Collins, Kaine Bill to Improve Accelerated Approval Process for Lifesaving Drugs Advances in Senate

WASHINGTON, D.C. — U.S. Senators Susan Collins (R-ME) and Tim Kaine (D-VA), members of the Senate Health, Education, Labor, and Pensions (HELP) Committee, announced that the Modernizing Accelerated Approval Act they co-authored advanced yesterday as part of the FDA Safety and Landmark Advancements (FDASLA) Act, a package to reauthorize the Food and Drug Administration’s (FDA) user fee amendments. Their bipartisan legislation would 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. 

The Modernizing Accelerated Approval Act aims to encourage the FDA to better utilize this pathway, sets benchmarks for accelerated approval development plans, and strengthens transparency throughout the process. Senators Collins and Kaine’s bill was included in the FDASLA—legislation reauthorizing the FDA’s prescription drug, generic drug, biosimilar, and medical device user fee agreements that passed the Senate HELP Committee by a vote of 13-9.

“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 bill would 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.”

“I’m proud this bipartisan bill to accelerate the FDA’s approval process for lifesaving medicines while also ensuring safety and transparency was incorporated into the user fee package,” said Senator Kaine. “This bill is an important step to ensuring patients with serious conditions can access more treatment options that work.”

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 would improve the accelerated approval pathway by:

• Establishing a council of senior FDA leadership that would 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.
• Clarifying that real-world evidence may be used to augment or support appropriate post-approval studies.
• 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 would bolster trials for rare diseases.

The legislation is supported by National Organization of Rare Diseases, Juvenile Diabetes Research Foundation, American Cancer Society Cancer Action Network, Parent Project Muscular Dystrophy, EveryLife Foundation for Rare Diseases, National MS Society, American Academy of Neurology, Haystack Project.

Click HERE to read the text of the legislation.

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