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    CAR T cell product development guidance: Comments ask for CMC details

    Both industry and clinicians asked the US Food and Drug Administration (FDA) for clarification related to the evaluation of cellular starting materials in its draft guidance on chimeric antigen receptor (CAR) T cell product development.   The public comments also sought more details on change management and how the guidance applies to other genetically modified products.   The draft guidance , issued on 15 March 2022, provides recommendations on chemistry, manufac...
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    FDA to form task force, public-private partnership on rare neurodegenerative diseases

    The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.   “The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “This action plan, especially including ...
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    RWD in clinical trials: External control arms take the lead

    The US Food and Drug Administration (FDA) has now published four draft guidances spelling out how it will consider real-world data (RWD) in regulatory decision-making, from the use of electronic health records and registries to RWD in non-interventional studies. More guidance documents are planned, including details on study designs that incorporate RWD for external control arms.   External control arms, which use data collected outside of the current trial to provide ...
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    CBER outlines recognition process for regenerative medicine therapy standards

    The US Food and Drug Administration (FDA) outlined its process for recognizing voluntary consensus standards (VCS) related to regenerative medicine therapies in a new draft guidance document. The draft guidance , issued 15 June 2022 by the Center for Biologics Evaluation and Research (CBER), outlines how VCS bodies and standards will be evaluated by agency staff, where these standards can be accessed, and how individuals can request recognition of a specific VCS. The g...
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    Clinical trial diversity: Sponsors seek clarity on data sources, timing

    Drugmakers and clinician organizations praised the US Food and Drug Administration’s (FDA) recent draft guidance containing recommendations on developing a Race and Ethnicity Diversity Plan to improve enrollment of underrepresented racial and ethnic groups in clinical trials. But in public comments, they also called for greater details on the types of data that should be used to assess adequate enrollment of these populations, how these diversity plans should be applied to...
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    Device experts criticize MDUFA V compromise, call for postmarket funding

    The compromise proposal to reauthorize the Medical Device User Fee Amendments (MDUFA V) could put stress on the US Food and Drug Administration’s (FDA) ability to recruit and retain expert personnel and fails to fund critical postmarketing surveillance programs, four medical device policy experts wrote in the New England Journal of Medicine .   The final MDUFA V proposal, which was released on 22 March 2022 after a two-month delay, faces a deadline of 30 September 2...
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    FDA issues Q&A on importation of prescription drugs from Canada

    The US Food and Drug Administration (FDA) has offered some clarity on the legal requirements for small entities to import prescription drugs from Canada.   In final guidance , issued on 25 May 2022, the agency provided 12 questions and answers aimed at providing a plain language guide to implementation of the Importation of Prescription Drugs final rule, which became effective on 30 November 2020. That regulation was aimed at reducing prescription drug costs by impl...
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    Study: Inhaler manufacturers use patents, exclusivity to shut out generic competition

    Brand-name inhaler manufacturers have successfully used patent hops, cross-listed patents, and other nonpatent regulatory exclusivities to keep generic manufacturers out of the US inhaler market over the last 35 years, according to an analysis published in Health Affairs . The result has been higher costs for patients and a lack of innovation, according to the study’s lead author William B. Feldman of Brigham and Women’s Hospital and Harvard Medical School, both in Bos...
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    FDA works to advance real-world data collection in pregnancy and lactation

    The US Food and Drug Administration (FDA) is working on ways to facilitate greater use of real-world data (RWD) to help understand the safety and efficacy of medications used during pregnancy and lactation.   Despite the widespread use of prescription and over-the-counter medications by pregnant and lactating women, there is often little or incomplete information to guide decisions about starting and stopping medications or changing doses.   “Although the lack of s...
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    FDA offers insight into product quality assessment principles

    The US Food and Drug Administration (FDA) has issued draft guidance outlining its benefit-risk principles when evaluating product quality assessments for new drug applications (NDA), biologics license applications (BLAs), and supplements.   The draft guidance , released on 10 May, provides details on possible mitigation strategies for quality issues and the circumstances under which unresolved quality issues could be addressed during a public health emergency or wides...
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    Posters highlight research, regulatory updates at Euro Convergence 2022

    This year’s RAPS Euro Convergence features research and updates on the use of new technology and regulatory tools as part of the conference’s poster session.   Featured posters include an analysis of the regulatory challenges for products with artificial intelligence/machine learning, the best use of regulatory intelligence and automation, evolution in medical device labeling technology, implications of reforms to China’s National Medical Products Administration (NMPA)...
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    FDA sheds light on conducting human radiolabeled mass balance studies

    The US Food and Drug Administration (FDA) has issued draft guidance outlining the clinical pharmacology considerations for human radiolabeled mass balance studies for investigational drugs, including when to conduct the study, the study design, and what elements to include in the study report.   The draft guidance , issued on 5 May, does not cover animal mass balance studies, safety testing of drug metabolites or recommendations for selecting the radioactive dose. (RE...