• Regulatory NewsRegulatory News

    ISPE: Global regulators discuss GMP deficiencies, data integrity, real-world evidence

    The COVID-19 public health emergency has prompted regulators to take a different approach to uncovering data integrity violations in virtual inspections compared to onsite inspections, said an official with Australia’s Therapeutic Goods Administration (TGA). The official also provided an update on the status of the EU’s good manufacturing practice (GMP) guide Annex 1, which addresses the manufacture of sterile medicinal products, at a 24 September panel discussion sponsore...
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    July’s Regulatory Focus: Real-world data, real-world evidence

    Feature articles during July focused on real-world data and evidence (RWD and RWE) and their application to approval processes, labeling, clinical use, and marketing of medical products. Articles examined RWD and RWE in the context of patient outcomes and regulatory challenges, especially when conducting noninterventional studies (NIS); regulatory application of RWD-generated external comparators; and from an interregional perspective in the EU, US, and Japan. Also include...
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    From real-world data to real-world evidence: An interregional perspective

    In recent years, data generation in clinical practice outside clinical trials has grown exponentially, largely because of an increase in the number of advanced signal detection technologies, including sensors, imaging, measurements, and mobile devices. At the same time, database capabilities have also expanded because of advances in storage technology. As a result of the innovative digitalization of the collection of real-world data (RWD) together with artificial intellige...
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    Considerations for regulatory application of RWD-generated external comparators

    Properly designed and analyzed external comparators (ECs) built from real-world data (RWD) and the resulting real-world evidence (RWE) can be compared in certain situations with data generated from single-arm or traditional randomized clinical trials to support regulatory decisions. Proper development, design, and use of ECs requires more than simple matching of the clinical trial inclusion and exclusion criteria within the RWD source. A focus on the specificity of the res...
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    Regulatory considerations for real-world research studies in Europe

    Real-world research studies are a valuable source of real-world evidence (RWE). In Europe, successful deployment of these studies is dependent on identifying, understanding, and demonstrating which regulations are not applicable, as well as identifying (and complying with) the regional (Europe Union) and national requirements that are applicable.   Introduction The European Medicines Agency (EMA) recently called for high-quality observational research of real-world...
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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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    Stakeholders weigh in on big data at EMA forum

    Effective use of real-world data and other data gathered outside the clinical trial arena requires a unified, big-picture approach, according to a new report from the first-ever big data stakeholder forum held by the European Medicines Agency (EMA).   The regulator’s report on the 15 December 2020 forum highlighted the importance of collaboration in addressing some of the challenges of bringing real-world data (RWD) into the drug development process. Some of these incl...
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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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    RWE to Supplement RCTs: FDA Projects Continue

    The US Food and Drug Administration (FDA) has been working with several different data and research companies to further its understanding of how real-world data (RWD) and real-world evidence (RWE) can help to inform its regulatory decision making. One such company that has signed an RWE research collaboration with FDA is COTA, Inc., which is working on a project related to replicating a breast cancer study. Andrew Norden, chief medical officer of COTA, told Focus ...
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    When Can RWE Translate Into Credible Evidence? EMA Officials Discuss

    Real-world evidence (RWE) may provide an opportunity to learn more about a drug’s benefits and risks, but officials from the European Medicines Agency (EMA) said in an article published Tuesday in Clinical Pharmacology & Therapeutics that there will need to be adequate statistical methods to extract, analyze and interpret RWE before they can translate into credible evidence. In order to ensure that any new analytical methods are acceptable for regulators, the EMA auth...
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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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    FDA’s OCE Taps Syapse for Research on RWE

    San Francisco-based data company Syapse said Wednesday that it has signed a multi-year research collaboration agreement with the US Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE) to work on using real-world evidence (RWE) to support regulatory decision making. Syapse and OCE will work with EHRs, labs and registries, among other data sources, to characterize the regulatory suitability of RWE derived from multiple sources. They will also work tog...