Does Your Physician Understand FDA’s Drug Approval Process?

Most patients assume that doctors know how the FDA approves the medications they prescribe. Most patients would be wrong! We suspect that is also true regarding pharmacists. As far as we can tell, there are few, if any, classes taught in medical, nursing or pharmacy schools about the FDA’s drug approval process. Most health professionals assume that the Food and Drug Administration knows what it is doing. That may not be a valid assumption.

FDA Says “Safe and Effective” Is the Standard! Is It?

I apologize! I know that I sound like a cracked record when it comes to FDA drug approvals, but this is really important! It affects everyone who takes a medicine.

The FDA has historically stated in no uncertain terms that it only approves medicines that are “safe and effective.” It still states on its website that for a drug manufacturer to get FDA’s approval for a pharmaceutical product it:

“…must demonstrate that its drug or biological product is safe and effective for the intended use…”

That has historically been the standard, but the FDA has modified this position. It also states:

“FDA approval of a drug means…the drug is determined to provide benefits that outweigh its known and potential risks for the intended population.”

That is a loophole that is big enough for a Boeing 747 to easily fly through. I won’t bore you with why, here, but you can read more about the FDA’s drug approval process at this link:

Why Have I Lost Faith in the FDA?

Should you wish to read more about the FDA’s Drug Approval Process, here is another article to give you pause:

How Safe and Effective Are Your Medicines? You Might Be Surprised!

How Well Do Doctors Fathom the FDA’s Drug Approval Process?

According to a survey published in the journal Health Affairs (Jan. 2024), not very well.

Of the more than 500 doctors who responded to the survey, less than half had a moderate or better understanding of how the FDA reviews and approves new medications and medical devices. Many physicians assumed that the FDA requires drug companies to provide evidence of clinical benefit.

The doctors in the survey included board certified internists, cardiologists and oncologists. So these were specialists who should be very familiar with the the way the FDA’s approval process affects the medicines they prescribe on a daily basis.

The investigators introduce the new study this way:

“Prior research has demonstrated that physicians have limited knowledge of the meaning of FDA approval. Specifically, in 2016 more than two-thirds of physicians believed that FDA drug approval meant comparable effectiveness to other approved drugs (which it does not) and that only drugs with statistically significant and clinically important effects received FDA approval (these are not requirements). Physicians overwhelmingly want drugs to be approved on the basis of at least two randomized trials demonstrating improved patient outcomes (that is, measuring how a patient feels, functions, or survives), not just surrogate measures (for example, blood test or imaging results), even though about 59 percent of drugs are currently approved based on changes to surrogate (nonclinical) measures.”

What Did the MDs Know About the FDA’s Drug Approval Process?

Over 40% of these clinicians believed that they had a good understanding of the FDA’s drug approval process. Most (59%) believed the way the FDA approves drugs was “about right.”

Many of the surveyed doctors doctors prioritize patient outcomes such as quality of life or reduced mortality. The majority of medications, however, are currently approved on the basis of “surrogate markers” such as lab results like cholesterol or blood glucose or blood pressure measurements.

In reality, though, such markers are not the real issue. What people care about is 1) will this medicine improve my quality of life? 2) Will this medicine prevent some bad outcome like a heart attack, blindness or nerve damage? And 3) will this medicine improve meaningful longevity?

We have likened surrogate markers to casino chips. The chips have no intrinsic value unless you can cash them in for real money. If you went to the cashier’s window with a pile of chips and the cashier refused to give you real money, you would be rightfully angry. And yet the FDA currently approves 59% of medicines based on “changes to surrogate (nonclinical) measures” according to the new study in Health Affairs.

What shocked us about the new study was that roughly one third of the doctors surveyed DID NOT KNOW what a “surrogate measure” was.

We have written about this whole issue in our article: Do Faster FDA Drug Approvals Mean Greater Danger?

The Big Revelations About the FDA’s Drug Approval Process:

The authors conclude:

“In this national survey, we found that many physicians lacked understanding of the level of evidence required for FDA approval and that they expect (or believe) it to be more rigorous than it is, particularly for drugs and medical devices that come to market through expedited pathways.”

Three fourths of the internists believed that most new drugs should be tested in at least two randomized controlled trials. That is no longer the case. The overwhelming majority of all physicians in the survey (98%) believed that the FDA should require drug companies to perform follow-up clinical trials if a medication were approved based on surrogate outcomes.

In theory, the FDA is supposed to require follow-up studies when a drug receives “accelerated approval” with a surrogate endpoint. That often does not happen.

In my opinion, these very smart doctors were unaware that the FDA rarely forces drug companies do such follow-up clinical trials.

Here is a direct quote about this very serious issue:

“We found that most physicians across all three specialties surveyed, but a lower proportion of oncologists, believed that when required postmarketing trials for drugs and devices are not completed, the FDA should withdraw approval and prohibit promotion of the products.”

The Reality About the FDA’s Drug Approval Process:

The FDA rarely requires drug companies to remove a medicine from the market or stop promoting it if critical follow-up clinical trials are not completed!

Final Words

The physicians who conducted the survey are brilliant.

One, Dr. Rita Redberg, is a cardiologist at the University of California, San Francisco. She was Editor of JAMA Internal Medicine for years. We have interviewed her on our nationally syndicated public radio show.

Aaron Kesselheim, MD, JD, MPH, is a Professor of Medicine at Harvard Medical School. His medical and legal background enable him to understand the FDA’s drug approval process better than most other health professionals. Dr. Kesselheim served on the FDA’s Peripheral and Central Nervous System Advisory Committee until he quit because of a disagreement over the approval of a new drug for Alzheimer’s disease.

Steven Woloshin, MD, MS, is an internist, Professor of Medicine and Director of the Center for Medicine and the Media at the Dartmouth Institute for Health Policy and Clinical Practice. He too has been a guest on our radio show and is one of the big thinkers in medicine today.

There are other prestigious authors of this research, but you get the idea. These were heavy hitters within medicine. Believe me when I tell you that they thought long and hard about this survey of their colleagues.

The results of this research are disappointing in the sense that they reveal that many doctors are not that knowledgeable about the FDA’s drug approval process. We suspect that pharmacists and nurses also lack a basic understanding of how the FDA determines drug safety and effectiveness:

“The prevalence of physicians reporting lack of understanding of drug and medical device approval reflects the limited coverage of these topics during medical education.”

The final sentence of this paper is revealing for what it says and does not say:

“In this national survey, we found that many physicians lacked understanding of the level of evidence required for FDA approval and that they expect (or believe) it to be more rigorous than it is, particularly for drugs and medical devices that come to market through expedited pathways.”

What Do You Think?

We would love to hear from you. Do you believe your doctors, pharmacists and nurses understand the FDA’s drug approval process? Does that concern you? Please share your thoughts in the comment section below. If you think this article might be of interest to family and/or friends, please scroll to the top of the page and click on an icon for email or social media. Thank you for helping us share this research with others.