Problematic FDA Regulations

The FDA is maladapted to helping doctors select the best drugs to promote the health and well-being of American patients

Here’s a puzzler: When can government regulations requiring something almost everyone wants be a bad thing?

We need a little background information before answering this question.

What does almost everyone want? They want to know if a new drug, already deemed safe, will be efficacious for them. Will it reduce their blood pressure? Will it stop their breast cancer? Will it prevent blood clots? Will it help them lose 25 pounds?

What do government regulations require? The U.S. Food and Drug Administration requires drug companies to test new drugs for efficacy in two large randomized controlled trials.

How can these regulations, which result in the efficacy data that everyone wants, be wrong? There are two main reasons.

First, the efficacy data is derived from clinical studies the government mandates, but that doesn’t ensure the results address what the medical community needs to treat patients. While the medical community might want answers to questions A, B, and C, the FDA reviewers, who don’t treat patients and who do need to follow the bureaucracy’s rules while trying to impress their bosses, might design clinical trials to answer questions D, E, and F.

Second, the efficacy data shows how the drug worked in groups of people. Typically, some percentage of patients, say 60%, were helped and some percentage, say 40%, weren’t helped. While this information is useful when selecting a therapy to try, because a drug that helped 60% of patients in clinical trials appears to have an advantage over a drug that helped only 50%, it isn’t as valuable as it first appears because the clinical trials for the two drugs might have been apples and oranges. It isn’t obvious which drug—the “60%-drug” or the “50%-drug”—is better.

Further, this information doesn’t answer the most important question. This information doesn’t help a particular patient being treated by a given doctor to know which group he or she will end up in. If the patient takes the “60%-drug,” will the patient get results similar to the 60%-group or the 40%-group? Some patients benefited from the drug in the clinical trial, but not all patients, and we don’t know a priori which patients will end up in which group. The FDA doesn’t know. The drug company doesn’t know. Treating physicians don’t know.

Instead, the patient and doctor team must actually try the product in that person to see the results. This trial-and-error process must be used whether the FDA requires efficacy trials or not. The FDA’s efficacy trial requirement provides surprisingly limited information for patients and doctors.

In other words, even though people want good efficacy data, the efficacy data currently produced is of low value. Worse, it is very expensive. This is where the ears of economists perk up. If something is expensive but provides little value, perhaps it should be ceased.

This rather long explanation serves as an introduction to a paper entitled “Deregulating Drug Development” that was recently published by David R. Henderson, Solomon S. Steiner, and me in Law & Liberty. It might be one of the most important articles I’ve ever written. It’s not long and I hope you read it.