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BOSTON, April 16, 2026 – The Institute for Clinical and Economic Review (ICER), in collaboration with Verdant Research, has published a new white paper today that examines the evolution – and perception – of the FDA accelerated approval pathway. The paper, titled “Strengthening the FDA's Accelerated Approval Pathway: Progress and Unfinished Business,” reviews recent regulatory and market changes and identifies several policies that could be adopted to further improve the pathway.
Downloads: White Paper
The recommendations include strengthening the evidence submitted to the FDA, ensuring that patients and clinicians understand the benefits and risks of a product approved under accelerated approval, aligning prices with evidence and value, and increasing regulatory transparency around accelerated approval decisions, among others.
“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. “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.”
The analysis relies on data and perspectives gathered from a targeted literature review and interviews with members of the Policy Leadership Forum (PLF) – which includes life sciences companies, health plans, pharmacy benefit managers, and purchasers – as well as patient groups, and regulatory and clinical experts.
The white paper went through several iterations following ICER’s Policy Summit in December 2025, where patient advocacy, regulatory, and Medicaid experts joined with ICER’s PLF to deliberate on the policies outlined and provided suggestions for revisions to a draft version of this paper. None of the individuals involved in this meeting, nor their organizations, should be assumed to support any part of this report, which is solely the work of the editorial team at ICER and Verdant Research.
The paper details the possible benefits and challenges of several policy options meant to strengthen the use of the pathway, including:
- Development of mechanisms to distinguish between promising surrogate endpoints and those that are less likely to succeed;
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Inclusion of outcomes that matter most to patients in clinical trials supporting accelerated approval;
- Standardized explanations from the FDA of accelerated approval decisions;
- A requirement of informed consent of patients treated with drugs that remain in confirmatory trials;
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Pricing tied to value, to enable price changes when additional information becomes available;
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Procedures to re-review or sunset accelerated approvals lacking confirmatory evidence.
This paper builds upon a previous ICER white paper, published in 2021, that identified several potential areas for reform in the accelerated approval pathway, including concerns about the selection of surrogate endpoints, lagging confirmatory trials, low levels of evidence, and problematic financial incentives that required greater accountability of the U.S. regulatory system, as well as pharmaceutical companies and payers.
ICER develops annual white papers in collaboration with the ICER Policy Leadership Forum. Previous papers have addressed topics such as Examining Strategies to Ensure Affordable Access for Obesity Medications, Managing the Challenges of Paying for Gene Therapy, Strengthening the FDA’s Accelerated Approval Pathway, Cornerstones of ‘Fair’ Drug Coverage, Reforms to Orphan Drug Development, Pricing, and Coverage, Alternative Models for Pharmaceutical Rebates, and White Bagging, Brown Bagging, and Site of Service Policies.
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