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Is FDA cracking down on accelerated approval for oncology drugs? Here are our takeaways.

By Lindsey PaulAshley Riley

May 17, 2021

    From April 27-29, FDA's Oncologic Drugs Advisory Committee (ODAC) met to review the status of six checkpoint inhibitor indications granted accelerated approval over the past five years. The meeting was part of FDA's industry-wide evaluation of accelerated approvals in oncology in which confirmatory trials did not confirm clinical benefit. Interestingly, the committee recommended that only two of the six indications be withdrawn from the market—although we're still awaiting the final decision from FDA. Keep reading to learn about the significance of the meeting and what FDA's decision could mean for cancer programs and patients.

    2021 oncology state of the union

    FDA evaluation calls accelerated approvals into question

    Since 1992, FDA's accelerated approval program has allowed treatments for life-threatening conditions that fill an unmet medical need to be approved based on surrogate endpoints aimed at predicting meaningful clinical benefit. Historically, these accelerated approvals have mainly been granted for oncology drugs. To confirm the treatments actually provide clinical benefit, such as increased survival, manufacturers must conduct further confirmatory trials after accelerated approval is granted. FDA can then use the results to determine whether to grant drugs traditional approval or remove them from the market.

    Recent attention from researchers and the media has shed light on shortcomings in manufacturers' completion of confirmatory trials and FDA oversight, leading to FDA's industry-wide evaluation. In the months leading up to the ODAC meeting, Bristol Myers Squibb, AstraZeneca, Merck, and Roche all proactively withdrew indications for their immune checkpoint inhibitors in consultation with FDA after confirmatory trials failed to demonstrate improvements in overall survival. These withdrawals, combined with the ODAC's recommendations, call into question how much pressure FDA will put on manufacturers to complete confirmatory trials and how strict it will be about withdrawing approval from drugs that don't demonstrate meaningful benefit. FDA's treatment of accelerated approvals in the future could have significant implications for cancer programs and patients.

    4 key takeaways for cancer programs

    1. Patient education and shared decision-making are more important than ever for treatment decisions that involve drugs with accelerated approval

    As the recent attention on the accelerated approval process highlights, cancer programs must make sure oncologists have the tools they need to talk with patients about the risks, benefits, and costs of treatments that have not yet demonstrated clinical benefit. They should also support oncologists in engaging patients in conversations about their goals for care and advance care planning. This is especially important because the drugs in question may be the only treatment available for some patients but could also leave them with unwanted side effects and high out-of-pocket costs.

    2. Oncologists and pharmacists need support staying on top of drug approval changes

    As the state of accelerated approvals remains in flux, it will be critical for cancer programs to help oncologists keep up with the changing treatment landscape. This will be even more important if FDA evaluation prompts a wave of accelerated approval withdrawals in the future. Cancer programs should work with their pharmacy teams ensure their health system formularies are regularly updated based on information coming from FDA, with systems in place to clarify which treatments physicians can order and to circulate news of any new guidelines.

    3. The pace of oncology drug development depends on manufacturers' response to FDA scrutiny

    FDA's increased scrutiny of accelerated approvals could make pharmaceutical manufacturers wary of pursuing this route to bring their drugs to market, perhaps slowing the rapid pace of oncology innovation. This could mean cancer programs would have to wait longer to offer patients new and potentially life-saving therapies. But if FDA follows the ODAC's recommendations, manufacturers may see its leniency as permission to continue to push drugs through the accelerated approval process without worrying too much about having to prove clinical benefit later. When it comes to these treatments, cancer programs should remain diligent about tracking clinical evidence to help inform treatment decisions.

    4. FDA evaluation signals growing debate on cancer drug value and pricing

    Although we're still waiting for FDA's final decisions in response to the ODAC's recommendations, these recent events highlight the larger industry conversation surrounding how cancer treatments are valued and priced. Pharmaceutical manufacturers are already facing mounting pressure to justify high drug prices, particularly in oncology. Until now, commercial payers have traditionally followed CMS in covering drugs with accelerated approval status. However, if a drug has failed to meet endpoints in a confirmatory trial, will the fact that it fills an unmet patient need be enough to rationalize a sticker price of hundreds of thousands of dollars? As questions about value, cost, and access continue to arise, cancer programs can provide valuable insight on how these issues play out in the clinical setting and should engage with other industry stakeholders to ensure treatments are affordable for patients. Cancer programs should also take another look at their current prescribing practices and continue to center patients' financial considerations in treatment decisions as changes to the accelerated approval landscape play out.

    Oncology state of the union for 2021


    This presentation will equip oncology leaders with strategic insights on major market forces impacting cancer programs in 2021 and beyond, including:

    • Key market forces impacting oncology
    • Implications of key market forces for cancer programs
    • Action items for adapting oncology strategy to key market forces
    Download now


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