• Regulatory NewsRegulatory News

    EU Court Rules on Orphan Product Exclusivity

    A European Court has issued a ruling in an unlikely scenario dealing with overlapping periods of orphan product market exclusivity. In its ruling, which has the potential to influence how companies develop orphan products, the court found that authorized orphan products are entitled to market exclusivity, even when they are similar to an already approved product. Background Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141...
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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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    Pfizer Launches Expanded Access Program for Breast Cancer Drug

    The US Food and Drug Administration's (FDA) expanded access program, sometimes referred to as its "Compassionate Use" program, has frequently been in the news as of late, as dozens of patients have taken to social media in an attempt to pressure companies into allowing them access to clinical trials they believe might save their lives. But while many companies have seemed reluctant to grant access to those trials, pharmaceutical giant Pfizer is taking a markedly differen...
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    FDA Finalizes Expedited Product Guidance With Hundreds of Major, Minor Changes

    The US Food and Drug Administration (FDA) has finalized its long sought-after guidance document on the use of its four expedited drug approval programs, including the recently instituted breakthrough product designation. Background FDA released the draft expedited programs guidance, Expedited Programs for Serious Conditions—Drugs and Biologics , in June 2013. In the draft, FDA outlined how it says it planned to use four tools at its disposal designed to expedite ...
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    FDA, Absent From Hearing, Makes the Case That it's Increasingly Innovative

    Yesterday, US legislators in the House Energy and Commerce Committee held a hearing to discuss how the federal environment—including the US Food and Drug Administration's (FDA) regulations—might be improved to bolster drug innovation. But while the pharmaceutical industry, device industry and patient groups found themselves well represented at the meeting, one prominent stakeholder found itself conspicuously excluded from the meeting: The US Food and Drug Administration. ...
  • When are Postmarketing Trials Required for Medical Devices? New FDA Guidance Explains

    Earlier this week, the US Food and Drug Administration (FDA) launched the Expedited Access Premarket Approval program (EAP) , a new program aimed at getting innovative medical device products to needy patients more quickly by expediting the regulatory review of the products and their manufacturing processes. But in addition to the guidance document detailing the program, FDA also issued a draft guidance document on which clinical data FDA will expect companies to have a...
  • FDA Launches Long-Awaited Expedited Approval Program Aimed at High-Need Devices

    The US Food and Drug Administration (FDA) has released a long-awaited draft guidance document outlining an expedited approval pathway that can be used by sponsors of medical devices intended to treat life-threatening or "irreversibly debilitating" diseases or conditions. Background: EAP Basics The guidance, which for months has been rumored to be near completion , introduces the "Expedited Access Premarket Approval" program, or EAP for short. Regulatory officials said ...
  • 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 ...
  • FDA Provides Update on Breakthrough Product Designation Program, Showing Industry Interest

    More than one year has passed since the US Food and Drug Administration (FDA) was first given the authority to give products "breakthrough" designation under the Food and Drug Administration Safety and Innovation Act (FDASIA) , granting companies a range of tools to accelerate the review of a product. But despite not having much in the way of information about the designation until June 2013, sponsors have been nothing but eager to obtain it, according to data released ...
  • In Landmark Move, First Breakthrough Product Obtains FDA Approval

    The US Food and Drug Administration (FDA) has announced its first-ever approval under the breakthrough product designation, a new approval pathway of sorts intended to expedite the process by which highly promising products reach the market. Background Under the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, FDA was given the authority to use a new expedited approval program known as breakthrough product designation. As defined by FDA, breakt...
  • Industry Weighs in on Expedited Products Guidance, Calling for Definitional Clarity

    In June 2013, the US Food and Drug Administration (FDA) released a long-awaited and much-anticipated guidance document on its new breakthrough product designation , and in the process of doing so overhauled much of its existing guidance on so-called "expedited programs" as well. Now one of the pharmaceutical industry's most prominent groups is weighing in on the topic, saying that while the guidance is "critical," extensive changes to definitions are warranted. Backgrou...