• 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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    Stakeholders seek clarity on FDA cell and gene therapy draft guidance

    Industry, medical societies and other stakeholders have weighed in on draft guidance from the US Food and Drug Administration (FDA) detailing how sponsors that want to study multiple versions of cell and gene therapies could combine them in a single “umbrella” trial.   The draft guidance, released in September 2021, outlined FDA’s proposed thinking for how this process would work. For cell and gene therapies intended to treat a single disease, sponsors would typically ...
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    Digital health tech in clinical investigations: FDA issues draft guidance

    The US Food and Drug Administration, as part of an end-of-year spate of guidance documents, has issued a draft guidance for sponsors who wish to use digital tech to aid in remote clinical data acquisition.   Digital health technologies (DHTs) stand to benefit clinical investigations in a multitude of ways, but regulatory considerations have to be weighed as the technologies are employed, noted the agency in setting out the background for the draft guidance, issued in l...
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    Pediatric drug development: Increasing success and overcoming obstacles

    This article discusses pediatric drug development in the EU and US, with an emphasis on overcoming regulatory obstacles. The authors present the commonalities and differences between US and EU regulations and guidelines and examine the legislative acts and subsequent regulatory requirements, while outlining the necessary steps for successful implementation of pediatric drug clinical trials. They conclude with a discussion of three case studies that highlight strategic init...
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    FDA expands eSTAR filing to de novo devices, IVDs

    US medical device and in vitro diagnostic sponsors who are using the Food and Drug Administration’s de novo pathway will be able to use the eSTAR filing format at the beginning of 2022. A pilot program for the eSTAR template that kicked off in 2020 began with a template for 510(k) device submissions.   Nine companies participated in that pilot program that trialed the interactive, PDF-based filing format. Use of eSTAR was expanded in September 2020 to include all 5...
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    Smiths Medical warned by FDA for multiple violations

    The US Food and Drug Administration (FDA) slapped Smiths Medical in Minneapolis, MN with a warning letter for inadequate procedures for handling medical device reports (MDRs), faulty handling of complaints, inadequate validation processes, and lax corrective and preventative actions (CAPA) procedures. The company makes blood warmers and infusion pumps.   The warning letter was issued on 1 October and posted on 23 November. It follows a recent recall of the company’s ...
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    FDA inspection turns up more problems for Philips Respironics

    Hundreds of thousands of complaints from users of Philips Respironics’ breathing assistance devices have been inadequately addressed by the firm, according to recent information released by the US Food and Drug Administration (FDA). The release of an inspection report and FDA’s accompanying statement provides more detail on what Philips knew, and what the firm did – or did not do – to remedy known problems with degradation of foam components in its breathing devices.   ...
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    FDA could do more to enforce ClinicalTrials.gov reporting requirements

    The US Food and Drug Administration (FDA) hasn’t been notifying most clinical trial sponsors they are in violation of reporting requirements.   That’s according to results of a Freedom of Information Act (FOIA) investigation by Reshma Ramachandran, of the Yale School of Medicine in New Haven, CT, and colleagues in a recent Viewpoint published in JAMA .   In January 2017, a final rule issued by the National Institutes of Health (NIH) determining ClinicalTrials.go...
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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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    Friends of Cancer Research urges early dose-finding studies to reduce treatment toxicity

    Friends of Cancer Research is calling on all stakeholders in the cancer community to move away from the idea of “more is better” in drug dosing and begin to incorporate dose-finding studies into the cancer trial process.   Dose optimization is the focus of a new white paper that the group released at its 25 th annual meeting. The paper offers recommendations for performing adequate dose optimization studies in oncology, with a focus on the pre-market setting. “These...
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    Strategies for pediatric clinical trials and drug development

    This article discusses the considerations for designing and conducting pediatric clinical trials from a pediatrician’s perspective. The author covers pediatric subpopulations, issues related to age, endpoints, and pharmacokinetic considerations, while presenting practical solutions and ethical considerations. Patient and family‒centric approach to studies and engagement with patient advocacy groups are also discussed. The author concludes with some recommendations about pe...
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    FDA issues draft guidance for device software in premarket submissions

    The US Food and Drug Administration (FDA) has released draft guidance for sponsors outlining its thinking about the documentation needed to support the agency’s evaluation of device software functions for premarket submissions.   The agency said the guidance recognizes the “rapidly evolving nature of digital health and recent FDA recognized consensus standards related to software” and, when finalized, will serve as an update to the Guidance for the Content of Premark...