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  • Regulatory NewsRegulatory News

    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...
  • 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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    Rare Disease Clinical Trials: FDA Awards $15M in Grants

    After receiving 89 clinical trial grant applications, the US Food and Drug Administration (FDA) on Tuesday selected 12 projects that will be funded with more than $15 million over the next four years.   The announcement of the new funding comes as approximately 30 million Americans are affected by 7,000 known rare diseases, but only a few hundred treatments exist for these rare diseases.  Two-thirds of the 2019 grants will go to studies affiliated with universities,...
  • Regulatory NewsRegulatory News

    Rare Pediatric Disease PRVs: FDA Updates Guidance

    The US Food and Drug Administration (FDA) on Monday released revised draft guidance on rare pediatric disease priority review vouchers (PRVs), which are awarded to companies developing certain rare pediatric disease treatments and can then be sold. From 2014 to today, FDA has issued 20 rare pediatric disease PRVs, with one selling for $350 million to AbbVie in 2015 and more recently selling for between $80 million and $130 million. The revisions to FDA’s guidance i...
  • Regulatory NewsRegulatory News

    Patient Groups, Industry Seek Changes to Rare Disease Drug Guidance

    Patient groups, drugmakers and other organizations are calling for changes to the US Food and Drug Administration’s (FDA) recently revised draft guidance on developing drugs to treat rare diseases.   The 24-page draft guidance was revised last February after first being published in 2015. The revision included updates to the agency’s approach to natural history studies, a discussion of issues for evaluating biomarkers for consideration as surrogate endpoints and a ne...
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    European Regulatory Roundup: Critical Moment for EMA (15 December 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Commission Creates Roadmap for Evaluation of Pediatric Rare Disease Legislation The European Commission has created a roadmap for evaluating legislation covering pediatric rare diseases. Officials are embarking on the nearly two-year evaluation to understand why regulations on pediatric and orphan medicines have failed to translate into the hoped-for medical advances. ...
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    Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss

    The priority review voucher (PRV) programs, created by Congress with an eye to incentivizing the development of new rare pediatric and neglected tropical disease drugs, have so far rewarded a wide range of small and large biopharma companies and products, though whether PRVs have actually spurred new research remains a question. Andrew Robertson, head of regulatory science and policy at Sanofi, and Rachel Cohen, regional executive director for the Drugs for Neglect...
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    More Efficient Rare Pediatric Drug Development: FDA Drafts Guidance

    The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could help speed the development of treatments for rare pediatric disease drugs. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, said the draft guidance "could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful...
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    FDA Finalizes Chagas Disease Guidance for Blood Collection Establishments

    The US Food and Drug Administration (FDA) on Tuesday finalized guidance on the use of serological tests to reduce the risk of transmission of T. cruzi , a parasite that causes Chagas disease, in blood and blood components. This guidance supersedes 2010 guidance and finalizes a draft from 2016 that amended the 2010 guidance by expanding its scope to include the collection of blood and blood components for use in manufacturing a product, including donations intende...
  • Feature ArticlesFeature Articles

    Shaking Palsy, aka Parkinson's Disease, is Still a Mystery

    This article discusses disease etiology, history and symptoms of Parkinson's disease as well as ongoing research and new treatment options. Introduction There are hundreds of diseases named after a person. These are called "eponymous diseases," and a small sample includes Crohn's disease, Salmonellosis, Huntington's disease, Hodgkin's lymphoma, Lou Gehrig's disease, Tourette's syndrome and the well-known Alzheimer's disease and Parkinson's Disease (PD). PD is one of...
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    FDA Issues Disease-Specific Draft Guidance for Pediatric GERD

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance detailing clinical considerations for drugmakers looking to develop products to treat pediatric gastroesophageal reflux disease (GERD). FDA says the draft is meant to be a starting point for discussions between the agency, drugmakers, academia and the public and sets out the agency's current thinking on potential study populations, endpoints and clinical pharmacology issues related to dosing. Sp...