Accelerated approval reforms take center stage at House hearing, but a way forward remains unclear
The House Energy & Commerce committee’s health subcommittee on Thursday dug into almost two dozen different FDA- and pharma-related bills that may be tacked onto a larger bill or even the biopharma user fee bills, which have to be reauthorized before the end of September.
The massive batch of bills making their way through the House includes two critical bills that seek to reform the FDA’s accelerated approval process in different ways.
On the one hand is E&C chair Frank Pallone (D-NJ), who, to the chagrin of industry, is seeking expiration dates (5 years max) on accelerated approvals given the fact that companies often drag their feet on confirmatory trials. His bill is running into opposition from Republican Cathy McMorris Rodgers of Washington, who sought to make clear the current accelerated pathway is functioning well, but that the pathway needs to be modernized for some new drugs, like those for ALS, that don’t have a clear surrogate endpoint.
McMorris Rodgers’ bill would make it so that the evidence used to support that an endpoint is reasonably likely to predict a clinical benefit under an accelerated approval “may include epidemiological, pathophysiological, therapeutic, pharmacologic, or other evidence developed using biomarkers, for example, or other scientific methods or tools.”
Pallone, meanwhile, offered the example of Biogen’s controversial new Alzheimer’s drug Aduhelm, which won an accelerated approval last June.
“Here we are nine months later, and the sponsor has not screened a single patient for its required confirmatory trial,” Pallone said. “Other drugs have stayed on the market for eight or nine years without proving a clinical benefit.”
He also noted how CDER Director Patrizia Cavazzoni testified last month on how “the process for removing these drugs from the market is cumbersome and can take months or even years.”
Ranking member Rep. Brett Guthrie (R-KY), however, took the other side on the Aduhelm debate, saying that CMS’ decision to restrict coverage to only clinical trials could have a chilling effect on other investments in Alzheimer’s.
Supporting the accelerated approval status quo, Jeff Allen, CEO of Friends with Cancer Research, noted that accelerated approvals converting to full approval took a median time of 3.1 years. He also said that if accelerated approval drugs are inappropriately limited, there could be delays in access, but the hallmark of the accelerated pathway is balancing these uncertainties.
“I think the legislation proposed today to strengthen the post-market surveillance side will help to expand the development of surrogate endpoints,” he noted.
Reshma Ramachandran, physician-fellow in Yale’s National Clinician Scholars Program, testified that pharma companies often market drugs via the accelerated pathway for long periods of time prior to the confirmatory trial results. And sometimes that marketing continues even after confirmatory trials have failed.
She noted that Pallone’s bill builds in flexibility for FDA on when appropriate confirmatory trial completion date can be set, and it allows for negotiations between the sponsor and FDA. That would mean drugs won’t be automatically withdrawn if such a drug is still in a confirmatory trial.
In addition to accelerated approvals, the bills discussed at the hearing also include further funding and structure for ARPA-H, a new NIH-housed agency (with $1 billion in starter funds) that will correct the gap between the basic research at NIH and the development of products in the private sector, according to health subcommittee chair Anna Eshoo (D-CA). Eshoo also praised the years’ worth of work in the latest iteration of the 21st Century Cures Act.
Rep. Fred Upton (R-MI), who co-sponsored both Cures bills with Rep. Diana DeGette (D-CO), noted on Thursday that Covid “has taught us a valuable lesson that FDA can work quickly and that RWE will improve its decisionmaking.”
Three other bills discussed would seek to improve clinical trial diversity, while others will help with generic and biosimilar development, including one that will allow some generics to be approved with a “temporarily different” label than its reference product.