FDA advisory panel votes against approval of DMD drugWhen one makes the decision to take a prescription medicine, the assumption is that the drug is both safe and effective. Indeed, perhaps the medication’s safety is presumed even over its effectiveness; whether you know anything about the approval process, there’s a tacit belief that prescriptions have undergone rigorous safety trials before they can be sold on the open market.

But if Trump’s nominee for heading up the U.S. Food and Drug Administration (FDA) runs the agency in the spirit of his past sentiments, and if he follows Trump’s lead (Trump has said he plans to “slash the restraints” on the FDA’s “slow and burdensome approval process”), drugs might make their way to market quicker than at present. And this leads some industry professionals to worry about consumer safety.

Gottlieb disparages FDA for drug trial: “unreasonable hunger for statistical certainty”

In a 2012 article that appeared in National Affairs, Scott Gottlieb discussed an FDA trial for a drug to treat Hunter syndrome, a rare, debilitating genetic disease that affects children. An experimental drug (Elaprase) was developed in 2002, and parents understandably clamored to take part in the clinical trial, hoping, of course, for a safe and effective treatment for the disease.

Gottlieb wrote: “Instead, what these families experienced exemplified a broken and dysfunctional approach to drug trials, driven by an FDA culture poorly suited to serving the needs of the sickest patients. […] In an effort to satisfy an increasingly unreasonable hunger for statistical certainty on the part of the FDA, the trial imposed extraordinary hardships on the children and families involved.”

Among the hardships, Gottlieb noted that the FDA required the clinical drug trial to include nearly 100 patients with the disease (“some 20% of all Americans afflicted”) and also stated that unlike other enzyme-replacement therapy studies, participating patients were randomly placed in a placebo group or a drug group. He criticized the FDA for this protocol, saying that the experimental therapy group “could easily have been compared against readily available historical databases that track the normal course of the disease,” rather than against those children blindly receiving the inert placebo.

FDA crucial in limiting thalidomide tragedy in U.S.

Gottlieb went on to acknowledge the roots of the FDA’s cautionary approach to newly developed drugs in the thalidomide tragedy of the 1960s, what he called a “dreadful episode.” In the U.S. and Europe, thousands of pregnant women were prescribed the drug to treat morning sickness; because prenatal imaging was not available then, the devastating harm done was not known until childbirth, when infants were born with severe birth defects.

Because an FDA reviewer delayed thalidomide’s approval in the U.S., citing safety concerns (even before the results were discovered elsewhere), the tragedy was not as widespread as in Europe.

However, Gottlieb expressed frustration with the FDA rigidly sticking with an approval process that he believes has outlived its necessity. He said that the FDA should take Europe’s example and allow “a body of politically appointed (and therefore politically accountable) officials … [to] ultimately [decide] on whether a new drug should be approved.”

Study reveals that U.S. already ahead of European counterparts

Despite Gottlieb’s relative criticism of the FDA’s approval rate and his lauding of the European Union’s example, a study recently published in the New England Journal of Medicine reveals that the U.S. is currently outpacing its European counterparts regarding time to market.

Analysts looked at activity from 2011 and 2015 (during which 170 new drugs earned the FDA’s approval for U.S. marketing) and concluded that the FDA’s median time for reviewing applications for “new therapeutic agents” was 306 days. During the same timeframe, the European Union counterpart gave the green light to 144 new drugs with a median time to review applications of 383 days.

Additionally, the FDA was “ahead” in another regard—the agency granted approval to a greater number of “orphan” drugs (those intended to treat rare conditions that lack other reliable means of treatment) than the European Medicine Agency (43% to 25%).

Gottlieb has his eye on the government’s “regulatory buck”

“We need to make sure we’re getting the most bang for our regulatory buck,” Gottlieb said in his confirmation hearing opening statement. “That means being cognizant of risks and being sure that we’re not adding to consumer costs without improving consumer safety.”


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