• Regulatory NewsRegulatory News

    FDA seeks new members for Patient Engagement Collaborative

    The US Food and Drug Administration is seeking eight new members for its Patient Engagement Collaborative (PEC), a group charged with gaining meaningful patient input into medical product development and regulatory decision making.   The 16-member panel includes patients, caregivers, and advocates who serve for 2 to 3 years. The panel is a joint effort between FDA and the Clinical Trials Transformation Initiative . Panel members are expected to participate in two to f...
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    EMA consults on ICH S12 guideline, shares comments on PFDD reflection paper

    The European Medicines Agency (EMA) on Thursday opened a public consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution (BD) considerations for gene therapies.   The S12 guideline recently reached Step 2b of the ICH process after being endorsed at the ICH Assembly virtual meeting earlier this month. (RELATED: ICH reports ‘significant milestones’ reached on guidelines ranging from impurity testing to eCTD standa...
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    FDA releases draft guidance on assessment of PROs for cancer trials

    The US Food and Drug Administration (FDA) has released a draft guidance that recommends how sponsors can collect “meaningful” patient-reported outcome (PRO) measurements to assess results from cancer clinical trials.   The draft guidance focuses on a set of “core” PRO measures that can be used to gather data on patients’ symptoms, symptomatic adverse events and physical function and is specific to registration trials for anti-cancer treatments intended to demonstrate...
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    Patient-Focused Drug Development: New FDA Draft Guidance Delves Into What’s Important

    The US Food and Drug Administration (FDA) on Monday published the second in a series of four guidance documents on patient-focused drug development, with this latest draft helping sponsors identify what is most important to patients with respect to their experience with disease burden and treatment. In determining what’s important, FDA recommends that sponsors use qualitative research methods, which can generate in-depth thoughts from patients in their own words through...
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    Patient Engagement in Device Trials: FDA Drafts Guidance

    The US Food and Drug Administration (FDA) on Monday released new draft guidance explaining how sponsors can improve the design and conduct of medical device clinical investigations by using patient experience, perspectives and other relevant information. The 10-page draft mainly addresses four questions: What approaches might sponsors use to engage patient advisors (defined by FDA as those who have dealt with or are dealing with a particular condition but are not inv...
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    FDA Draft Guidance on Patient Experience Data Incomplete, Critics Argue

    Industry group BIO, the National Association for Rare Disorders (NORD) and the National Center for Health Research (NCHR) called for clarification on how regulators will use draft guidances proposed by external stakeholders on patient experience data. The comments on the US Food and Drug Administration (FDA) draft guidance from December 2018 on developing and submitting proposed draft guidances related to patient experience data showed some consensus around including ...
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    Patient Experience Data: FDA Drafts Guidance

    Thanks to the 21st Century Cures Act , the US Food and Drug Administration (FDA) on Thursday published new draft guidance to help stakeholders submit a proposed draft guidance on patient experience data. The 12-page draft guidance, which provides information in a Q&A format, addresses questions relating to both guidance development and other potential pathways for contributing patient experience data. “Today’s guidance document is part of our commitment to advance p...
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    CDRH Works on Clinical Objectivity in Patient-Reported Outcomes

    A new study conducted by mathematical statisticians at the US Food and Drug Administration (FDA) bridges the gap between an objective endpoint and a patient-reported outcome (PRO). The research , led by statisticians Chul Ahn and Xin Fang at FDA’s Center for Devices and Radiological Health (CDRH), seeks to arrive at an acceptable level of probability for a PRO measure to reveal the “truth” of a subject’s disease or health condition. It comes at time when the science of ...
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    FDA Finalizes Guidance on PRO Assessment Tool for COPD Trials

    The US Food and Drug Administration (FDA) on Monday finalized guidance to help sponsors developing drugs to treat chronic obstructive pulmonary disease (COPD) to use the St. George’s Respiratory Questionnaire (SGRQ), a patient-reported outcome measure (PRO) assessment tool, in clinical trials. The 8-page guidance, which was revised in draft form in May 2016, notes that sponsors can can use the SGRQ as a PRO assessment of efficacy in submissions to investigational new ...
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    Regulatory Challenges for Direct-to-Patient Clinical Trials

    p> On-site clinical trials may present a number of participation obstacles for individuals living at distances from clinical trial sites or for patients with mobility problems. Direct-to-patient clinical trials can be an alternative. This article reviews applicable regulations and analyzes current strategies for conducting direct-to-patient clinical trials using technologies, such as mobile 'apps' and wearables to report data, as well as the potential for sending trial dr...
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    CDRH Sees Spike in Pre-Market Submissions With Patient-Reported Outcome Measures

    The US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) on Tuesday issued its first report on patient-reported outcomes (PROs), noting a significant uptick in PRO data submitted in 2017. Over a six-year period, the report says CDRH observed a more than 500% increase in the number of pre-market submissions that include PRO measures. And in FY 2017, more than 75% of approved, pivotal original and new study investigational device...
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    Patient Engagement in Device Trials: FDA Meeting Discusses Challenges and Opportunities

    Last week, the US Food and Drug Administration's (FDA) Patient Engagement Advisory Committee (PEAC) met for the first time to look at ways to increase patient engagement in clinical trials for medical devices. Owen Faris, clinical trials director at the Center for Devices and Radiological Health (CDRH), said the opportunity to engage with patients goes beyond using patient reported outcomes (PROs) and patient preference information (PPI) in regulatory decision-making. A...