Collins, Kaine Bill to Improve Accelerated Approvals for Lifesaving Drugs Signed into Law

Washington, D.C.—U.S. Senator Susan Collins (R-ME) announced that the Modernizing Accelerated Approval Act, legislation she co-authored with Senator Tim Kaine (D-VA), was signed into law as part of the year-end government funding package. 

The Modernizing Accelerated Approval Act will make significant improvements to the FDA’s accelerated approval pathway, an important regulatory mechanism that provides early access to treatments for patients with serious and life-threatening conditions.  It aims to encourage the FDA to better utilize this pathway, sets benchmarks for accelerated approval development plans, and strengthens transparency throughout the process. 

“Over the past three decades, accelerated approvals have allowed hundreds of innovative and effective treatments to reach patients years faster than they otherwise would have, while maintaining the FDA’s rigorous approval standards,” said Senator Collins.  “Our bipartisan legislation will build on this success and encourage more ground-breaking treatments by standardizing the process across the FDA, expediting the completion of post-approval studies, and increasing transparency.  Taking these commonsense steps will improve the accelerated approval process and help ensure that this pathway remains a lifeline to patients with serious illnesses.”

The accelerated approval pathway—which was established by the FDA in 1992 and codified in 2012 —helps new treatments reach patients with serious illnesses more quickly. Under this regulatory process, manufacturers must meet the same “substantial evidence” standard as regular approval. Accelerated approval can be granted, however, if there is “substantial evidence” that a new drug can improve a marker—such as reducing tumor size for cancer patients or reducing viral loads for HIV patients—that can predict a drug’s effectiveness at treating the underlying condition. After the drug is made available to patients, the manufacturer is still required to complete studies confirming the drug’s benefits.

In recent years, some shortcomings of the accelerated approval pathway have come to light. For instance, some FDA divisions, such as the Oncology Center of Excellence, have vast experience and success using this pathway, while others, such as the Division of Neurology, only use it infrequently. Additionally, confirmatory trials are a critical part of the accelerated approval pathway, but they can be delayed by challenges. Transparency around the status of these trials is necessary to ensure patient and provider confidence in these important therapies.

The Modernizing Accelerated Approval Act will improve the accelerated approval pathway by:

• Establishing a council of senior FDA leadership that will ensure consistent and appropriate use of the accelerated approval pathway across and within FDA centers and divisions.

• Clarifying that the Secretary may require post-approval studies to be underway prior to approval, and clarifying that the Secretary may specify the conditions for a post-approval study or studies, which may include enrollment targets, the study protocol, milestones, and a target date for study completion.

• Requiring FDA to report to Congress on the use of real world evidence to support post approval studies and issue guidance on novel surrogate

endpoints and clinical trial designs.

• Increasing transparency by (1) requiring FDA to publish on its website why a study is not appropriate or necessary if the agency does not require that a product approved under accelerated approval conduct a post-approval study, and (2) requiring that sponsors of drugs approved under accelerated approval submit to the Secretary a report of progress on required post-approval studies every six months and that the FDA post that information in an easily accessible format.

• Requiring the Secretary to issue guidance on the use of novel endpoints and clinical trial designs in accelerated approval and on the expedited withdrawal procedures.

• Authorizing the Rare Disease Endpoint Advancement Pilot that FDA agreed to in the PDUFA VII commitment letter, which will bolster trials for rare diseases.

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