In February, after speaking with top pharma executives at the White House, President Trump described the FDA’s approval process as “slow and burdensome” and said he would back measures to get promising medications to patients faster. His nominee for FDA commissioner, Scott Gottlieb, has expressed a similar viewpoint. But critics say the proposed hurry-up mode may sacrifice patient safety, according to an article in the Boston Globe.
The concerns were voiced last week during the Senate’s confirmation hearing for Gottlieb, who pledged to maintain what he called the FDA’s “gold standard” of unbiased and science-based drug reviews.
He is expected to win the job.
Dr. Aaron S. Kesselheim, of Brigham and Women’s Hospital in Boston, told the Globe that the medical world will need to monitor Gottlieb and the FDA in the new era of deregulation. The agency’s review periods are currently the fastest that they have ever been, Kesselheim said, with more than two-thirds of applications for new drugs given an expedited review.
“There’s no evidence the FDA is [in] a bottleneck,” he said, dismissing such assertions by Trump administration officials as “ideologically driven” and nonscientific.
“Patients want drugs that work,” Kesselheim said. “To reduce the FDA’s role in the name of free-market deregulation is wrongheaded.”
Under the Prescription Drug User Fee Act of 1992, the FDA committed itself to completing standard drug reviews within 10 months and said that it would make “priority” reviews—for drugs offering a treatment where none exists—within six months. The agency has since added other categories, including fast track, accelerated approval, and breakthrough therapy designations, to speed up the process.
Some practices permitted under these accelerated pathways have been controversial, according to the Globe. For example, drug companies sometimes are allowed to design clinical trials that focus on “surrogate endpoints”— changes in laboratory measures that are not felt by patients—rather than on tangible clinical benefits, such as reducing symptoms, keeping patients out of the hospital, and helping them to live longer.
In addition to the pace of the approval process, questions have been raised both within the FDA and beyond about whether the agency has been letting drugs onto the market without sufficient evidence to support their claimed efficacy.
Last year, for instance, FDA staffers were divided over the value of eteplirsen, developed by Sarepta Therapeutics, with some arguing that the company’s tests on a small number of patients failed to prove a clinically meaningful benefit. The drug was ultimately approved as the first treatment for Duchenne muscular dystrophy.
Both the FDA and the industry can ensure that treatments are safe and effective by better-monitoring patients during clinical trials and afterward, according to Michael Ringel, a managing director at Boston Consulting Group, which focuses on health care businesses.
“There are smart ways to take advantage of the data tools to get drugs on the market faster and monitor them to make sure they do what they’re supposed to do,” Ringel told the Globe. “The industry wants to make money, but it also wants to get drugs out that work.”
Source: Boston Globe; April 10, 2017.