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    Stakeholders offer input on educational materials for rare disease drug development

    After the US Food and Drug Administration (FDA) solicited comments on an educational program for a new Empower Rare Disease Drug Developers (LEADER 3D) initiative in the Accelerating Rare disease Cures (ARC) program, nearly two dozen stakeholders offered feedback on what topics they believe are important for inclusion in educational materials on rare disease drug development.   FDA’s Center for Drug Evaluation and Research (CDER) asked for comments from rare disease st...
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    Industry requests more information from FDA on dosage and administration labeling

    While the US Food and Drug Administration’s (FDA) latest draft guidance on improving the consistency of information in the dosage and administration section of prescription drug labeling is significantly larger than an earlier guidance issued in 2010, industry stakeholders said they wanted more information from the agency on how labeling of specific cases should be handled.   FDA’s draft guidance on improving development of the dosage and administration section of drug...
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    Study: Certain cancer drugs saw widespread use before accelerated approval withdrawal

    In the period between when three accelerated approval (AA) drugs received approval from the US Food and Drug Administration (FDA) for oncology indications and were later withdrawn due to negative confirmatory trials, more than one quarter of patients with breast, bladder, hepatocellular, gastric, or small cell lung cancer received an AA drug rather than standard of care, according to a research letter recently published in JAMA Oncology .   “Given the growth of withdr...
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    Study: One-fifth of recent FDA approved drugs not authorized, reimbursed in other countries

    More than one-fifth of drugs recently approved by the US Food and Drug Administration (FDA) were not approved or recommended for public reimbursement in other countries, according to a recent study published in JAMA Internal Medicine .   “This cross-sectional study demonstrated that more than one-fifth of the new drugs approved by the FDA in 2017 through 2020 were either refused marketing authorization or not recommended for reimbursement in Australia, Canada, or the ...
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    Study: 10% of FDA approved drugs since 2018 had pivotal trials that missed primary endpoints

    About 10% of drugs approved by the US Food and Drug Administration (FDA) between 2018 and 2021 had pivotal trials with null findings, according to a research letter published in JAMA Internal Medicine .   “Our findings underscore the complexity of regulatory decision-making, as exemplified by evidence of effectiveness despite a null primary end point finding,” James L. Johnston, MD, of the department of medicine at Brigham and Women’s Hospital in Boston, and colleague...
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    Stakeholders want more clarity on FDA's statistical approach to assessing bioequivalence

    Commenters on the US Food and Drug Administration’s (FDA) draft guidance outlining principles for in vivo or in vitro bioequivalence studies (BE) for investigational new drugs (INDs), new drug applications (NDAs), abbreviated new drug applications (ANDAs) indicated that they want the agency to elaborate on new topic areas introduced in the guidance.   The draft guidance, which replaces a 2001 guidance for industry published under the same name, added new informatio...
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    Califf: Current evidence generation system in clinical research needs an overhaul

    Clinical research in the US needs an overhaul to better optimize health outcomes in a way that matches advances in biomedical science, according to a recent perspective authored by US Food and Drug Administration Commissioner Robert M. Califf, published in Clinical Trials journal.   “Biomedical science and technology are undergoing a remarkable period of discovery and development, one driven in large part by the innovations of US scientists and engineers. Unfortunate...
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    A closer look at CDER’s novel drug approvals in 2022

    The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) released a report on the 37 new drug therapies the Center approved in 2022, highlighting which had received expedited development and review status, first cycle approval, and met Prescription Drug User Fee Program Act (PDUFA) VII goal dates for acting on applications.   “We approved many drugs in 2022 for patients with few or no treatment options,” CDER Director Patrizia Cavazzon...
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    CDER approved 37 novel drugs in 2022, its lowest number since 2016

    After several years with high numbers of novel drug approvals, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 37 new drug therapies in 2022.   CDER’s 37 new drug approvals in 2022 represents the lowest number of novel drugs approved by the Center since 2016 . The Center approved 50 novel drugs in 2021 , 53 novel drugs in 2020 , 48 drugs in 2019 and 59 drugs in 2018 . (RELATED: FDA approved more first-in-class...
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    FDA issues draft guidance on pulmonary tuberculosis drugs

    The US Food and Drug Administration (FDA) has released new draft guidance on developing drugs for pulmonary tuberculosis.   FDA’s latest draft guidance is intended to update the agency’s 2013 draft guidance with the same name, which was never finalized. “Since the 2013 final guidance was issued, there have been improvements in nonclinical models and further interest in streamlined clinical development programs as well as consideration for combination regimens with trea...
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    Study: Most new antibiotics approved by FDA based on non-inferiority trials with surrogate outcomes

    All new antibiotics approved by the US Food and Drug Administration (FDA) used surrogate outcome measures, and about half had pivotal trials with a non-inferiority design, according to recent research published in BMJ Medicine .   Mayookha Mitra-Majumdar, MPH, of the Program On Regulation, Therapeutics, And Law (PORTAL) in the Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine at Brigham and Women's Hospital/Harvard Medical School in Boston...
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    FDA releases guidance on compounding beta-lactam products in shortage

    The US Food and Drug Administration (FDA) has released a new guidance with immediate effect outlining its enforcement policy for preparation of certain beta-lactam oral antibiotic suspension products.   The agency said it has been asked to clarify how compounded versions of products from FDA-approved tablets and capsules currently in shortage can be prepared, as FDA’s insanitary conditions guidance states beta-lactam drugs are required to be processed with “complete ...