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    FDA: 30 Percent of Drugs Used in Expanded Access Programs are Later Approved

    A new paper by a team of US Food and Drug Administration (FDA) officials finds that only about 30%of drugs requested for expanded access go on to be approved by the agency. "Expanded access provides just that: access. There is no guarantee that the product sought will be effective and/or safe, much less that it will be effective and/or safe for the particular patient," the authors write. Background In recent years, the debate over patient access to unapproved drugs has...
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    GAO: FDA Improved Expanded Access but More Clarity Necessary

    With the rise of Right-to-Try legislation across states in the US, it might seem like patients with serious or life-threatening ailments and no other comparable medical options might have difficulties in obtaining access to investigational drugs outside of a clinical trial because of the US Food and Drug Administration (FDA). But a new Government Accountability Office report released Tuesday lends credence to many who have voiced concerns with the Right-to-Try laws,...
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    10 Years in, EU Pediatric-Use Marketing Authorization Called a 'Disappointment'

    Biopharmaceutical companies and regulators across Europe have said that in the 10 years since the Pediatric Regulation took effect, some pediatric therapeutic areas have seen progress, though the pediatric-use marketing authorization (PUMA) has not lived up to expectations. Overall, according to a summary of the comments received from 75 stakeholders on the consultation from last November , the European Commission noted that the reward system built into the regul...
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    FDA Amends Humanitarian Device Exemption Regulations

    The US Food and Drug Administration (FDA) on Tuesday announced that it is amending its regulations governing humanitarian device exemptions (HDE) to reflect changes brought on by the 21st Century Cures Act . These exemptions provide a pathway for device makers looking to develop medical devices for small patient populations by exempting them from demonstrating a device's effectiveness. Instead, a company must demonstrate that the humanitarian use device (HUD) "will no...
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    FDA Approved Nearly All Expanded Access Requests in FY2016

    The US Food and Drug Administration (FDA) granted more than 99% of requests for patients to access unapproved drugs and biologics in fiscal year 2016, according to data released last week. Through FDA's expanded access program, the agency allows patients with serious or life-threatening diseases to access unapproved drugs. Under the program, physicians can request access to a drug for individual patients or a group of patients under an expanded access investigational ne...
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    FDA Delays Final Rule on Intended Uses

    The US Food and Drug Administration (FDA) on Friday announced it will delay implementation of its final rule clarifying when tobacco products are regulated as medical products until March 2018 in response to a petition from biopharmaceutical industry groups that raised questions about how the rule would alter the agency's definition of intended uses for drugs and medical devices. The petition, filed by on behalf the Medical Information Working Group (MIWG), the Pharmaceu...
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    Industry Groups Petition FDA Over Revisions to ‘Intended Use’ Rule

    The Medical Information Working Group (MIWG), the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) have petitioned the US Food and Drug Administration (FDA) following its “unexpected decision” last month to revise the definitions of “intended use” for drugs and medical devices. The industry groups call for this final rule to be stayed indefinitely and reconsidered because FDA failed to give fair no...
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    Industry Argues Against FDA Home-Use Device Label Database

    The Advanced Medical Technology Association (AdvaMed) is at odds with a recent proposal by the US Food and Drug Administration (FDA) to create a public facing electronic database for home-use medical device labels. In October, FDA announced the proposed rule , which would require the makers of Class II and Class III medical devices intended for home-use to submit the labels and package inserts for their products electronically. These electronic labels and package insert...
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    FDA Looks to Decrease Adverse Events With New Home-Use Device Label Database

    As part of efforts to reduce an increasing number of adverse events from home-use medical devices, the US Food and Drug Administration (FDA) is proposing to create an online database of all such device labels and to require companies to submit the labels and package inserts in an electronic format.  The shift comes as FDA reviewers claim that the agency receives reports of approximately three to five of the most serious types of adverse events (deaths, fires, explosions...
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    UK’s MHRA Introduces Re-Manufacturing of Single-Use Devices

    The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on Tuesday said that single-use medical devices may be re-manufactured for use in the UK, though the remanufacturers should meet all relevant criteria under the appropriate device directives and place a CE mark on their product. The shift to allow such re-manufacturing comes with new guidance from MHRA, which notes that the re-manufacturing of single-use devices and similar processes have existed outsid...
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    FDA Looks to Revitalize Compassionate Use Program With Simplified Form, Final Guidance

    The US Food and Drug Administration (FDA) on Thursday finalized two question and answer guidance documents on charging for investigational drugs and expanded access for the compassionate use of investigational drugs for those with life-threatening diseases or conditions, as well as a new streamlined form for applying for expanded access. The new form, known as the Form FDA 3926, is part of an expedited process to help patients gain access to drugs that are still in clini...
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    Off-Label Drug Use and Pharmacovigilance: EMA Outlines What MAHs Need to Collect and Report

    Pharmaceutical companies operating in the EU will need to collect and report information on the off-label use of their medicines under new obligations making their way from draft to final form at the European Medicines Agency (EMA) this summer. In late April, EMA offered a reflection paper , open for comment through 29 July, which offers the agency’s proposal for the collection and reporting of information on off-label use for marketing authorization holders (MAHs), in ...