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    FDA launches online orphan submissions portal

    The US Food and Drug Administration (FDA) on Monday launched a new cloud-based submission portal for orphan drug designation requests, shifting away from the current paper and CD-based submission process. The new portal, first announced in January, is part of the agency’s orphan drug technology modernization effort and fits in with its broader technology modernization plans. The shift to electronic submissions follows a decade of increasing orphan drug designation reques...
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    FDA orphan drug updates at NORD Summit

    Officials from the US Food and Drug Administration (FDA) provided updates on the agency’s efforts to support the development of medical products for rare diseases at the National Organization for Rare Disorders (NORD) Summit this week.   In his keynote address on Thursday, FDA Commissioner Stephen Hahn said that FDA had already approved 51 orphan indications through July of this year and acknowledged that the agency’s Office of Orphan Products Development (OOPD) has se...
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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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    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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    Orphan drug incentives reviewed at BIO

    Orphan drug designation has been a fruitful pathway for drug development, but unmet need remains. Whether changes are needed to the legislation, and how orphan drug research can incorporate patient-focused drug development principles and make room for modern-day collaboration were topics for expert discussion at an orphan drug-focused session during BIO Digital 2020.   The future looks bright for drug development for rare diseases, said session moderator Natasha Bonhom...
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    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...
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    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,...
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    FDA to Congress: Pediatric Information Lacking in 36% of Relevant Orphan Drug Labels

    In a report to Congress, the US Food and Drug Administration said that there is a public health need for additional pediatric information in labeling for over one-third of approved orphan indications that are relevant in the pediatric population. FDA research, conducted as part of the FDA Reauthorization Act of 2017  (FDARA), found that of all drugs that were approved for an orphan indication between 1 April 1999 and 31 August 2018, a total of 548 orphan indications we...
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    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...
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    Drugmaker Petitions FDA to Revoke Sublocade Orphan Designation

    A citizen petition filed last week by law firm Goodwin Procter on behalf of Pennsylvania-based drugmaker Braeburn calls on the US Food and Drug Administration (FDA) to revoke the orphan designation granted to Indivior’s opioid use disorder treatment Sublocade (buprenorphine extended-release injection).   “Sublocade is not now, nor was it ever a bona fide orphan drug, particularly since more than two million Americans currently are afflicted by opioid addiction,” the pe...
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    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...