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What to Know About the FDA Accelerated Approval Draft Guidelines

A hand is holding a marker symbolizing the new guidelines from the FDA on accelerated approvals

Earlier this year the FDA released new draft guidelines for their accelerated approval process that expedite’s a drug’s entry into the US market. This is one of four programs the FDA uses to speed up the drug approval process. The other three include fast track, breakthrough therapy, and priority review. To understand these new draft guidelines we will briefly explain what the accelerated approval process is, why the FDA is creating new guidelines, and what the draft guidelines are recommending.

What Is The FDA Accelerated Approval Process

This process shortens the time to approval for drugs indicated to treat a serious condition and fulfill an unmet medical need based on a surrogate endpoint. Surrogate endpoints are used to determine whether a treatment works. They replace a stronger indicator that would take longer to measure.

While surrogate endpoints are not always a true marker for how well a drug or treatment might be working, they can be used to predict clinical benefit. These clinical benefit predictions are used by the FDA to approve drugs in less time. Although these approvals are seen in various therapeutic areas, oncology is typically the predominant category where these take place.

Even if the drug company receives accelerated approval for their drug, they are still required to conduct studies to confirm the drug’s anticipated clinical benefit. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants a traditional approval. Otherwise, the accelerated approval is revoked.

Why Is The FDA Creating New Accelerated Approval Guidelines

The new draft guidelines are an attempt to reform the accelerated approval process program which has received criticism over the last few years due to:

  • A number of accelerated approval drugs having their approval withdrawn by the FDA after confirmatory studies failed to verify clinical benefit, and

  • Significant delays in sponsors completing the required confirmatory studies

With this in mind the FDA drafted these recommendations aimed at continuing the accelerated approval program while maintaining the quality of clinical trials investigating new therapeutics, particularly in oncology.

What The New FDA Draft Guidelines Recommend

Some of the specific recommendations from the FDA in their draft accelerated approval guidelines are:

  • Emphasizing the value of using randomized controlled trials (RCTs) in lieu of the single-arm design that has commonly supported accelerated approvals in the past

    • RCTs are advantageous as they typically use a comparator drug to assess safety and efficacy data rather than historical trials of other therapies

    • The new draft guidance still permits single-arm trials to be used for accelerated approval filings, but caveats that only for specific situations such as when a RCT design is not feasible. In these cases, the FDA recommends the sponsor hold discussions with the FDA before and during conducting the trial

    • Also of note is that RCTs can often be performed in an earlier treatment setting whereas single-arm trials tend to enroll patients who lack other treatment options

  • Suggesting that a two prong RCT approach be designed so that the first trial supporting accelerated approval has an early primary endpoint (such as response rate) and the second, confirmatory trial verifying clinical benefit targets longer-term end points such as progression free survival (PFS) and overall survival (OS)

  • Recommending that the second confirmatory trial should be underway by the time the accelerated approval action is taken, since enrollment will be more challenging if the drug is already commercially available and timely completion is needed to verify that the drug is safe and effective

  • Giving the head’s up that the FDA may request a summary of survival data when considering accelerated approval to assess the impact of the treatment’s toxicities on survival and could also ask for additional updates as the application is reviewed

  • Encouraging the engagement of the FDA early in the development process to gain alignment on study design and execution

  • Calling attention to the role of Project Confirm, an initiative by the FDA Oncology Center of Excellence to promote the transparency of outcomes related to accelerated approvals, including a database tracking the status of all oncology accelerated drug approvals

The FDA’s express preference in the new guidance for RCTs and endpoint selection echoes recent concerns the FDA raised regarding the ability of surrogate endpoints to predict the clinical benefit for oncology drugs. While these draft guidelines came out at the end of March 2023, the FDA allowed for comments and feedback on the draft guidelines through the end of May 2023. The FDA plans to hold a series of workshops this year to discuss the role of surrogate efficacy endpoints, their ability to predict OS, and the information necessary to make a risk–benefit determination for novel oncology drugs.

Though there has yet to be an official date for when the finalized guidelines for accelerated approval will be published, we at SAKS Health are keeping an eye on the developments to not only help inform ourselves, but to also better serve our clients and partners down the line.


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