• Feature ArticlesFeature Articles

    March's Regulatory Focus: Regulatory intelligence

    Feature articles during March focused on regulatory intelligence (RI), including first-hand accounts of setting up a dedicated process for RI monitoring and developing an in-house RI database, as well as an examination of the implications of the US Food and Drug Administration’s (FDA’s) new integrated review template for RI. Also included were articles on the importance a global unique device identification (UDI) system, the FDA’s Bad Ad program, and the importance of writ...
  • Feature ArticlesFeature Articles

    FDA’s new integrated review template and implications for regulatory intelligence

    US Food and Drug Administration (FDA) review summaries, previously referred to as summary basis of approval documents, can serve as invaluable sources of regulatory strategy information, the details of which are not publicly available from any other source. When the FDA announced its plans to use a more streamlined approach to communicating regulatory decisions through an integrated assessment process and review document, it raised concern among those who contribute to and...
  • Regulatory NewsRegulatory News

    Orphan product designation requests climb in the US, remain steady in the EU

    The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.   There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019...
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    FDA Rare Disease Day: Collaboration begins with patients

    A few ingredients are essential to advancing therapies for rare diseases: organized patients, the involvement of academic researchers, and support and flexibility from the US Food and Drug Administration and industry. That’s according to Frank McCormack, MD, professor of medicine at the University of Cincinnati. “It begins with the courage and resolve of patients. The patients had the foresight to organize, and facilitate and fund research, and then they lined up for tr...
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    FDA on Rare Disease Day: New initiatives and an upcoming conference

    On Monday, the US Food and Drug Administration (FDA) announced a series of efforts and a day-long conference centered on addressing the unmet needs of patients with rare diseases. FDA leaders announced the initiatives in recognition of Rare Disease Day, observed on February 28.   A new request for applications (RFA) issued by FDA augments the agency’s Orphan Products Grants Program. The RFA is seeking natural history studies to address unmet needs in rare diseases, l...
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    Stakeholders call for regulatory clarity in rare disease research network

    Stakeholders weighing in on a proposed rare disease clinical trials network called for regulatory clarity, smart use of existing resources, and a move toward harmonized trial standards and assessments.   As part of the launch of the US Food and Drug Administration (FDA)’s Rare Disease Cures Accelerator , the agency asked for stakeholder input on how FDA and other agencies can achieve a more cooperative approach in supporting the drug development pipeline for rare dise...
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    European Commission reviews impact of orphan, pediatric regulations

    The European Commission on Tuesday published the results of a yearslong evaluation of the EU’s orphan and pediatric medicines regulations it says will be used to guide future legislative changes and shape the EU pharmaceutical strategy.   The more than 100-page evaluation reviews the positive impacts and shortcomings of the orphan regulation, Regulation (EC) No 141/2000 , and the pediatric regulation, Regulation (EC) No 1901/2006 , based on external studies and var...
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    FDA officials update on orphan drugs, gene therapies at DIA

    Officials from the US Food and Drug Administration (FDA) discussed the agency’s recent efforts to support the development of products to treat rare diseases during a session at DIA’s Global Annual Meeting on Wednesday.   Orphan and rare pediatric disease designations   While the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.   Janet Maynard, director of F...
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    Experts address clinical challenges for ultra-rare diseases at BIO

    A panel of experts at BIO Digital on Wednesday discussed some of the challenges and regulatory considerations for sponsors developing treatments for ultra-rare diseases where clinical trials could involve a very small number of patients.   When asked where companies should begin their development for extremely rare diseases, Brad Glasscock, global vice president and head of global regulatory affairs at BioMarin said, “First and foremost I think is understanding whether...
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    FDA seeks input on rare disease clinical trials network

    To further the establishment of a rare disease clinical trials network, the US Food and Drug Administration (FDA) is asking for input from the public and a broad array of stakeholders.   In a press release, the agency noted that it is “looking to provide a more cooperative approach” in supporting the drug development pipeline for rare diseases.   The call for information comes as the FDA is launching a Rare Disease Cures Accelerator . This accelerator will establi...
  • ExplainersExplainers
    Regulatory NewsRegulatory News

    Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers

    Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers (PRVs) which allow its recipient to expedite the review of any one of its new drug products. What are these vouchers, why is FDA issuing them and what benefit might they have for society? Find out in our latest Regulatory Explainer on the Priority Review Voucher system. Last updated on 25 February 2020 to include: Vifor Pharma said on 17 February that it p...
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    N-of-1 Trials: FDA Plots Path to Regulation

    The US Food and Drug Administration (FDA) is taking notice of a unique and recent phenomenon where physicians and specialists can create a tailored treatment to help or even cure an individual patient with a rare genetic mutation that could prove fatal. In an editorial published Tuesday in the New England Journal of Medicine , Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, and Peter Marks, director of the agency’s Center for Biologics Evalua...