• Regulatory NewsRegulatory News

    FDA offers draft guidance for registries as RWD

    The US Food and Drug Administration (FDA) released draft guidance on the use of registries in regulatory decision making, adding another piece to the puzzle of how the agency will consider real-world data (RWD) when considering a new drug indication or assessing post-approval requirements.   The draft guidance , which was published Monday, outlines considerations for sponsors and stakeholders when proposing to design a registry or use an existing registry to support r...
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    FDA drafts data standards guidance for RWD

    The US Food and Drug Administration (FDA) has released draft guidance for industry that outlines the agency’s thinking on how a sponsor should submit drug and biological product study data from real-world data (RWD) sources.   FDA acknowledged that standardizing data for use as RWD is fraught with such challenges as inconsistent formats and sources, different source data captured by region, differences in terminology and exchange, a wide range of methods to build datas...
  • Feature ArticlesFeature Articles

    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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    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’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...
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    FDA Discusses RWD, RWE With Industry, Academia

    Industry, academia and US Food and Drug Administration (FDA) officials gathered Friday in Bethesda, MD to discuss the use of real-world data (RWD) and real-world evidence (RWE) in the premarket and postmarket settings. Jacqueline Corrigan-Curay, director of CDER’s Office of Medical Policy, kicked off the joint FDA-American Association for Cancer Research meeting to discuss FDA’s framework for using RWE, which was unveiled in December 2018 . The framework is meant to gu...
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    Gilead and Novartis Seek to Expand What FDA Considers as Real-World Data and Real-World Evidence

    Just over two months ago, the US Food and Drug Administration (FDA) released draft guidance explaining to drugmakers what constitutes real-world data (RWD) and real-world evidence (RWE) and how to submit such data to the agency. The draft guidance spells out what the agency considers to be an RWD source (i.e. electronic health record data, medical claims or billing data, etc.) and what relevant submissions may include RWE (i.e. single arm trials that use RWE as an ext...