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    FDA to form task force, public-private partnership on rare neurodegenerative diseases

    The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.   “The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “This action plan, especially including ...
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    RWD in clinical trials: External control arms take the lead

    The US Food and Drug Administration (FDA) has now published four draft guidances spelling out how it will consider real-world data (RWD) in regulatory decision-making, from the use of electronic health records and registries to RWD in non-interventional studies. More guidance documents are planned, including details on study designs that incorporate RWD for external control arms.   External control arms, which use data collected outside of the current trial to provide ...
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    Orphan drug experts discuss new book on developing rare disease treatments

    Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...
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    New public-private consortium will tackle gene therapies for rare diseases

    Two federal agencies are partnering with pharmaceutical companies and non-profit organizations to form a consortium aimed at boosting the development of gene therapies for rare diseases.   In a Wednesday announcement, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) revealed the launch of the Bespoke Gene Therapy Consortium (BGTC). The consortium’s work will be managed by the Foundation for the NIH and sit within the NIH Acceleratin...
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    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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    EFPIA, others question EC's proposed orphan and pediatric drug development updates

    An initiative by the European Commission (EC) to boost the development of pediatric drugs and medicines for people with rare diseases has received stakeholder feedback that in many cases expresses concern with the proposed upending of the status quo.   A 60-day period of public consultation for the initiative’s roadmap closed at the end of July; in all, 112 comments were received from individuals, non-governmental organizations (NGOs), public authorities, medical socie...
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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 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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    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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    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...
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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...