FDA chief's bid to speed drug reviews hits turbulence

• But it's prompting concern about legal challenges and rushed reviews as Makary faces broader scrutiny over turmoil at the agency, including a succession of departures at the top of the FDA's drug center.

State of play: The program has so far awarded 18 vouchers for accelerated reviews to treatments for cancers, sickle cell disease, obesity, deafness, infertility and other conditions.

• For products that receive the voucher, the agency aims for reviews in one to two months rather than the typical 10-12 months.
• "We've got to try new things, we have to innovate, we have to be creative, we have to do things differently," Makary said when he announced the first slate of nine awards in October.

Yes, but: Critics say the criteria and process for obtaining the vouchers are opaque and driven by political appointees, not career staff, raising the prospect of favoritism.

• "We have significant concerns that this program will enable corruption by creating a new, lucrative gift for drugmakers and allies politically favored by President Trump," Rep. Frank Pallone (D-N.J.) and Sen. Bernie Sanders (I-Vt.), the ranking members of House and Senate committees overseeing the FDA, wrote to Makary late last year.
• They also said the vouchers envision "absurdly short" review timelines that could strain an FDA staff that's already depleted from layoffs, potentially leading to safety issues.
• Harvard researchers warned in the New England Journal of Medicine that the program "could undermine trust in the FDA's approval decisions" and "is ripe for both abuse and legal challenge," due to "broad and opaque" criteria.

Between the lines: The program is a departure from the way the FDA has traditionally done drug reviews, in part because a key criteria for receiving a voucher is whether the sponsoring company increases affordability by lowering drug prices or reduces use of health services.

• The agency has in the past been careful not to consider the price of a drug, focusing instead on safety and efficacy.
• "When I was at FDA, we were very, very rigorous about saying we don't consider price or cost," said Dan Troy, a former FDA chief counsel under President George W. Bush.
• "That's not [FDA's] thing. ... I'm not going to say it's patently illegal, but it's certainly in tension with the way the agency has historically operated," said Dan Troy, a former FDA chief counsel under President George W. Bush.

A key question is whether pharmaceutical companies will launch costly drug development efforts based on a program that isn't written into law or wasn't developed through the regulatory process.

• "Is a company going to plan and make decisions that are five years out based on what is essentially a press release?" said Troy, now a managing director at Berkeley Research Group.

• Concerns about arbitrary decisions were amplified by reports that FDA career staff was prevented from voting on awarding the first priority review.

The other side: Department of Health and Human Services spokesperson Andrew Nixon said the program is a "forward-looking innovation" to address "urgent national health needs."

• "Since its launch, the program has demonstrated how the FDA can modernize its processes to deliver promising treatments to patients more quickly while fully preserving the agency's gold-standard scientific review," he added.
• Early rounds of the awards have included products for Type 1 diabetes, nicotine vaping addiction, blindness and pancreatic cancer.
• Other awards have coincided with larger Trump administration priorities. Makary touted vouchers for obesity medications from Eli Lilly and Novo Nordisk in the Oval Office during an announcement of an agreement with the companies to lower certain prices in November.

The intrigue: Many of the drugs receiving vouchers are not completely new drugs. For example, some are for approvals for new uses of already-approved medications.

• Ryan Conrad, a visiting fellow at the Brookings Institution and former FDA official, said some of those reviews for drugs that are not brand new would likely not have taken 10 months even without the voucher.
• "It seems like it's being used as sort of a catch-all to just sort of promote what's happening at FDA," he said.

What we're watching: How many of the drugs awarded vouchers will actually be approved, and whether any run afoul of safety or efficacy concerns.