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  • Regulatory NewsRegulatory News

    Califf, experts eye legislative fix for accelerated approval program

    Regulatory and industry experts agree that completing confirmatory studies quickly is essential to the success of the US Food and Drug Administration’s (FDA) accelerated approval program, with some suggesting that new legislation might be necessary to reliably get results from these trials in a timelier manner.   Robert Califf, head of clinical policy and strategy for Verily and Google Health and the Biden Administration’s nominee to be FDA commissioner, said a legisla...
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    FDA approved more first-in-class drugs, gave more accelerated approvals in 2021

    The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 50 new drug therapies in 2021. While the figure is a slight decrease from 2020 , more drugs were designated as first-in-class and used the accelerated approval pathway than in previous years.   The approvals reported by CDER do not include biologics license applications (BLAs) approved by FDA’s Center for Biologics Evaluation and Research (CBER). In 2021, CBER approved...
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    Exclusive accelerated approval drugs comprise ‘relatively small share’ of Medicare spending

    Policymakers looking to rein in Medicare spending should look beyond the lens of the accelerated approval pathway, as drugs that exclusively fall under this indication make up a small percentage of overall Medicare spending, according to a recent research letter written by Benjamin N. Rome, of the Program on Regulation, Therapeutics, And Law (PORTAL) research group at Harvard Medical School and Brigham & Women’s Hospital, and colleagues.   “Medicare is spending more an...
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    Study: RWD not ready for postapproval prime time

    For drugs and biologics that receive accelerated approval, could information drawn from real-world data sources supplant traditional postapproval clinical trials to confirm that the therapies have benefit? Real-world data (RWD) are not yet robust enough to confirm the benefit of drugs awarded accelerated approval based on surrogate endpoints, answered the authors of a recent study.   “The findings of this cross-sectional study suggest that none of the 50 [Food and Drug...
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    FDA’s accelerated approval program: Is change on the way?

    The U.S. Food and Drug Administration (FDA) launched its accelerated approval program in 1992, offering a pathway to bring life-saving drugs and biologics to the market faster. After nearly 30 years, could substantial reforms be on the way?   At RAPS Convergence 2021, David Dorsey of Janssen R&D, said Congress could choose to make changes to the accelerated approval program through the upcoming reauthorization of the Prescription Drug User Fee Act (PDUFA). Congress has...
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    Pazdur mounts defense of accelerated approval during Cures 2.0 panel

    During a listening session convened by the Friends of Cancer Research (FOCR) with Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) on their nascent Cures 2.0 package, Richard Pazdur, director of the US Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE) defended the agency’s accelerated approval program.   (RELATED: Cures 2.0: Discussion draft signals impact on FDA, creation of ARPA-H , Regulatory Focus 23 June 2021)   Asked by DeGette w...
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    FDA defends Aduhelm's accelerated approval, while others call for reform

    Three papers published as Viewpoints in JAMA Internal Medicine this week highlight the rationale provided by the US Food and Drug Administration (FDA) on the agency’s approval of aducanumab (Aduhelm; Biogen Inc), implications the approval could have for the Medicare program, and a contemplation on the future of FDA’s accelerated approval pathway.   Writing on behalf of FDA, Billy Dunn, MD, director of the Office of Neuroscience with FDA, and colleagues said the agenc...
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    FDA to study disclosure information for accelerated approval products in oncology

    The Office of Prescription Drug Promotion (OPDP) at the US Food and Drug Administration (FDA) wants to know how to better present disclosure information about products with accelerated approval to patients with cancer and their caregivers.   In two studies recently announced in the Federal Register , OPDP is seeking to confirm prior research on how patients interpret disclosure information for a product approved under FDA’s accelerated approval pathway on a direct-to-...
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    FDA calls in adcomm to review six more oncology accelerated approvals

    After drugmakers voluntarily withdrew four indications for their cancer drugs in recent months, the US Food and Drug Administration (FDA) on Thursday said it would convene an advisory committee to review six more oncology indications with accelerated approval after confirmatory trials failed to verify their clinical benefit.   Over the course of three days in April, FDA’s Oncology Drugs Advisory Committee (ODAC) will review six indications for Roche’s Tecentriq (atezol...
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    Industry-wide accelerated approval review yields four withdrawals

    Since late December, four drugmakers have voluntarily withdrawn indications for their cancer drugs amid an industry-wide review of accelerated approvals by the US Food and Drug Administration’s (FDA) Oncology Center of Excellence.   In each of their statements announcing the withdrawals, Bristol Myers Squibb, AstraZeneca, Merck and Roche said the withdrawal decisions were made in consultation with FDA as part of an industry-wide review of accelerated approvals with con...
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    Regulators discuss accelerated approvals, Project Orbis at DIA

    Regulators from the US Food and Drug Administration (FDA), European Medicines Agency (EMA) and Health Canada gave their perspectives on different issues related to accelerated approval pathways in their respective jurisdictions at the DIA Global Annual Meeting.   All three regulators offer their own form of accelerated approval based on less comprehensive clinical data than a traditional marketing approval would require. In the US, that pathway is dubbed accelerated ap...
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    Makena Withdrawal? A Look at the Timeline and Other Accelerated Approvals

    Earlier this week, an FDA advisory committee recommended that the agency withdraw Amag Pharmaceutical’s preterm birth drug Makena, a treatment that brought in more than $1 billion in sales before failing a confirmatory trial. The agency is now faced with the task of whether to take the committee’s vote and pull Makena from the market or allow it to remain until another confirmatory trial can be completed. Keeping Makena on the market would also present a rare situation,...