• Regulatory NewsRegulatory News

    EMA Begins Publishing Reports on Whether Approved Medicines Still Qualify as Orphans

    The European Medicines Agency (EMA) on Wednesday announced it has begun publishing reports detailing its decision-making on whether newly approved drugs to treat rare diseases still qualify for orphan designation when they receive marketing authorization. In the EU, drugs intended to treat diseases affecting fewer than five in 10,000 people in the EU qualify for orphan designation, which provides authorized medicines with ten years marketing exclusivity and reduced fees....
  • European Regulatory Roundup: France Targets 80% Biosimilar Penetration by 2022 (4 January 2018)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news . Ireland Offers to Waive Certain Fees to Ease Impact of Brexit Ireland is offering to waive the fee for certain Brexit-triggered regulatory requests. The offer will allow companies to change their reference member state (RMS) from the United Kingdom to Ireland for free. Officials from the Health Products Regulatory Authority (HPRA) of Ireland made the offer in guides on ...
  • Regulatory NewsRegulatory News

    FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements

    The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions. Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of th...
  • Regulatory NewsRegulatory News

    Senate, House Agree to Cut Orphan Drug Research Credit in Half in Tax Bill

    Members of the House-Senate Conference Committee discussing the GOP's tax overhaul plan on Friday released the final text of the bill, which would reduce the rare disease research tax credits biopharma companies can claim by half. Currently, companies can claim a 50% tax credit for qualified clinical testing expenses incurred in testing certain drugs for rare diseases or conditions, generally referred to as "orphan drugs," affecting fewer than 200,000 persons in th...
  • Regulatory NewsRegulatory News

    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. ...
  • Regulatory NewsRegulatory News

    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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    European Regulatory Roundup: EU Stands Firm on Demand for UK to Pay for EMA’s Exit (7 December 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EU Stands Firm on Demand for UK to Pay for EMA’s London Exit The European Commission has reiterated its demand for the UK to cover the cost of the European Medicines Agency’s (EMA) withdrawal from London. That would mean the UK paying the €400 million ($470 million)-plus bill EMA will face as result of breaching its rental agreement. European officials first stated th...
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    FDA's Frey Reflects on Final Year of PDUFA V

    As the year comes to a close, Patrick Frey, senior advisor to the director of the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), looked back at FDA's performance over the final year of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V) and what's to come in PDUFA VI. Speaking at the FDA/CMS Summit in Washington, D.C. on Tuesday, Frey said that before PDUFA, and during the first four PDUFA programs, FDA was approving ab...
  • 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...
  • 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...
  • Regulatory NewsRegulatory News

    Updated: House Passes GOP Tax Overhaul With Orphan Drug Tax Credit Repeal Included

    The US House of Representatives on Thursday passed a bill to reform the US tax code by a vote of 227 to 205, with all Democrats voting against it and all but 13 Republicans voting for it. The House-passed bill includes several provisions that would be attractive to the biopharma industry, though industry groups BIO and the National Organization of Rare Disorders took issue with a provision to repeal the orphan drug tax credit, which amounts to half of all qualified ...