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  • Feature ArticlesFeature Articles

    Regulatory Focus, July issue: Cell and gene therapy

    Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational tria...
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    Regulation of advanced therapy medicinal products in the EU

    This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies...
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    Update on RMAT designations

    This article discusses the scope and purpose of the special designation for regenerative medicine advanced therapies (RMAT) created by the passage of the 21st Century Cures Act. The authors explain the benefits expected to be realized with RMAT and provide a tally of products receiving the special designation to date and a current count, by year, of products for which RMAT designation has been requested.   Introduction Section 3033 of the 21st Century Cures Act, titl...
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    Not Quite a Breakthrough Device, FDA Introduces New Safer Technologies Program

    The US Food and Drug Administration (FDA) on Wednesday released draft guidance to introduce a new, voluntary program for certain medical devices and device-led combination products that are expected to improve the safety of currently available treatments or diagnostics but are used for morbidities and mortalities less serious than those eligible for the Breakthrough Devices Program. Devices that may be included in this Safer Technologies Program” or “STeP” may include t...
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    FDA-EMA Workshop Discusses Quality Challenges for PRIME, Breakthrough Therapies

    Last November, officials from the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) met with industry representatives in London to discuss the various quality challenges that arise when the development of investigational products is accelerated. EMA’s launch of its priority medicines (PRIME) scheme and FDA’s breakthrough therapy designation have enabled companies to speed certain treatments to market, although there have been difficulties in ...
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    CMS Plots to Increase Breakthrough Device Payments

    The Centers for Medicare and Medicaid Services (CMS) late Tuesday proposed a new rule that would increase payments for medical devices designated by the US Food and Drug Administration (FDA) as breakthrough devices. CMS explains how at the time of approval for these devices with a breakthrough designation, real-world data on outcomes in different patient populations is often limited. So, it can be challenging for device firms to meet the requirement for demonstrating a ...
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    HMA Offers Recommendations on Complex Clinical Trials

    A subgroup of the EU’s Heads of Medicines Agencies (HMA) issued new recommendations Friday on conducting complex clinical trials.   The HMA’s Clinical Trials Facilitation Group (CTFG)—coordinated by the Danish Medicines Agency—identified eight key recommendations to establish a framework on the initiation and conduct of complex trials for the development of personalized medicine. They were prepared “in recognition of the fact that the development of personalized medici...
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    510(k) Modernization: FDA Floats Need to Pursue New Authority

    Plans at the US Food and Drug Administration (FDA) to “modernize” the most commonly used pathway for new medical device clearances “might require new authority,” FDA leadership said on Monday. FDA Commissioner Scott Gottlieb and Jeff Shuren, director of FDA’s Center for Devices and Radiological Health (CDRH), issued a joint statement to announce 2018 progress on novel device approvals, pointing out that the breakthrough 510(k) is one of two submission types that was new...
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    Industry Calls for Metrics on CDRH’s Breakthrough Device Program

    A consensus of industry participants at a US Food and Drug Administration (FDA) webinar on Thursday pressed for metrics on the medical device priority review program run by FDA’s Center for Devices and Radiological Health (CDRH). The webinar led by Maureen Dreher, policy analyst at CDRH’s Office of Device Evaluation, underscored the need for additional transparency around the Breakthrough Device Program. This request was supported across several participants during the ...
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    Gottlieb, Marks Detail Plans to Advance Development of Cell and Gene Therapies

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks on Tuesday detailed plans for the agency to keep pace with an expected influx of applications for cell and gene therapies over the coming years.   "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications,...
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    FDA Finalizes Breakthrough Device Guidance, Outlines Safer Technologies Program

    The US Food and Drug Administration (FDA) on Tuesday finalized guidance detailing its breakthrough devices program and announced plans to bring safer medical device alternatives to market via a new safer technologies program (STeP).   “We continue to encourage device manufacturers to consider the breakthrough devices program for innovative, lifesaving devices, and, in the near future, our goal is to have STeP as an option for those devices with new advances and adaptat...
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    TGA Proposes Regulation on IVD Companion Diagnostics

    Australia’s Therapeutic Goods Administration (TGA) issued a consultation paper on Tuesday to propose a new framework for the regulation of companion in vitro diagnostic (IVD) devices. The framework proposes 10 different modifications, subject to ministerial approval, to the current regulation on the assays, otherwise known as IVD companion diagnostics (CDx). TGA argues the modifications are needed to address existing “concerns with the current regulatory model, part...