult to see in bright sunlight or at night.

• Jain pinpointed a common denominator against his patients — Elmiron, a drug prescribed for a bladder condition called interstitial cystitis, was affecting his patients’ eyes by causing odd patches of pigment to form on the retina. Jain and his colleagues even published a study on this condition, dubbing it “pigmentary maculopathy.”

• However, it wasn’t the only side effect — The FDA has also received reports of dozens of patient deaths attributed to Elmiron. Some patients were also hospitalized due to severe colitis.

• Drugs with 4 points are marked green — All four scientific standards were met. However, this does not guarantee the drug’s effectiveness or safety; it only means the findings are more likely to support the manufacturer’s claims.

• Drugs with 3 points are marked yellow — One of the four minimal standards was not met; it suggests that the submitted evidence was not optimal.

• Drugs with 2 to 0 points are marked red — Two, three, or all of the standards have not been met. This means the evidence submitted is insufficient to verify claims of efficacy and safety.

1. Ask one simple question before starting any new drug — Before you agree to take any medication, ask your physician: “Is this drug proven to help with real-world outcomes, like survival or quality of life — or was it approved based on a lab result or surrogate marker?” If your doctor doesn’t know, that’s your cue to dig deeper. You need to know if the benefit you’re chasing is real — not just a shift in a blood test or a scan.

2. Look up the FDA approval history yourself — Go directly to the FDA’s Drugs database or search for the medication’s approval letter. You’ll often see whether the drug was approved under the Accelerated Approval pathway and what evidence was used. If it’s based only on surrogate endpoints like plaque reduction or tumor shrinkage, and not clinical improvement, that’s a red flag. Don’t assume a drug is effective just because it’s on the shelf.

3. Use the power of timing to your advantage — If a drug is new to market, wait. Most serious adverse effects show up after approval, once thousands of people are using it in the real world. If you’re not in an emergency situation, give it 12 to 24 months to see what post-market data reveals. History shows that many of these drugs never prove themselves once follow-up studies are done — and those studies often show risks that weren’t public before.

4. Limit polypharmacy if you’re over 50 or managing multiple conditions — If you’re someone dealing with multiple prescriptions — especially for chronic or age-related conditions — review every drug you’re on with a focus on whether it’s still necessary. The more medications you take, the higher your risk for interactions, especially when drugs were approved on weak evidence.

Start with the newest medications and work backward. Anything fast-tracked without proven long-term benefits should be first on your review list.

5. Work with a healthcare provider who prioritizes results over protocol — You want someone who thinks critically, not someone who simply follows pharmaceutical guidelines without question.

If your provider is quick to prescribe a brand-new drug that’s all over the news, ask what real outcomes it improves and what they’d recommend if the drug didn’t exist. Their answer will tell you whether they treat patients or promote products. You always deserve someone on your side who puts your outcome above a company’s sales pitch.