• Regulatory NewsRegulatory News

    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...
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    What's a Rare Disease for Dogs? FDA Proposes Maintaining Current Levels for Major Species

    Drug regulation is in many ways as much about incentives as it is about requirements. Especially for drugs intended for smaller populations, an incentive—a free review voucher or a speedy review time, for example—may be the difference between a drug existing as a concept or as one available to consumers. Background In the US, patients have long benefited from the Orphan Drug Act , which defines a rare disease as one affecting fewer than 200,000 patients in a given year...
  • FDA Guidance: How to Improve Your Meetings With the Office of Orphan Product Development

    A new draft guidance document published by the US Food and Drug Administration (FDA) aims to clarify the processes and procedures of meetings with its Office of Orphan Products Development (OOPD). Background OOPD's mission is to help facilitate the development of drugs, devices, biologics and other medical products to treat patients with rare diseases. Those diseases, as defined by the Orphan Drug Act of 1983, affect fewer than 200,000 patients in the US in any given ...
  • EMA, TGA Launch New 'Collaboration' to Accelerate Access for Orphan Medicines

    EU and Australian regulators have announced that they will begin sharing information about orphan medicines in an attempt to accelerate approval for new drugs intended for rare diseases. Background Orphan medicines are those intended for diseases or conditions which affect a small number of individuals-so-called "rare diseases"-and are thus less likely to have a ready market to incentivize investment in new therapies. The definition of "rare disease" differs throughout...
  • As US Population Grows, Rare Diseases Become Even Rarer

    In 1983, the population of the United States was approximately 233 million people. More than 30 years later , the US population is now closer to 313 million-an increase of 82 million people. But while the increase has been good for many things, it has been perversely disadvantageous for patients with rare diseases. Background In 1983, Congress passed the Orphan Drug Act , a piece of legislation meant to provide incentives for manufacturers to develop therapies to me...
  • FDA Announces $14 Million in Grants for Trials in Support of Rare Disease Therapies

    The US Food and Drug Administration (FDA) is often seen as a reactive entity-reacting, that is, to the information contained within applications in order to render an approval decision, or to postmarketing adverse event data to determine if a product remains safe for use. But as it illustrated in an announcement made on 21 October 2013, it can also be proactive when it needs to be. FDA said it will be issuing 21 grants through its Orphan Products Grants Program with the...
  • OECD Calls for Regulatory Harmonization to Support Global Clinical Trials System

    The Organization for Economic Co-operation and Development (OECD), a long-standing global governance group focused on economic issues, has called for countries around the globe to harmonize their clinical trials regulations in an attempt to save both costs and lives. Background In a statement, OECD said it was troubled by "increasingly complex and inconsistent clinical trial regulations," which it said were causing delays, increasing costs and leading to fewer clinical ...