• Regulatory NewsRegulatory News

    News in Focus: PDUFA VII is a step in the right direction for the rare disease community

    Craig Martin is chief executive officer of Global Genes, a nonprofit organization whose mission is to increase public awareness for rare disease and globally connect, empower and educate individuals and rare disease foundations in person, print and online. More than 400 million people worldwide are affected by 7,000+ rare diseases. In the U.S., around 1 in 10 people (more than 33 million Americans) have a rare condition. More than half of rare disease patients are chi...
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    EMA begins rolling review of Sanofi Pasteur COVID vaccine

    The European Medicines Agency (EMA) has taken a fifth COVID-19 vaccine candidate into its rolling review scheme, evaluating data as they become available to speed a regulatory decision amid the ongoing public health emergency of the COVID-19 pandemic.   EMA’s decision to evaluate Sanofi Pasteur’s Vicprevtyn vaccine by rolling review is based on non-clinical data and early clinical studies “which suggest that the vaccine triggers the production of antibodies that target...
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    FDA panel split on approach to COVID-19 vaccines for younger children

    Members of the US Food and Drug Administration’s (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) disagreed as to whether the agency should grant emergency use authorizations (EUAs) for COVID-19 vaccines for children at a 10 June meeting.   Some said that EUAs were necessary to get the pediatric population vaccinated quickly to achieve herd immunity as the country emerges from the pandemic and that these vaccines should be available soon as th...
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    FDA approves aducanumab for use in Alzheimer’s disease

    The U.S. Food and Drug Administration (FDA) on Monday announced the approval of Biogen’s human monoclonal antibody Aduhelm (aducanumab) for use in patients with Alzheimer’s disease under the agency’s accelerated approval pathway.   FDA’s approval of aducanumab is based on results from two Phase 3 clinical trials of patients with early Alzheimer’s disease (AD), EMERGE and ENGAGE . Both trials were stopped in March 2019 due to apparent futility by a data safety monito...
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    FDA clarifies deferral policy on pediatric studies for new cancer drugs

    Final guidance from the US Food and Drug Administration (FDA) specifies when sponsors must conduct pediatric studies for new cancer drugs as well as when those requirements can be waived or deferred.   Sponsors are required to evaluate these oncology drugs in pediatric populations for applications submitted on or after 18 August 2020, under changes made by the FDA Reauthorization Act of 2017 (FDARA).   The guidance addresses molecularly targeted oncology drugs for ...
  • 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...
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    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...