• Regulatory NewsRegulatory News

    Long-Awaited UN Report Calls for Breaking Link Between Drug R&D Costs and Prices

    The United Nations (UN) High-Level Panel on Access to Medicines released its long-awaited report on Wednesday, with recommendations sure to rile up the pharmaceutical industry as it calls for World Trade Organization (WTO) members to no longer link the cost of research and development (R&D) with drug prices, as well as for an increase in the issuance of compulsory licenses and to require the disclosure of pharmaceutical R&D costs. The call for more compulsory license...
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    Industry, Patient Groups Respond to FDA Guidance on Sharing Patient-Specific Device Data

    Several groups representing the medical device industry and patients are calling for more clarity from the US Food and Drug Administration (FDA) on its recent draft guidance on sharing patient-specific device data with patients. In June, FDA released its draft guidance, Dissemination of Patient-Specific Information From Devices by Device Manufacturers , to clarify that device makers may share patient-specific information recorded, stored, processed, retrieved and/or d...
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    Ulcerative Colitis Drugs: FDA Offers Draft Guidance on Clinical Development

    Pharmaceutical companies looking to understand FDA’s current thinking on efficacy endpoints for clinical trials to develop new ulcerative colitis (UC) treatments will be interested in new draft guidance released Friday. And though the guidance spells out what primary endpoints should be used, the agency also bemoans the fact that there are not well-defined and reliable clinician-reported, patient-reported and observer-reported outcome instruments for clinical trials. Ba...
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    Convergence Keynote Speaker Lori Hartwell Passionate About Patients

    At the start of a recent phone conversation with patient advocate and 2016 Regulatory Convergence closing keynote speaker, Lori Hartwell, she paused for a moment to warn that if there is any audible squawking in the background, it’s just her pet parrot, Johnny. Hartwell, an animal lover, has four dogs and a cat in addition to Johnny, but they are more than just pets. They are an important part of her philosophy on living happily with chronic illness. Hartwell i...
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    Researchers: Patient Reported Outcomes for Heart Failure Fail to Meet FDA Criteria

    A team of researchers say that no currently available patient-reported outcomes for heart failure meet the US Food and Drug Administration's (FDA) criteria for supporting product approval. In a recently published review in the Journal of the American College of Cardiologists , the researchers checked the patient-reported outcomes against nine criteria adapted from the 14 listed in FDA's 2009 guidance, Patient-Reported Outcome Measures: Use in Medical Product Developme...
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    EMA and FDA Begin Collaborations on Patient Engagement

    The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Wednesday began exchanging more information on the best practices of involving patients in the development, evaluation and post-authorization tracking of medicines. The EMA/FDA teleconference, also known by EMA as a "cluster," on patient engagement seeks to increase the exchange of information on engaging with and involving patients and their organizations within the regulators’ wor...
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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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    FDA Official Dispels ‘100 Hours’ Myth on Time it Takes to Fill Out Compassionate Use Form

    The simplification of the US Food and Drug Administration’s (FDA) compassionate use process (announced last February and expected to be finalized very soon) may not actually be all that much of a simplification in terms of the amount of time it takes a physician to fill out the form, an FDA official told Focus on Tuesday. Richard Klein, director of the Patient Liaison Program in FDA’s Office of Health and Constituent Affairs, explained to Focus , following Monday’s ...
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    Expedited Compassionate Use for Investigational Drugs Coming Soon, FDA Says

    The fight over patients gaining access to investigational drugs via the compassionate use process is expected to get a lot easier in the near future, just as the US Food and Drug Administration’s (FDA) nonprofit arm, the Reagan-Udall Foundation (RUF), is working to build an online navigator to help patients and physicians understand that process better. Compassionate use, also known as expanded access, is a process by which physicians can request that FDA allow the use o...
  • Feature ArticlesFeature Articles

    Market Access in Europe: Bridging Regulatory and Health Technology Assessment (HTA) Gaps

    This article introduces the roles of HTA bodies within the EU member states healthcare system and provides key elements to consider for manufacturers who would like to enter this geographical market. In addition, solutions to maximize the product development strategy are discussed with the intention of bridging regulatory and market access gaps. The rapid diffusion of health technologies has presented governments with unprecedented challenges to provide high quality an...
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    FDA’s Nonprofit Arm Plots Out New Compassionate Use Navigator

    Patients and physicians desperate for access to a potentially life-saving investigational drug will be able to take some solace in the fact that the US Food and Drug Administration’s (FDA) nonprofit, known as the Reagan-Udall Foundation, is currently working on an online tool to help aid the process of obtaining what are known as "compassionate use" or "expanded access" treatments. The small foundation, which is currently operating with six employees, has drafted a plan ...
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    EMA’s Open Trial Data Policy After Five Years: Long Waits, Lengthy Correspondence

    The European Medicines Agency’s (EMA) five-year-old policy of allowing some academic researchers and journalists to access what’s considered commercially confidential information in the US has resulted in a lengthy process that can take considerable back and forth correspondence with the agency, according to a new study. Since 2010, EMA has allowed clinical study reports to be subject to its “reactive” freedom of information policy, which makes it the only regulator in t...