Federal drug regulators should provide greater transparency when fast-tracking medications for serious medical conditions, according to a new report from a prominent pharmaceutical research organization.
The Institute for Clinical and Economic Review (ICER), a leading drug pricing watchdog, issued recommendations Thursday calling for improvements to the Food and Drug Administration’s accelerated approval process.
The FDA’s fast-track system allows quicker authorization of medications for severe conditions that lack existing treatments. This process relies on substitute markers that suggest clinical benefits rather than direct measurements, dramatically reducing the timeline for regulatory approval.
Companies receiving fast-track approval must still complete additional studies to confirm their drugs’ expected benefits.
Recent medications approved through this expedited process include Denali Therapeutics’ rare disease treatment Avlayah and Rocket Pharmaceuticals’ gene therapy Kresladi for pediatric disorders.
The rapid approval system has sparked debate about treatment safety and effectiveness. A notable controversy arose in 2021 when Biogen received accelerated approval for its Alzheimer’s medication Aduhelm despite lacking clear evidence of patient improvement. The company eventually discontinued the drug three years later.
ICER CEO Sarah Emond explained to Reuters that concerns exist on both sides of the issue. “Some are concerned that tougher evidence standards mean fewer drugs are being approved under the pathway while others say too many drugs are approved with weak evidence and without the required confirmatory follow-up,” Emond stated.
Last year, the FDA released updated guidelines clarifying its position on evidence requirements for expedited approvals, including developing substitute markers that are “reasonably likely” to predict clinical benefits.
The ICER report suggests multiple policy improvements for the accelerated approval system, including better selection of substitute markers, mandatory advisory committees for expedited reviews, and enhanced decision-making transparency.
“A scoring system for clinical trial and evidence quality could allow FDA to track AA pathway performance over time and across study sponsors and might serve as an early warning system to identify trials at risk of delay or failing to provide the necessary data for conversion to full approval,” the report stated.
Additional recommendations include connecting insurance coverage for fast-tracked drugs to fair, value-based pricing, adjusting costs as new evidence becomes available, and limiting patient expenses when no alternative treatments exist.
The organization also proposed stronger enforcement requirements for completing follow-up studies and conducting targeted re-evaluations by drug category or medical condition, focusing on those with the most uncertain evidence.
