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    Clinical trial diversity: FDA pushes real-world evidence, diffusion of trial sites

    Real-world evidence, post-marketing studies, and migrating clinical trial sites into underserved communities are all strategies the U.S. Food and Drug Administration (FDA) is pushing to help increase data on the outcomes of minority patients, according to Amy Abernethy, MD, PhD, the agency’s principal deputy commissioner.   “There is real a gap between the makeup of our American public and the representation of our Black community and Black women in clinical trials,” A...
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    Shuren and Stenzel: Bending the arc of testing for COVID – and beyond

    The U.S. Food and Drug Administration’s efforts to rapidly authorize point-of-care and at-home tests during the COVID-19 pandemic could offer a roadmap for a broad effort to bring diagnostic and screening tests to homes and remote locations for a variety of diseases. “The development and authorization of COVID-19 [point-of-care] and at-home tests has been remarkable; a process that can often take more than a year took only a few months in many cases. We believe that les...
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    COVID-19, reorg dominated 2020 for FDA Office of New Drugs

    Staff in the U.S. Food and Drug Administration’s Office of New Drugs (OND) began 2020 with a focus on completing the office’s reorganization, but they quickly shifted focus to the global pandemic and supporting accelerated development of new therapeutics to prevent or treat COVID-19, according to the OND’s inaugural annual report.   “Despite the added workload, there were no significant disruptions to OND’s work as staff managed to meet and/or exceed many of the Prescr...
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    FDA offers real-world evidence examples in device applications

     The US Food and Drug Administration (FDA) is offering a detailed look at how real-world evidence sources – from registries to claims data – can be used to support marketing applications for medical devices.   A new report from the FDA’s Center for Devices and Radiological Health (CDRH) breaks down 90 examples in which real-world evidence was used to support final premarket or postmarket regulatory decisions from Fiscal Year 2012 through 2019. These examples include ...
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    Orphan product designation requests climb in the US, remain steady in the EU

    The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.   There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019...
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    Trials without clinical sites offer chance for improved access, generalizability

    The COVID-19 pandemic has fueled the rise of clinical trials without clinical sites and opened the door to continuing these innovative models after the public health crisis has faded. While these trials offer the potential to expand access and the generalizability of results, there are also some limitations to conducting drug trials in this format.   In a special communication in  JAMA Internal Medicine , Steven R. Cummings, MD, director of the San Francisco Coordina...
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    FDA Rare Disease Day: Collaboration begins with patients

    A few ingredients are essential to advancing therapies for rare diseases: organized patients, the involvement of academic researchers, and support and flexibility from the US Food and Drug Administration and industry. That’s according to Frank McCormack, MD, professor of medicine at the University of Cincinnati. “It begins with the courage and resolve of patients. The patients had the foresight to organize, and facilitate and fund research, and then they lined up for tr...
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    CHMP’s February recommendations: Evrysdi, Jemperli, and a generic for prostate cancer

    The European Medicines Agency’s Committee for Medicinal Products for Human Use (EMA’s CHMP) recommended six new medicines for approval when it met in February, including two biosimilars and one generic medicine.   The committee recommended marketing authorization for Roche’s Evrysdi (risdiplam) for the treatment of certain types of spinal muscular atrophy (SMA). It is the first oral treatment for this condition and was reviewed under the EMA’s accelerated assessment pr...
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    England and Canada diverge from US on cancer drug decisions

    Regulatory and health technology assessment agencies in Canada and England are diverging from the US when it comes to oncology drugs. But they often rely on the same surrogate endpoints to make their decisions, according to two new analyses published in JAMA Internal Medicine .   The first study , led by Avi Cherla, MSc, of the London School of Economics and Political Science, revealed that among 68 cancer indications with accelerated approval in the US that also rec...
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    2020 Drug Safety: CDER spotlights COVID-19 activities, nitrosamine contamination

    COVID-19 was a big safety focus in 2020 for the US Food and Drug Administration (FDA), from the evaluation of drugs for emergency use authorization (EUA) to surveillance for critical drug shortages and medical product supply chain disruptions. The 2020 Drug Safety Priorities report from the FDA’s Center for Drug Evaluation and Research (CDER) chronicles a busy year in safety monitoring and enforcement, including COVID-related activities, nitrosamine impurities, and re...
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    OGD’s 2020 approvals, regulatory work kept pace despite COVID-19

    The US Food and Drug Administration (FDA) approved or tentatively approved 948 generic drug applications in 2020, with a significant portion aimed at potential treatments and supportive therapies for patients with COVID-19, according to the annual report from FDA’s Office of Generic Drugs (OGD).   In response to the pandemic, OGD devised a system for identifying generic drugs with the potential to treat COVID-19 and accelerating those assessments, according to OGD’s ...
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    Analysis: Potentially high NDMA levels after ranitidine ingestion

    A new analysis showed elevated levels of the contaminant N-nitrosodimethylamine (NDMA) after the ingestion of ranitidine capsules across a range of physiologic conditions, with levels exceeding federal guidelines by several orders of magnitude in some conditions.   The study, conducted under conditions that simulate the human gastric environment, bolsters the U.S. Food and Drug Administration’s request in April 2020 for all ranitidine manufacturers to pull their prod...