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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...
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    FDA allows modifications to coagulation monitoring systems for COVID-19

    The US Food and Drug Administration (FDA) is allowing manufacturers to modify coagulation systems for measuring whole blood viscoelastic properties to allow for greater use in hospitals for the supportive management of patients with COVID-19.   The enforcement policy , which was issued in January 2021, is temporary and will remain in effort only for the duration of the COVID-19 public health emergency.   “As hypercoagulability has been observed in patients with CO...
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    PREPP initiative: FDA’s COVID-19 response and the path forward

    The US Food and Drug Administration (FDA) has adapted in its response to the COVID-19 pandemic, from increasing transparency of its risk-benefit analyses for emergency use authorizations (EUAs) to providing guidance on vaccine development, according to a summary report of the agency’s COVID-19 Pandemic Recovery and Preparedness Plan (PREPP) initiative.   The report catalogues the agency’s work during the pandemic, noting that it has published and revised more than 65...
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    FDA offers first thoughts on neurodegenerative disease gene therapies

    The US Food and Drug Administration (FDA) has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released on 5 January 2021, also highlights approval pathways for these novel products.   The draft guidance, which applies to products for both adult and pediatric populations, emphasizes the importance of early communication with FDA before the submission of an investigational new drug...
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    Groups seek clarity on interchangeability in BsUFA III

    Industry groups and biosimilar manufacturers are seeking explicit guidance from the US Food and Drug Administration (FDA) on interchangeable biosimilar products. The comments were made to the agency as part of the reauthorization process for Biosimilar User Fee Act (BsUFA III) program.   The recommendations for improvements in the third iteration of FDA’s biosimilars review program were made as part of a public docket opened by FDA. The agency also recently held a ...
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    FDA: 2 years until SPL E-submission for REMS docs

    The US Food and Drug Administration (FDA) has issued final guidance on the electronic submission of documents related to the Risk Evaluation and Mitigation Strategies (REMS) using the Structured Product Labeling (SPL) format. The final guidance document starts the clock on compliance, with the new formatting requirements taking effect on 28 December 2022.   In the final guidance document, the FDA outlines how it will implement the requirements for e-submission of REM...