• Regulatory NewsRegulatory News

    Industry cites outdated standards as flaw in FDA’s real-world data draft guidance

    The US Food and Drug Administration (FDA) should adopt updated data standards that accommodate the “evolving and emerging” nature of real-world data not currently reflected in the Data Standards Catalogue, according to industry comments on the agency’s draft guidance.   Overall, comments from industry generally supported the principles in FDA’s draft guidance on data standards for drug and biological products submissions containing RWD (RELATED: FDA drafts data sta...
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    FDA issues draft guidance on study designs using real-world data

    The US Food and Drug Administration (FDA) has issued draft guidance to industry on how real-world data (RWD) could be used in clinical trials and how it could be incorporated into the design of non-interventional studies.   The draft guidance , released Thursday, outlines the applicability of FDA’s investigational new drug application (IND) regulations to clinical study designs that use RWD and clarifies the agency’s expectations on new drug applications (NDAs) or bio...
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    Study: RWD not ready for postapproval prime time

    For drugs and biologics that receive accelerated approval, could information drawn from real-world data sources supplant traditional postapproval clinical trials to confirm that the therapies have benefit? Real-world data (RWD) are not yet robust enough to confirm the benefit of drugs awarded accelerated approval based on surrogate endpoints, answered the authors of a recent study.   “The findings of this cross-sectional study suggest that none of the 50 [Food and Drug...
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    Convergence: EMA study reveals need for RWE framework, submission structure

    The European Medicines Agency (EMA) has identified a need for more consistency in how real-world evidence (RWE) is submitted, Xavier Kurz told attendees at RAPS Convergence 2021.   Kurz, the head of surveillance and epidemiology at EMA, said the agency reached the conclusion after analyzing the use of RWE in applications for marketing authorization and extension of indication in 2018 and 2019. The study, preliminary findings from which were shared at RAPS Convergence 2...
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    HMA-EMA plans real-world metadata framework for regulatory decision-making

    Officials at the Heads of Medicines Agencies – European Medicines Agency (HMA-EMA) are moving forward with an effort to create a usable catalogue of metadata to aid the scientific community in identifying real-world evidence that can inform regulatory decisions.   Metadata is defined generally as “a set of data that describes and gives information about other data” and can be further subcategorized by generation, location, ownership, and governance of the data; process...
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    EMA goes all in on big data

    The European Medicines Agency (EMA) and the EU’s Heads of Medicines Agencies (HMA) have issued an ambitious workplan for a joint steering group to tackle issues related to big data in healthcare and medicines development.   The steering group’s 2021-2023 work plan, adopted in June and published Friday, “aims to increase the utility of big data in regulation, from data quality through study methods to assessment and decision-making,” according to EMA’s big data webpage...
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    RWD, RWE, and improved patient outcomes: From FDA's Mini-Sentinel to real-world examples

    Uses of real-world data and evidence (RWD and RWE) have proliferated in recent years, driven by advances in data-gathering tools and methods and enhanced understanding of the value of such data and evidence. Under the 21st Century Cures Act of 2016, Congress codified RWD and RWE in the drug development and approval process. There are many opportunities for the use of RWD and RWE in the approval, labeling, clinical use, and marketing of medical products to enhance individu...
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    FDA offers real-world evidence examples in device applications

     The US Food and Drug Administration (FDA) is offering a detailed look at how real-world evidence sources – from registries to claims data – can be used to support marketing applications for medical devices.   A new report from the FDA’s Center for Devices and Radiological Health (CDRH) breaks down 90 examples in which real-world evidence was used to support final premarket or postmarket regulatory decisions from Fiscal Year 2012 through 2019. These examples include ...
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    A military-civilian perspective on real-world evidence to support regulatory decision making

    This article summarizes the framework promoting the collection and analysis of real-world data (RWD) in the healthcare system. The authors emphasize how the US Military Health System (MHS) used RWD during the Iraq and Afghanistan Wars to achieve historic rates of survival and describe a new era of collaboration between the US Department of Defense (DoD) and the US Food and Drug Administration (FDA). The article reviews the FDA evidentiary standards for medical product appr...
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    FDA Developing Guidance on Real-World Data Quality Issues, Officials Say

    As the US Food and Drug Administration (FDA) grapples with how to use real-world data for regulatory decisions, two FDA officials wrote in a perspective published last Friday in the Clinical Journal of the American Society of Nephrology that the agency is developing guidance on data quality issues unique to the real-world data setting and related study design considerations. Aliza Thompson and Mary Ross Southworth of the Division of Cardiovascular and Renal Products w...
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    EU Taskforce of Pharma Regulators Issues its First Report on Big Data

    A European drug regulators’ taskforce on big data published its first report on Friday. The Heads of Medicines Agencies (HMA) and the European Medicines Agency (EMA) formed the HMA-EMA Joint Big Data Taskforce to harness the potential of data in development and oversight, starting with the new report. They examined clinical trial data, spontaneous adverse drug reports (ADRs), social media data and real-world data (RWD) to inform the work and reach “an important mileston...
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    FDA Proposes New Framework on Prescription Drug-Related Software

    The US Food and Drug Administration (FDA) issued a request for comment on Monday that sets forth a proposal for a new framework on the regulation of software applications for use with one or more prescription drug products.   The agency’s newly adopted digital health approaches “are still in their early stages,” FDA Commissioner Scott Gottlieb said Monday in remarks at the Reagan Udall Foundation annual public meeting. “A lot of these technologies haven’t advanced as...