As Congress prepares to pass some form of Food and Drug Administration user fee reauthorization, lawmakers are considering whether to include other pharma-related provisions, including accelerated approval reform and clinical trial diversity efforts.
This week, the Senate released a draft version of its user fee reauthorization bill, which would reauthorize all user fees for drugs, medical devices, generic drugs and biosimilars.
The user fee process, which the FDA first authorized in 1992, entails pharma and medical device companies paying the agency when submitting regulatory applications. User fees have historically been used to bolster the FDA’s work in reviewing the safety as well as the efficacy of drugs, and must be renewed every five years.
Rachel Sher, a regulatory expert and partner at Manatt, Phelps & Phillips, explained that the user fees must be reauthorized by the end of the fiscal year or the FDA loses its statutory authority to collect fees from any of the programs.
“The user fee reauthorization bill itself is always conceived as a must-pass vehicle because user fees today represent such a large proportion of the respective medical product center’s budget and the critical activities those centers do,” Sher said.
However, whether that must-pass bill will incorporate accelerated approval reforms or other provisions remains to be seen. The Senate version of the user fee reauthorization bill doesn’t include any reform related to accelerated approval – the FDA pathway that helps drugs get approved more quickly.
Additionally, the Senate version includes amendments to the federal Food, Drug and Cosmetic Act that would entail changes to cosmetic products, dietary supplements and in vitro clinical tests.
In comparison, the House version of the bill – which a bipartisan committee voted to advance Wednesday – includes both an accelerated approval reform provision and one that aims to improve diversity in clinical trials.
“It’s unusual to see the House and Senate contain such significant provisions that are not reflected in the other chamber’s bill,” Sher noted.
Still, she believes that accelerated approval reform is likely to emerge in the Senate again, possibly as a tweak or provision in later drafts of the bill – even if it was left out of the discussion this time around.
“Given the strong bipartisan interest that we saw in the House for looking at necessary changes and improvements to the accelerated approval pathway, this is a provision that I think we can expect to see added somewhere along the line in the Senate process too,” Sher said.
The debate over the FDA’s accelerated approval pathway has been ongoing since the agency’s controversial decision to approve Biogen’s Alzheimer’s drug, Aduhelm. FDA Commissioner Robert Califf has also pledged to tackle the issues associated with accelerated approval during his time as commissioner.
Given the level of criticism that the process has endured, some sort of adjustment is overdue, according to Henrietta Awo Osei-Anto, director of FasterCures at the Milken Institute.
“The accelerated approval process has been an important mechanism for getting treatments to many people that are living with serious conditions that have no therapeutic options,” Awo Osei-Anto said. “I don’t think there are any doubts about the importance of preserving that mechanism. But with the recent approval of Aduhelm, the mechanism in the process has been passed back under scrutiny, and that’s reinforced the need to strengthen or refine the process.”
One example of the challenges the FDA has faced with the accelerated approval pathway is removing products from the market if they don’t meet post-market requirements.
Awo Osei-Anto noted that it’s too soon to tell what the final legislation may look like – but she hopes this offers an opportunity for lawmakers to start moving the needle on improving the accelerated approval pathway for the future.
“The FDA needs bipartisan congressional support in order to be able to make any meaningful changes to the process and strengthen it,” Awo Osei-Anto said. “We still have a ways to go, but this is an important opportunity to address some of the components that are controversial right now and ensure that we’re moving forward with a process that benefits patients and multiple stakeholders.”