Issues & Insights

The FDA’s Bad Medicine

By Henry I. Miller

Before leaving office, former Food and Drug Administration Commissioner Scott Gottlieb told the Senate Appropriations Committee that Congress should create a new requirement for opioid approvals. New opioids should have to demonstrate superiority over those already on the market, he said.   

That would be a departure from the current statutory requirement that drugs must simply be shown to be safe and effective for their intended use. And it would be a bad idea. It shows how out of step Dr. Gottlieb was with the intention of Congress and the Trump administration to increase competition in the marketplace and put downward pressure on drug prices.

For a variety of reasons, having another drug on the market that appears from clinical trials data to be no better than alternatives might be important. First, there are important differences between drugs, even if they act through similar mechanisms. A study in the Journal of Clinical Lipidology found that some formulations of statins, which are used to control abnormal blood lipids, were more likely to cause patients to experience side effects when administered with certain other drugs, making it critical for clinicians to be able to select among statins to minimize drug-drug interactions. 

Second, even if two drugs are both effective in 40 percent of patients with a particular symptom or disease, each of them may not work in the same 40 percent. Practicing physicians know that for certain conditions — such as treating pain or the symptoms of multiple sclerosis, or to prevent blood clots — patients whose conditions seem indistinguishable from one another sometimes do better on different drugs. If the drugs are effective in different populations, the unavailability of the second drug because it didn’t show overall superiority in clinical trials could deprive a large number of patients of the medicine they really need. Having alternatives is in the best interests of patients.

Third, “supplemental,” or secondary, approvals of drugs — which include new dosages, formulations and uses — account for “a substantial share of drug utilization and associated economic and medical benefits,” according to a study of three important classes of medicines by the Massachusetts Institute of Technology economist Ernst Berndt and his collaborators.

This suggests that if regulators had denied approval because initial studies failed to show the drug’s superiority to others already on the market, the benefits discovered later would have been missed. Consider, for example, interferon alfa-2a, which was first approved in 1986 to treat a rare blood condition called hairy cell leukemia. It soon stopped being the drug of choice for that disease but was later found to be effective for melanoma, Kaposi’s sarcoma and various viral infections. If the drug had been denied the initial marketing approval because other, equally good drugs already were available for hairy cell leukemia, its more important uses probably wouldn’t have been discovered.

Proving that a drug is better than existing alternatives often is much more difficult and vastly more expensive than just proving that it is safe and effective: If the efficacy of two medicines is only marginally different, the clinical trials must be very large in order to attain statistical significance. And worst of all, drug sponsors might not know that their drug isn’t superior to the alternatives until the very end of the years of clinical trials and analysis of the data. Typically, it takes 12 to 15 years and costs, on average, more than $2.5 billion to get a new drug to market.

Thus, new opioids useful for some patients may founder if the new criterion suggested by Dr. Gottlieb is implemented, thereby reducing competition in the drug market and putting upward pressure on prices. Robert Essner, the onetime chairman and CEO of the pharmaceutical company Wyeth, described the implications of requiring a demonstration of superiority this way:

“If you’re the first company to get approved in a certain area and competitors can’t get on the market, the FDA is now establishing monopolies. And that’s certainly not their mandate.”

Whatever one thinks of the FDA’s regulation to ensure safety and efficacy, surely we should not have regulators discouraging competition and making useful drugs unavailable. Dr. Gottlieb’s successor, (Acting) Commissioner Dr. Norman Sharpless should abandon the notion of requiring superiority. The demonstration of safety and efficacy is surely a high enough bar.  

Henry I. Miller, a physician and molecular biologist, is a Senior Fellow at the Pacific Research Institute. He was the founding director of the FDA’s Office of Biotechnology.

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