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    FDA Launches Expanded Access Pilot ‘Project Facilitate’

    The US Food and Drug Administration (FDA) on Monday launched a new pilot program, dubbed Project Facilitate, aimed at helping physicians complete expanded access requests for cancer patients.   “The FDA has been working diligently to improve the Expanded Access framework, including development of an updated and more streamlined application form, but despite recent improvements, we understand that for many patients or health care professionals, especially those not fami...
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    Right to Try One Year Later: Limited Patient Involvement but More FDA Clarity Coming

    The Right to Try Act has officially been in place for one year and although just two patients have publicly announced that they have used the law to gain access to experimental therapies, hundreds more may follow. The US Food and Drug Administration (FDA) on Thursday offered a new website to explain what patients and what investigational drugs are eligible under the law. The website includes eight questions and answers on the law, clarifying that companies are not r...
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    FDA Warns Ocular Therapeutix Over Failure to Comply With Postapproval Requirements

    Ocular Therapeutix drew a US Food and Drug Administration (FDA) warning letter for failing to comply with post-approval study requirements (PAS) set as part of its ReSure Sealant’s conditional approval. The warning letter, dated October 2018, cites the company’s failure to collect any data for its FDA-approved protocol—identified as a Device Exposure Registry (DER) study—as part of the PAS requirements in the 4.5 years since the conditional approval. Ocular Therapeutix’...
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    Physicians’ Favorable Views of FDA Drug Approval Standards Underscore ‘Disconnect,’ Survey Finds

    A research letter published in JAMA Internal Medicine on Tuesday highlights the “disconnect” between many physicians’ perceptions of the US Food and Drug Administration (FDA) drug approval process and the “current reality.” The findings are based on a survey aimed at gaging physicians’ attitudes toward FDA drug approval standards and off-label promotion, with a total of 686 survey (48% response rate) respondents listed by the American Board of Internal Medicine Diplom...
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    PMA Denials: Why so Few?

    In the pharmaceutical industry, complete response letters, or rejections, are a common occurrence. And though they aren’t frequently explained in detail publicly, the public is still usually notified that the company has received such a letter. But in the world of medical devices, failing to win approval for a high-risk device is a much more closely held secret, and companies can withdraw their applications rather than be denied approval.   A Federal Register notice ...
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    Bioethicists Say Federal Right-to-Try Legislation Will Have Limited Impact on Access

    In a perspective published in the New England Journal of Medicine on Wednesday, bioethicists Holly Fernandez Lynch and Steven Joffe of the University of Pennsylvania warn that federal "right-to-try" legislation could undermine the US Food and Drug Administration's (FDA) public health mission and will have a limited impact on improving patient access to investigational products. Background The debate around right-to-try centers around patients' ability to access unappr...
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    What New CDRH Guidance is Coming in FY 2018: Alternative 510(k) Pathway and More

    While recognizing that comparison testing for 510(k) applicants can be outdated, especially for newer medical devices, the US Food and Drug Administration (FDA) announced Monday that in the first quarter of 2018, new draft guidance will be released to address an alternative pathway for demonstrating substantial equivalence. The creation of this voluntary, alternative 510(k) pathway, first announced back in September , "will allow more flexibility to use more moder...
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    European Regulatory Roundup: ex-GSK CEO to Chair UK Accelerated Access Collaborative (9 November 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. UK Tasks ex-GSK CEO With Highlighting Drugs Eligible for Accelerated Access Pathway The UK has tasked former GlaxoSmithKline CEO Sir Andrew Witty with highlighting products suitable for its incoming accelerated access pathway (AAP). Products that follow AAP will benefit from support with evidence generation and a more seamless journey to market, features the government ...
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    FDA Widens Expanded Access Navigator as Johnson Presses House to Pass Right-to-Try

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb on Thursday announced that a web platform to help patients find information on clinical trials for cancer drugs would be expanded to include orphan disease drugs. The decision follows a House Energy & Commerce Committee hearing last month on a bill that passed the Senate in August and seeks to speed and increase access to experimental therapies for terminally ill patients. In written comments to...
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    FDA Issues 8 Guidances on Device User Fees, MDUFA IV Goals & Pre-Submissions

    The US Food and Drug Administration (FDA) has issued seven new or updated guidances offering advice to medical device makers on its user fee programs and how FDA and industry actions impact the performance goals set by the fourth iteration of the Medical Device User Fee Amendments (MDUFA IV). In its MDUFA IV commitments, FDA agreed to tighter timelines to reach decisions for most medical device submissions in exchange for increased user fees and the introduction of a n...
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    FDA Seeks Tweaks to Right-to-Try Bill in House E&C Hearing

    The House Energy & Commerce Committee on Tuesday held a hearing to discuss a "Right-to-Try" bill passed in the Senate that the US Food and Drug Administration (FDA) is seeking to change. The hearing discussed a bill that would seek to undercut FDA’s oversight of a program, known as expanded access, whereby terminally ill patients and their doctors can request access to an investigational drug outside of a clinical trial. Right-to-Try laws have now passed in 37 states...
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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...