• Regulatory NewsRegulatory News

    US Senate Unveils Major Changes to FDA Program in Hopes of Fighting Ebola

    Legislators in the US Senate have unveiled a new piece of legislation meant to incentivize companies to go after the Ebola virus by making critical and long-sought changes to a US Food and Drug Administration (FDA) regulatory program. Background As Focus explained in October 2014 , a recent outbreak of Ebola has led to a scramble for new drugs and vaccines to help treat and prevent the virus. At present, there are no recognized treatments for the disease. Some publi...
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    To Meet Ebola Threat, Father of FDA's Priority Review Voucher System Calls for Reforms

    One of the fathers of a little-known and lesser-used regulatory pathway meant to incentivize the approval of new drugs for so-called "neglected tropical diseases" says Congress needs to include Ebola on the short list of diseases eligible for incentives. Background So called "tropical" diseases are  defined by statute  as infectious diseases which do not affect developed nations and disproportionately affect poor and marginalized nations. Such diseases are typically not...
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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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    Tropical Disease Priority Review Voucher Fee Set to Rise

    The US Food and Drug Administration (FDA) is set to increase the fee it charges users of a special, transferable voucher meant to reward developers of tropical disease treatments with an expedited review process for any subsequent drug product. Background The voucher, known as a tropical disease priority review voucher, was established under the FDA Amendment Act of 2007 (FDAAA) . Under the law, companies that receive approval for a tropical disease treatment are eligi...
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    FDA Voucher for Accelerated Drug Review up for Sale

    • 13 August 2014
    Hot on the heels of the first ever sale of a pediatric review voucher for a whopping $67 million, Canadian pharmaceutical manufacturer Knight Therapeutics announced Tuesday (12 August) that it plans to auction off a similar voucher of its own. FDA currently maintains two priority review voucher systems: one known for tropical diseases, and the other for pediatric rare diseases. Background: Tropical Disease Vouchers The  tropical disease priority review voucher system...
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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 Tries to Invigorate Development of Therapies for 'Neglected' Tropical Diseases

    New guidance released earlier this month by the US Food and Drug Administration (FDA) aims to make it easier for some companies to develop products for so-called "neglected" tropical diseases (NTDs) affecting the developing world. Background Tropical diseases are typically not subject to the same levels of investment and research as are other diseases, in part because the markets for those products are generally less affluent, leaving companies less able to obtain...
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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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    FDA Adds Three New Pathogens to GAIN Act Qualifying List, But Impact Remains Unclear

    The US Food and Drug Administration (FDA) has released an updated and final list of pathogens that are eligible for special incentives under the 2012 Generating Antibiotics Incentives Now (GAIN) provisions of the Food and Drug Administration Safety and Innovation Act (FDASIA) . Background The GAIN Act was passed in an attempt to incentivize the development of new antibiotics—a response to both growing rates of microbial resistance to antibiotics and a dearth of...
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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...
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    EMA, FDA Team Up to Propose Guideline on Pediatric Gaucher Disease

    US and EU regulators have come together to release an unusual joint proposal that they say will help speed up the development of new treatments affecting pediatric patients with Gaucher disease. Background Gaucher disease is a rare, inherited lysosomal storage disorder which affects patients by causing the buildup of lipids in cells and organs, such as the liver, spleen, kidneys and brain. It is thought that about 20,000 patients have Type I Gaucher disease in the US, a...