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  • Regulatory NewsRegulatory News

    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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    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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    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 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...
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    FDA Explains Emergency Expanded Access Application Process

    The US Food and Drug Administration (FDA) has laid out the process by which doctors can access an investigational drug for treatment in an individual patient in an emergency situation, both during and after normal business hours in a new Manual of Policies and Procedures. The release of the MAPP coincides with a bill passed in the Senate and making its way through the House that aims to ease the process by which terminal patients can access such investigational dr...
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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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    UK Patients to Get Early Access to AbbVie's HCV Combo

    Patients in the UK will get early access to AbbVie's experimental pan-genotypic hepatitis C (HCV) drug combination, the Medicines and Healthcare products Regulatory Agency (MHRA) announced Wednesday. The combination, known as glecaprevir/pibrentasvir, is currently undergoing an expedited review by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) for its potential to treat patients across all six major genotypes of HCV, with or wi...
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    Sanofi, CDC and FDA Work to Avoid Yellow Fever Vaccine Shortage

    To head off an impending shortage of YF-VAX, the only US-licensed vaccine for yellow fever, Sanofi Pasteur, the US Centers for Disease Control and Prevention (CDC) and the US Food and Drug Administration (FDA) are working to provide travelers with an alternative vaccine. In the coming months, Sanofi will begin importing Stamaril, a yellow fever vaccine developed from the same substrain as YF-VAC, and will distribute it to select clinics around the US. While Stamaril is ...
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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...