Last year’s approval of Biogen Alzheimer’s drug Aduhelm led to a flurry of skepticism over the wisdom of green-lighting drugs whose clinical benefit is still somewhat up in the air. Now, the issue is being taken up in Congress. 

Lawmakers are debating whether to reform or adjust the U.S. Food and Drug Administration’s accelerated-approval pathway, after what many deemed a dubious use of the regulatory route by Aduhelm. Proposed changes to the process feature in two of some 22 health bills put before lawmakers in a recent hearing

One, a Republican-supported measure, would make it easier for the FDA to use the pathway. The other, a Democratic-led bill, would restrict it. The latter would also empower the agency to hold drugmakers accountable if they violate the pathway by not providing follow-up studies proving the drug’s clinical benefit.

Frank Pallone (D-NJ), who chairs the House Energy and Commerce Committee, introduced his bill, Accelerated Approval Integrity Act of 2022, this month. The measure aims to strengthen requirements for post-approval studies for drugs traveling the accelerated route.

Pallone’s bill would place expiration dates – with a five-year maximum – on such drugs, in order to ensure manufacturers complete their confirmatory trials in a timely manner.

“It’s critically important for patients and for us as clinicians to know the true benefit and safety, especially for these drugs getting approved early on,” Dr. Reshma Ramachandran, a family physician and fellow in the Yale National Clinician Scholars Program, said at the hearing.

“The reason why these post-approval studies are so important is because we’re prescribing these drugs with a lot of uncertainty to our patients,” Ramachandran added. “[We need to know the drugs] are truly clinically beneficial, that the surrogate endpoint the drug was initially approved on is predictive and demonstrates clinical benefits for our patients.”

The alternative to that, Ramachandran pointed out, is the possibility of patients turning to a drug with no clinical benefit or that is potentially unsafe. She added that the financial ramifications of paying for a drug with unproven benefit are large for such patients, as well.

On the other side of the debate is Rep. Brett Guthrie (R-KY), who pushed for the removal of restrictions on the accelerated-approval pathway, arguing that speeding up the process would be more beneficial to patients.

“Approval of this new Alzheimer’s treatment through the accelerated-approval pathway could lead to other potential benefits, including the development of more effective treatments and encouraging investments in finding a cure for this terrible disease,” Guthrie noted

“Instead of adding more red tape, we should be focused on developing policy solutions that are intended to break down regulatory barriers and promote more collaboration between the regulatory community and private-sector partners.”

Guthrie has also introduced a bill, dubbed the Pre-Approval Information Exchange Act, which he said would aim to reduce the time between a drug approval and coverage of the drug by a payer.

The debate around accelerated approval heated up in the wake of Aduhelm’s June 2021 approval. In addition to skepticism from the medical and scientific communities, the controversy was further stoked when internal memos showed that the discord started within the FDA’s own walls. The regulator overruled its own biostatisticians in order to approve the drug.

The issue will be one of the first priorities for FDA’s newly confirmed FDA commissioner, Dr. Robert Califf. Although, he’s sure to face pushback against any potential legislation seeking to crack down on drugmakers. Industry lobbying group PhRMA has voiced support for the accelerated-approval pathway in its current form, arguing that it’s a safe and effective way to get medicines to the public faster.

“The ability for the FDA to approve products under accelerated approval has improved and even extended patients’ lives,” Andrew Powaleny, senior director of public affairs at industry group PhRMA, wrote in a recent post.

Powaleny also argued that the process has proven as rigorous as traditional FDA approvals, and that, “of the 165 therapies that received accelerated approval over the last 10 years, only six indications have been withdrawn.”

The proposed accelerated-approval bills will have a chance of being included in the FDA funding legislation to be authorized by Congress by September. Thus far, any movement on the issue remains uncertain.