ICER Urges Greater Transparency in FDA’s Accelerated Drug Approval Process

The Institute for Clinical and Economic Review (ICER) has called on the U.S. Food and Drug Administration (FDA) to improve transparency in its accelerated drug approval pathway, citing ongoing concerns about evidence standards and follow-through on clinical validation.

The accelerated approval program is designed to fast-track treatments for serious conditions lacking effective therapies. It allows drugs to be approved based on surrogate endpoints—biological markers that suggest potential benefit—rather than direct clinical outcomes, significantly reducing time to market. However, drugmakers are required to conduct confirmatory trials post-approval to validate effectiveness.

Growing Scrutiny Over Evidence Standards

Recent approvals under the pathway include therapies from Denali Therapeutics and Rocket Pharmaceuticals. While the program has enabled faster access to treatments, it has also sparked debate over safety and efficacy.

A notable example is Biogen’s Alzheimer’s drug Aduhelm, which received accelerated approval in 2021 despite limited evidence of clinical benefit and was later withdrawn. The case intensified scrutiny of the FDA’s decision-making process.

ICER CEO Sarah Emond highlighted the tension between speed and rigor, noting concerns that some drugs are approved with insufficient evidence, while stricter standards could also limit access to new therapies.

Recommendations for Reform

ICER’s report outlines several proposals to strengthen the pathway, including:

• Enhancing the selection and validation of surrogate endpoints
• Requiring advisory committee reviews for accelerated approvals
• Increasing transparency in regulatory decision-making
• Introducing a scoring system to track clinical trial quality and outcomes
• Strengthening enforcement of confirmatory trial requirements

The report also suggests linking drug pricing to evidence development, with value-based pricing models that adjust as more clinical data becomes available, and reducing patient cost burdens when no alternatives exist.

Industry Perspective

The National Pharmaceutical Council, whose members include major pharmaceutical companies such as Eli Lilly, Biogen, and Johnson & Johnson, defended the pathway, stating that it generally delivers value to patients and that its benefits outweigh associated risks.

Balancing Speed and Accountability

The FDA has already taken steps to refine the process, issuing updated guidance on evidence requirements, including stricter criteria for surrogate endpoints that are “reasonably likely” to predict clinical benefit.

As demand grows for faster access to breakthrough therapies, ICER’s recommendations highlight the need to balance innovation with accountability—ensuring that expedited approvals are supported by robust evidence and transparent oversight.