Dr. Scott Gottlieb, President's Trump's nominee to head the Food & Drug Administration, has said the FDA displays an "unreasonable hunger for statistical certainty" and a "profound lack of confidence in the ability of doctors to make careful judgments."
In a bid to speed reviews and "change the FDA review culture itself," Gottlieb proposed in a 2012 article that the FDA should follow the lead of its European Union counterpart, and let "a body of politically appointed (and therefore politically accountable) officials … ultimately [decide] on whether a new drug should be approved."
This week, as Gottlieb came before a Senate panel, a study published in New England Journal of Medicine suggests that, when it comes to approving drugs, the agency he would lead outpaces others'.
In an analysis of the process by which 170 new drugs were approved for marketing in the United States, researchers found that the FDA has taken a median of 306 days to review applications for "new therapeutic agents."
During the period studied — 2011 to 2015 — the FDA's European Union counterpart approved 144 new drugs as safe and effective for the European market, and took a median of 383 days to review those applications. And compared to the European Medicine Agency, the FDA approved more "orphan" drugs — those proposed to treat rare disorders or diseases for which other effective therapies do not exist.
Between 2011 and 2015, the FDA approved 43.5% of applications for drugs with the "orphan" designation. The EMA approved 25% of orphan drug applications submitted during that period. With Congress poised to consider the reauthorization of the PDUFA before it expires in October 2017, the speed of the FDA regulatory review process will come under renewed scrutiny.
On Wednesday, Gottlieb told the Senate's Health, Education, Labor and Pensions Committee, "People's lives are literally on the line when it comes to the decisions FDA makes, its oversight, and its enforcement of Congress's laws."
"I know what's at stake here," said Gottlieb in his opening statement.
But Gottlieb's past statements, along with Trump's call to "slash the restraints" on the FDA's "slow and burdensome approval process," have mobilized supporters of the agency's cautious approach to drug safety.
Compared to other nominees reportedly considered by Trump to lead the FDA, Gottlieb appears to support a less radical overhaul of FDA policies. In recent weeks, however, critics have warned that reforms hinted at by Trump and Gottlieb would endanger patients and erode the U.S.'s reputation for setting a world standard for establishing the safety and effectiveness of drugs and devices it approves.
Writing recently in NEJM, Harvard University's Daniel Carpenter suggested that Gottlieb's libertarian ideas about drug regulation would politicize the drug approval process, give deep-pocketed pharmaceutical companies a dangerous edge in the approvals process, and erode the safety and effectiveness of drugs approved for the U.S. market.
The controlled clinical trials the FDA demands "provide the best attainable evidence that drugs and devices are trustworthy," wrote Carpenter. The standards the FDA currently upholds "provides a foundation for confidence in our entire medicine cabinet," he added.
Gottlieb's idea of placing approval decisions in the hands of a politically appointed panel "would undermine [the agency's] credibility by injecting overtly political considerations into drug approval," Carpenter wrote. "Drug approval would become a political, even partisan, ball game, since decisions would rest with officials who can be directly and immediately fired by the president."
American insurers and other countries "would rightly view these decisions with suspicion," added Carpenter. And FDA staff, in a defensive crouch over changes, "might well become more cautious, not less," he added.
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