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    Priority Review Voucher Fees to Decline in FY 2017

    The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year. Tropical Disease Priority Review Voucher User Fee Year Voucher Fee FY 2011 $4,582,000 FY 2012 $5,280,000 FY 2013 $3...
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    Industry Weighs in on FDA Draft Guidance on Infectious Disease NGS-Based Diagnostics

    Diagnostic companies Illumina, Roche, AstraZeneca and others are offering a peek inside how they view next-generation sequencing (NGS) diagnostic devices, with the release of 20 comments on recent US Food and Drug Administration (FDA) draft guidance. "Next generation sequencing has the capability to replace previous methods with a single approach to accomplish what might have required several different tests in the past," FDA explains. "In contrast to human sequencing di...
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    EMA and FDA Set Up New Working Group on Rare Diseases

    The new collaboration between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) announced Monday will improve the sharing of information on various aspects of the development and scientific evaluation of medicines for rare diseases. According to EMA, rare diseases affect 30 million people in the EU and approximately the same number in the US, though each disease individually concerns a limited number of patients. And since only a limited n...
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    EMA Official: Data Sharing and Collaboration Can Fill Scientific Gaps in Alzheimer's

    Speaking at a panel at RAPS' 2016 Convergence on Monday, Enrica Alteri, head of human medicines research and development support at the European Medicines Agency (EMA) called on drugmakers to share data to close the gaps in Alzheimer's disease (AD) research. According to Alteri, doing so could help the scientific community not only learn from past failures in Alzheimer's research, but enable the development and validation of new targets. Scientific Gaps "There are gaps...
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    European Regulatory Roundup: EMA Begins Review of First Herceptin Biosimilar (1 September 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EUnetHTA Nears Start of Third Phase of HTA Cooperation Agenda The European network for Health Technology Assessment (EUnetHTA) is on the cusp of entering the third phase of its agenda. Members of the European Commission and the Dutch National Health Care Institute began a 30-day countdown to the official start of the program by signing a grant agreement.  When the of...
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    FDA Issues Form 483 for Alexion’s Rhode Island Site

    Rare disease drugmaker Alexion disclosed late Monday in an SEC filing that the US Food and Drug Administration (FDA) recently completed a routine current Good Manufacturing Practice (GMP) inspection of the company’s Smithfield, Rhode Island manufacturing facility and issued a Form 483 with three observations. “These observations are inspectional, and do not represent a final FDA determination of compliance. The observations pertain to: completion and closure of certain ...
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    European Regulatory Roundup: EMA to Revise Trial Guidance on Hemophilia, Crohn’s, Ulcerative Colitis, Diabetes and Bipolar Disorder (4 August 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Boom in Hemophilia R&D Prompts EMA to act to Prevent Enrollment Bottleneck The European Medicines Agency (EMA) is planning to revise its guideline on development of drugs to treat hemophilia to cut the risk of patient enrollment bottlenecks slowing progress. EMA began the process after becoming concerned that the minimum data requirements of its current guideline are i...
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    European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation

    The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress. Fifteen years after the implementation of the orphan legislation, the commission says it is currently launching initiatives to improve the implementation of the regulatory framework with a view to ensuring timely access to medicinal products.  In this context, the Comm...
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    House Looks to Reform Tropical Disease Priority Review Voucher Program

    The House Energy and Commerce Committee on Wednesday passed by voice vote an amendment (to an unrelated bill on hospital preparedness for dangerous threats) that would significantly change the priority review voucher (PRV) program for tropical diseases. Background Since 2009, the US Food and Drug Administration (FDA) has issued nine PRVs to reward companies for developing new drugs to treat rare pediatric and tropical diseases with a voucher that can be sold on the op...
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    Researchers Urge Caution on Expanding Priority Review Voucher Programs

    As Congress looks to further expand current priority review voucher (PRV) programs (and even create entirely new ones), researchers are now cautioning that the worth of the vouchers could decline significantly and diminish incentives to develop drugs for neglected diseases. Background Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers to pharmaceutical and biotech companies developing drugs and biologics fo...
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    Regulators, Industry Weigh in on Proposed EU Orphan Regulation Changes

    As the number of orphan drug approvals continues to rise in the EU, the European Commission is now considering feedback from European governments, regulators and industry on how changes to five aspects of the Orphan Regulation may further incentivize the development of drugs for rare diseases. To date, the commission has authorized 117 orphan medicines, 82% of which consist of new active substances, according to a new report .  And while the number of products authorize...
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    Neurological Device Trials: FDA Offers Draft Guidance

    The US Food and Drug Administration (FDA) on Friday released draft guidance to assist sponsors who intend to submit an investigational device exemption (IDE) to conduct clinical trials on medical devices targeting neurological disease progression, including for Alzheimer’s disease, Parkinson’s disease or primary dystonia. “Medical devices intended to slow, stop, or reverse the effects of neurological disease face challenges with regard to collecting safety and efficacy d...