Drugs

White Paper on Improving Accelerated Approval Path

The Institute for Clinical and Economic Review (ICER) has released a white paper with recommendations for improving the FDA accelerated approval pathway. The paper, “Strengthening FDA’s Accelerated Approval Pathway: Progress and Unfinished Business,” reviews recent regulatory and market changes and identifies several policies that could be adopted to improve the pathway further.

“The goal of the accelerated approval pathway has always been to ensure that patients receive innovative treatments faster,” says Sarah Emond, 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 paper discusses the potential benefits and challenges of these policy recommendations:

develop mechanisms to distinguish between promising surrogate endpoints and those that are less likely to succeed;
include outcomes that matter most to patients in clinical trials supporting accelerated approval;
standardize FDA explanations of accelerated approval decisions;
require informed consent of patients treated with drugs that are still in confirmatory trials;
tie price to value, to enable price changes when additional information becomes available; and
establish procedures to re-review or sunset accelerated approvals lacking confirmatory evidence.

ICER says this paper builds on a 2021 white paper that identified several potential areas for reform in the 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 drug companies and payers.

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