A new ICER white paper calls for reforms to the FDA’s accelerated approval pathway, citing ongoing challenges with regulatory consistency, confirmatory trial delays, and transparency.
The Institute for Clinical and Economic Review (ICER), in collaboration with Verdant Research, released a new white paper examining the evolution of the FDA’s accelerated approval pathway.
The paper highlights both progress and persistent challenges as stakeholders call for reforms to improve consistency, transparency, and patient access.
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“The goal of the accelerated approval pathway has always been to ensure that patients receive innovative treatments faster,” said Sarah K. Emond, MPP, president and CEO of ICER.
Titled “Strengthening the FDA’s Accelerated Approval Pathway: Progress and Unfinished Business,” the report evaluates how the pathway has functioned in recent years and outlines a series of policy recommendations aimed at reinforcing its original intent, expediting access to innovative therapies while ensuring robust evidence generation.1
“We can point to several successes where patients have benefited from expedited access to transformative medications. However, there are also many instances of regulatory inconsistency, lagged development of confirmatory trial data, and access restrictions that have limited patient use. Patients, manufacturers, payers, and purchasers are frustrated. The policy options laid out in this paper are designed to build on the successes of the accelerated approval pathway and address the challenges that remain, in service to affordable access for patients,” said Emond.2
The analysis draws on a targeted literature review and stakeholder input, including interviews with members of ICER’s Policy Leadership Forum, comprising life sciences companies, health plans, pharmacy benefit managers, and purchasers, as well as patient groups and regulatory experts.1
The findings were further shaped by discussions at ICER’s December 2025 Policy Summit, where participants provided feedback on earlier drafts of the report.2
At the core of the paper is a set of policy proposals aimed at strengthening evidentiary standards and improving how accelerated approvals are communicated and managed over time. Among the key recommendations are mechanisms to better distinguish between surrogate endpoints that are likely to predict clinical benefit and those that carry greater uncertainty, as well as the inclusion of outcomes that more directly reflect patient experience in clinical trials.
The report also calls for greater transparency from the FDA in explaining its accelerated approval decisions, alongside clearer communication to patients and clinicians about the benefits and risks of therapies that have not yet completed confirmatory trials.2
Additional proposals include requiring informed consent for patients receiving such treatments and implementing pricing models more closely tied to the level of available evidence, with adjustments as further data emerge.
The paper also revisits ongoing concerns about delays in confirmatory trials, recommending formal processes to reassess, or potentially withdraw, approvals when required evidence is not delivered in a timely manner.2 These issues have been a longstanding point of tension across the healthcare ecosystem, particularly as payers and policymakers weigh the balance between early access and clinical certainty.
The new report builds on ICER’s previous report from 2021 analyzing the accelerated approval pathway, which similarly identified gaps related to surrogate endpoint selection, evidence quality, and financial incentives.1
Together, the findings reflect a growing consensus that while the pathway remains a critical tool for speeding innovation to patients, particularly in oncology and rare diseases, its long-term credibility will depend on more consistent execution and accountability.
