• Regulatory NewsRegulatory News

    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    The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could help speed the development of treatments for rare pediatric disease drugs. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, said the draft guidance "could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would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...
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    FDA, CMS: Second Parallel Review Decision Ever for NGS Test

    The US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS) on Thursday announced joint approval and coverage decisions for Foundation Medicine's FoundationOne CDx next generation sequencing (NGS) based test. The test was reviewed under FDA and CMS' parallel review program, and is only the second test to be approved and cleared under the program since its inception in 2011, the first being Exact Sciences' Cologuard colorectal cance...
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    As Transparency on Priority Review Vouchers Fades, Prices Level Off

    In the early days of the priority review voucher (PRV) programs, back in 2014 and 2015, companies publicly disclosed who they sold their PRVs to, how much they cost and which products they were eventually used to speed the review of. But now that 17 PRVs have been issued by the US Food and Drug Administration (FDA), news of PRV sales has slowed to a trickle of SEC filings and the rare press release. Three times already in 2017, with GlaxoSmithKline and Teva’s...
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    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 Expands Generic Drug Priority Reviews

    Talk of bringing down the price of pharmaceuticals often hinges on generic competition, and the US is seeing approvals of new generic drugs faster and more consistently than ever – a trend likely to continue. The progress comes as US Food and Drug Administration (FDA) Commissioner Scott Gottlieb on Thursday indicated that the agency will expand which abbreviated new drug applications (ANDA) will see priority reviews. "Earlier this year we made changes to how we pri...
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    Amgen Challenge of FDA Decision on Pediatric Exclusivity Could Have Wider Implications

    Back in May, the US Food and Drug Administration (FDA) denied a six-month extension of market exclusivity for Amgen's blockbuster Sensipar (cinacalcet), resulting in lawsuit filed by the drugmaker. A JAMA viewpoint published Wednesday argues that a decision in Amgen's favor could end up diminishing FDA's ability to encourage clinically meaningful pediatric studies. The case centers on the interpretation of how companies "fairly respond" to FDA's written requests ...
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    FDA Revises Priority ANDA Draft Guidance

    The US Food and Drug Administration (FDA) on Friday issued a revised version of its draft guidance on the information that must be pre-submitted in order to receive a priority review for an abbreviated new drug application (ANDA). As part of the negotiations to reauthorize the Generic Drug User Fee Amendments (GDUFA II), FDA agreed to shorten its review of eligible priority generics by two months if sponsors submit a pre-submission facility correspondence (PFC) at leas...
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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...
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    EU Pediatric Regulation’s 10-Year Anniversary Report Highlights Shortcomings

    Ten years ago, the European Commission created a new regulation as part of an effort to try to encourage more pediatric research. But in the years since, companies have been slow to increase their development of treatments for pediatric populations, particularly in oncology, where many of the medicines used were developed in the 1990s, “if they exist at all,” an EC report released Thursday said. The report highlighted that the number of completed pediatric investig...
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    Breakthrough Devices: FDA Issues Draft Guidance

    The US Food and Drug Administration (FDA) on Tuesday issued a draft guidance detailing the agency's new program for breakthrough medical devices created by the 21st Century Cures Act . The new breakthrough devices program supersedes and combines several of the agency's existing programs to speed access to new devices. "Earlier and more frequent interaction between the FDA and manufacturers … should allow manufacturers to make the best use of their resources to bring st...
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    EMA Revises Reflection Paper on Pediatric Extrapolation

    The European Medicines Agency (EMA) on Friday released an updated draft reflection paper for public consultation detailing the agency's framework for pediatric extrapolation in drug development. EMA defines extrapolation as extending information and conclusions from studies of a particular subgroup (source population), condition or drug to make inferences for another subgroup (target population), condition or drug. By extrapolating data from adult or other pediatric popu...