For biotechs, waiting for new drugs to be approved is not as hard as it used to be. Since late last year, federal regulators have acted months earlier than expected to OK a bumper crop of new drugs from Massachusetts companies.
The speedy decisions are welcomed by businesses that have made massive investments in developing experimental medicines and are eager for them to start generating revenue.
But critics say the Food and Drug Administration’s hurry-up mode may sacrifice patient safety — accelerating under pressure not only from the industry and investors, but from a regulation-averse Trump administration.
In late December, Biogen Inc. received an early Christmas present when regulators approved Spinraza, the Cambridge company’s drug to treat spinal muscular atrophy. Earlier, Biogen had been told by the FDA to expect a decision in May of this year.
Combined, the new drugs are expected to bring in many billions of dollars annually.
And expediting reviews of treatments that could save or extend lives has long been an explicit goal of the FDA.
But after meeting with top drug company executives at the White House, President Trump
described the FDA’s approval process as “slow and burdensome” and said that he would back measures to get promising medicines to patients faster.
His nominee for commissioner of the FDA, Scott Gottlieb, who has strong ties to biopharmaceutical businesses, has similarly complained about the “cumbersome” approval process.
Yet there are dissenters.
“We’re concerned that the FDA has swung too far toward speedy approvals and away from making sure drugs are safe and effective,” said Michael Carome, who directs the health research group at the advocacy organization Public Citizen.
Those concerns bubbled to the surface during last week’s Senate grilling of Gottlieb, who pledged to maintain what he called the FDA’s “gold standard” of unbiased and science-based drug reviews.
He is widely expected to be confirmed.
Dr. Aaron S. Kesselheim, director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital in Boston, said the medical world will need to closely monitor Gottlieb and the FDA in the new era of deregulation. The agency’s review times are currently the fastest that they have ever been, Kesselheim said, with more than two-thirds of applications for new drugs given some kind of expedited review.
“Patients want drugs that work,” Kesselheim said. “To reduce the FDA’s role in the name of free-market deregulation is wrongheaded.”
A report in the New England Journal of Medicine last week
said that the FDA has approved more drugs than European regulators in recent years, and has done so two to three months faster, on average.
Each of the treatments approved last month from Massachusetts companies — EMD Serono’s drug for Merkel cell carcinoma, Tesaro’s for ovarian cancer, and Sanofi Genzyme’s for atopic dermatitis — was reviewed under at least one of the FDA’s expedited programs designed to bring products to market quickly, according to a spokeswoman for the agency, Sandy Walsh.
Walsh said that every drug candidate approved for US sale must be done so on its merits, and the product must demonstrate “meaningful results” for patients in clinical trials.
“For drugs in an expedited program, we make every effort to complete our reviews as quickly as possible, and in some instances this may allow a decision to be made prior to the [scheduled] goal date,” she said.
Under the Prescription Drug User Fee Act of 1992, the FDA committed itself to completing standard drug reviews in 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 designation, to speed up the process.
Some practices permitted under these accelerated pathways have been controversial, however.
For instance, drug makers sometimes are allowed to design clinical trials that focus on “surrogate endpoints” — meaning changes in laboratory test measures 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.
Drug companies, which help fund FDA reviews under the User Fee Act, have proposed provisions to quicken and streamline the process every five years, when the law comes up for reauthorization. But they deny they have pushed for steps that would compromise review standards.
“Our industry does not believe that the FDA should skirt safety and efficacy,” said Andrew Powaleny, public affairs director at the Pharmaceutical Research and Manufacturers of America, a trade and lobbying group.
“We want to provide the resources to make sure they can do their jobs. . . . There are tools in the toolbox, and surrogate endpoints is one of them.”
Aside from the pace of the approval process, there are questions within the FDA and beyond about whether the agency has been letting drugs onto the market without sufficient evidence to show they’re effective.
Last year, FDA staffers were divided over the value of a drug developed by Sarepta Therapeutics Inc. of Cambridge, 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 — after lobbying by patient advocates — as the first treatment for the muscle-wasting disease Duchenne muscular dystrophy.
While the FDA’s expedited pathways sometimes permit faster clinical trials with fewer patients, the agency and the industry can also ensure treatments are safe and effective by better monitoring patients during clinical trials and afterward, said Michael Ringel, a partner and managing director at Boston Consulting Group who 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 said.
“The industry wants to make money, but it also wants to get drugs out that work.”