• Regulatory NewsRegulatory News

    Pediatric Priority Review Voucher Program Set to End After FDA Approves New Drug

    The US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program is slated to end in one year, after the agency awarded its third-ever rare pediatric voucher, thereby triggering a little-known provision in the voucher program. Background Regular readers of Regulatory Focus will be familiar with FDA's priority review voucher programs. We've written about them extensively over the years, and the vouchers are the focus of our most rec...
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    With New Data in Hand, Could Sanofi and Regeneron Use a Regulatory Shortcut?

    A new drug being co-developed by drugmakers Sanofi and Regeneron could, according to new data , dramatically decrease low-density lipoprotein (LDL) cholesterol in patients and lessen cardiac events. But it's a reduction in something else that could be most important for Sanofi and Regeneron: the time it might take the US Food and Drug Administration (FDA) to review the new drug, Praluent. Background In July 2014, Sanofi and Regeneron announced they had purchased what...
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    For Second Time Ever, FDA Awards Special Voucher Meant to Accelerate Drug Reviews

    Drugmaker United Therapeutics has become just the second company in US history to obtain a new type of voucher which allows a company to potentially get its drug approved by the US Food and Drug Administration (FDA) in 40% less time than it normally takes. Background The voucher in question is known as a rare pediatric disease priority review voucher (PRV), and is modeled off a similar program intended to help spur the development of new drugs for so-called "neglected" ...
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    Rare Disease Patient Group Commends EMA, Wants EU-Wide Access to Treatment

    A rare disease advocacy network has applauded the European Medicines Agency’s (EMA) efforts to incentivize the development of treatments for rare diseases , but says  the variance in treatment access across the EU is “unacceptable.” In an interview with EurActiv , Terkel Andersen, president of the advocacy group EURODIS, said that EMA and the European Commission (EC) are “doing their utmost to try to make rare diseases ‘attractive’ for the pharmaceutical industry.” ...
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    Orphan Drugs in the EU: A Record-Breaking Year

    The European Medicines Agency (EMA) recommended 17 drugs with orphan product designation for approval in 2014, the most ever in a single year. This is much higher than the average of 6.75 per year between 2010 and 2013 and is a significant increase from 2013, when EMA recommended 11 products with orphan designation for approval. Background: Orphan Designation Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141/2000 . The reg...
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    Applying Ethics to Conflicting Healthcare Philosophies: The Ebola Example and the RAPS Ethics Code

    The Ebola crisis has brought more than anxiety to the US; it has brought controversy in the form of conflicting treatment recommendations for individuals returning from African countries affected by hemorrhagic fever disease. How does a regulatory professional determine how to respond in the face of conflicting philosophies on best practice for developing a treatment or prevention protocol? This article looks at the Regulatory Affairs Professionals Society (RAPS) Co...
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    FDA Explains How its New Incentive Program for Rare Pediatric Disease Treatments Works

    The US Food and Drug Administration (FDA) has released a long-anticipated guidance document intended to explain how a new incentive program known as the Rare Pediatric Disease Priority Review Voucher system works. Background Created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA) , the Rare Pediatric Disease Priority Review Voucher is modeled closely off a similar program known as the Tropical Disease Priority Review Voucher system. T...
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    Leprosy––Not Yet Consigned to History

    The word leprosy brings to mind a gruesome disease that separates its sufferers from society and continues to strike fear into communities, much like Ebola. While the genome for leprosy has been sequenced and there is a drug cocktail that effectively cures the disease, its mode of transmission still is unknown. A disease that dates back through millennia, leprosy so far has eluded efforts to find the final piece of information that might eradicate it from the world per...
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    Orphan Drugs Around the World: The Regulations and Requirements You Need to Know

    "Orphan drugs" are product intended for use in the treatment of a "rare" disease—those diseases that occur infrequently or rarely in the general population of a specific location.  Rare diseases affect a limited number of people out of the whole population, and the definition varies on a country-by-country basis; it is country-specific and population-dependent.  It is estimated that between 5,000 and 8,000 (typically averaged to 7,000) distinct rare diseases exist today,...
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    First Pediatric Priority Review Voucher Goes up for Sale, Fetching $67M

    As far as regulatory incentives go, some are pursued more than others. The US Food and Drug Administration's (FDA) orphan drug exclusivity provisions, for example, have brought about a renaissance of therapies meant to treat patients suffering from rare diseases and conditions. But on the other side of the coin are incentives not often sought out by companies. And perhaps no incentive maintained by FDA is used less than its priority review vouchers. Background: Vouchers...
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    Head of FDA's Rare Disease Division Departs, Leaving Agency Looking for New Leader

    The US Food and Drug Administration (FDA) is looking for a new leader for its Rare Diseases Program. In an email to staff on 16 July 2014, John Jenkins, director of the Office of New Drugs (OND), which oversees the rare disease program, said the program's current leader would be transitioning to a new role at FDA. Anne Pariser, OND associate director for rare diseases, will take on a new position in the Office of Translational Sciences, Jenkins said. Pariser will still ...
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    FDA Planning Five Guidance Documents to Help Children With Rare Diseases

    • 10 July 2014
    The US Food and Drug Administration (FDA) has released a new report indicating four core ways it plans to accelerate the development of treatments for pediatric rare diseases (PRD). The report, Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases , was required under Section 510 of the Food and Drug Administration Safety and Innovation Act ( FDASIA ) and based off a...