September is a great time for journalists to examine how well U.S. regulators are handling the accelerated approval pathway for medicines. There’s been growing concern about the ability of the Food and Drug Administration (FDA) to compel drugmakers to hold up their end of the bargain on agreements made to speed seemingly promising medicines to market based on little pools of data.
In this two-part blog post, we’re going to explain today why Congress faces pressure this month to pass a major FDA law, which could be the vehicle for addressing accelerated approvals. Then tomorrow we will provide some background on the concerns about how well the FDA can hold pharmaceutical companies to the bargains they make to get a quick clearance to sell seemingly promising drugs for serious conditions based on only limited evidence of benefit.
The current version of the Prescription Drug User Fee Act (PDUFA) expires on Sept. 30.
First passed in 1992, PDUFA created a system in which companies pay application fees for reviews of their products. This system creates streams of revenue that then allow the FDA to expand the pool of staff able to review these applications and thus speed the turnaround. Congress replicated the PDUFA model with laws for medical devices and generic drugs. There’s long been opposition to this approach.
Critics say PDUFA set the stage for what’s called “regulatory capture.” In an April letter to members of Congress, the watchdog nonprofit Public Citizen said the PDUFA model has caused the FDA’s work to be “skewed heavily towards placating the interests of the prescription-drug and medical-device industries with ever faster, and too often hasty, reviews of marketing applications.”
But PDUFA has strong bipartisan support in Congress. Lawmakers set five-year terms on PDUFA reauthorizations, giving themselves a regular opportunity for making changes to FDA policies. There have been five reauthorizations of the PDUFA law since 1992 — in 1997 (PDUFA II), 2002 (PDUFA III), 2007 (PDUFA IV), 2012 (PDUFA V) and 2017 (PDUFA (VI).
The pharmaceutical industry in the past has gotten enviable service from Congress in terms of PDUFA reauthorizations, as the law firm Akin Gump notes in this concise summary about the law. The current PDUFA reauthorization was signed into law on Aug. 18, 2017. The one before that became law on July 9, 2012.
Contrast that with how Congress sloppily manages the bills that fund most of the federal government’s operating expenses.
The federal fiscal year always starts on Oct. 1, a deadline that lawmakers know well in advance. In recent decades, though, Congress has failed in most years to pass the spending bills, known as appropriations, on time. Instead, it passes short-term measures known as continuing resolutions to buy more time to finish the appropriations bills. These stopgap measures force the leaders of federal agencies to cope with the disruptions and distractions of uncertain and tardy funding.
This year, there’s a threat that the FDA’s user-fee-funded programs could share in some of the drawbacks of stopgap funding that its appropriations-funded programs long have faced. Under continuing resolutions, federal agencies generally have to put new projects and hiring on hold, leaving staff operating in limbo. Congress sometimes allows continuing resolutions to lapse, leading to temporary government shutdowns.
But it’s likely possible that this month, Congress t will put off two of its pressing deadlines through a single measure.
It might add a short-term extension of the PDUFA law to an expected fiscal 2023 CR to buy time for further negotiations on the FDA bill, reported veteran Washington journalist John Wilkerson in Inside Health Policy on Sept. 1 (subscription required). This stopgap approach to PDUFA may be necessary to buy time to negotiate with Sen. Richard Burr of North Carolina, the top Republican on the Senate’s Health, Education, Labor and Pensions (HELP) Committee, Wilkerson reported.
The House in June passed its version of the 2022 PDUFA reauthorization in a 392-28 vote, with 176 Republicans supporting the measure.
But progress on a new PDUFA bill stalled in the Senate. The Senate HELP committee approved its version of a new PDUFA bill in a 13-9 vote in June. In July, Burr introduced what he called the Food and Drug Administration Simple Reauthorization Act. This streamlined measure left out many of the policies in the other Senate HELP and House-passed bills, including provisions meant to aid the FDA in managing delays in studies needed to show whether drugs cleared by accelerated approval work as expected.
“As the authorizing deadline approaches, this clean reauthorization represents the clearest path forward,” Burr said in a statement.
With this approach, Burr is seeking to use the threat of disruptions to the FDA’s drug-review workforce to block policies he doesn’t favor. Writing in Regulatory Focus, a publication of the Regulatory Affairs Professionals Society (RAPS), Michael Mezher reported that the FDA’s top leader fears the threat of layoffs because of a delayed PDUFA reauthorization could cause drug-review staff to reconsider whether they want to work for the agency.
“We’re in the period of the ‘Great Resignation’,” FDA Commissioner Rob Califf, M.D., says in the article. “Who wants to work in an organization if you’re afraid that these jobs are not even going to exist in the next short period of time? We really need to avoid that.”
The FDA does have some ability to delay layoffs for about five weeks if the PDUFA reauthorization stalls, Califf told the agency staff. In a memo that the FDA released publicly via Twitter, Califf said there were about five weeks of funding leftover to carry beyond the Sept. 30 cutoff.
“That means Congress has a tiny cushion of about a month, until Nov. 4, to get its act together,” Zachary Brennan wrote in Endpoints News in August.
Over the summer, Burr drew opposition from Senate HELP Chairwoman Patty Murray (D-WA), and both the top Democrat and Republican on the House Energy and Commerce Committee urged negotiations on broader PDUFA measures than Burr’s streamlined bill.
“Pink slips are unacceptable to me, and they should be unacceptable to all of us — that is why it makes absolutely no sense to back out of bipartisan negotiations now,” Murray said in a July statement. “The fastest way to get this done is to move forward with the comprehensive bipartisan bill we have already advanced out of committee, and which includes so many desperately needed, long overdue, and bipartisan policies. I urge Senator Burr to return to the table and finish what he started.”
In a joint statement, House Energy and Commerce Chairman Frank Pallone, Jr. (D-NJ) and Rep. Cathy McMorris Rodgers of Washington, the committee’s ranking Republican, urged their Senate colleagues to proceed with the bill they already passed.
“There is no reason for the Senate to reject the strongest and best path forward to ensure this reaches the president’s desk,” Pallone and Rodgers said. “For patients, it means lower health care costs, stronger supply chains, critical access to life-saving prescription drugs, and more hope in the promise for new cures and treatments that will improve their quality of life.”