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  • Feature ArticlesFeature Articles

    September’s Regulatory Focus: Countdown to EU MDR and IVDR

    Feature articles during September focused on aspects of the EU Medical Device Regulation (MDR) and In Vitro Diagnostic Regulation (IVDR), with articles on compliance and manufacturer obligations, and perspectives examining the current status of the regulations. The issue also included the second part of the cell and gene therapy series, and an article on regulatory tools for generic drug companies.   MDR and IVDR, close up Full implementation of MDR is slated for ne...
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    Convergence: EMA close to finalizing guidance for advanced therapies

    The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies. The “Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells” was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to...
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    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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    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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    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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    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...
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    FDA Finalizes Guidance on Targeted Therapies in Rare Genetic Subsets

    The US Food and Drug Administration (FDA) on Monday finalized guidance on developing targeted therapies in rare molecular subsets of diseases.   The 9-page guidance finalizes a draft version released in December 2017 and has been updated to incorporate comments submitted to the public docket and includes minor edits to improve clarity.   “By providing clarity on the regulatory and scientific frameworks for product developers, safe and effective targeted treatment...
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    European Regulatory Roundup: Rise of Cell Therapies Prompts EC to Float Orphan Drug Changes (2 November 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Rise of Cell Therapies Prompts Commission to Float Changes to Orphan Drug Rules The European Commission has proposed changes to its orphan drug regulation. Officials want to make changes to the 17-year-old text to account for the rise of cell therapies and other advanced medicinal products, the nature of which are a poor fit for aspects of the existing regulation. If t...
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    House Reps Seek More Transparency on Novartis-CMS Pricing Deal for Newly Approved CAR-T Therapy

    Rep. Lloyd Doggett (D-TX) and other House Democrats on Wednesday sent a letter to Seema Verma, administrator of the Centers for Medicare & Medicaid Services (CMS), seeking to find out more information about an outcomes-based payment approach for Novartis’ newly approved chimeric antigen receptor T-cell (CAR-T) treatment Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). The outcomes-based pay...
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    'Historic Action': FDA Approves First CAR-T Therapy

    The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...