Examining the Impact of Real-World Evidence on Medical Product Development Proceedings of a Workshop Series (2019) / Chapter Skim
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1 Introduction
1 Introduction
Pages 1-12
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Evidence generated from real-world use -- based on sources such as patient registries, electronic health records (EHRs) , and medical claims data -- may provide valuable information, alongside RCTs, to inform medical product decision making.
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, in Chapter 10) . To explore the potential for using RWE in medical product decision making, an ad hoc workshop planning committee of the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series, sponsored by FDA and hosted by the National Academies' Forum on Drug Discovery, Development, and Translation (see Box 1-1 for the Statement of Task)
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Topics to be covered at the workshop include • Aligning incentives and addressing barriers to support collection and use of high-quality evidence derived from real-world data sources in health product review, payment, and delivery; • Definitions surrounding the core components of RWE; • Sources of data that are curated, standardized, and analyzed to derive RWE, such as safety surveillance, observational studies, registries, claims, or patient-centered outcomes research; • Gaps in data collection activities, and priority areas and pilot opportunities that RWE incorporation could address; • Standards and methodologies for collecting and analyzing RWE in sup port of new indications or postapproval studies, and the circumstances under which that evidence could be applied; • Applications for using RWE to supplement traditional clinical trials, p ragmatic/effectiveness trials, or routine clinical applications; • Mechanisms for determining which discrete types of RWE could support regulatory decisions; and • Operational challenges and barriers for generating and incorporating RWE in the context of a learning health system and how clinicians can best be involved in the collection and utilization of RWE.
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The decision aids were developed to guide discussion at the third workshop, and they may also be useful in helping workshop a ttendees and other stakeholders think about and evaluate opportunities to use RWD and RWE for medical product decision making and make informed choices about the design of prospective or retrospective tudies, primarily for regulatory review (akin to clinical decision aids that s are designed to help patients make informed decisions about treatment options)
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Chapter 1 introduces the topic and frames the workshop series; Chapter 2 presents various stakeholder perspectives on RWE; hapter 3 discusses successful examples of the use of RWE in research; C Chapter 4 discusses barriers and disincentives to the use of RWD and RWE; and Chapter 5 considers RWE in the context of the current evidencegeneration system. Chapters 6 through 9 focus primarily on in-depth discussions from the second and third workshops.
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Mark McClellan, director of the Duke-Margolis Center for Health Policy, offered the definitions of RWD and RWE that were developed through the work of a collaborative agreement between FDA and the DukeMargolis Center. RWD are "data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources." RWE, said McClellan, is "evidence derived from RWD through the application of research methods." In the specific context of RWE for regulatory applications, RWE can be further defined as "clinical evidence regarding the use and potential benefits or risks of a medical product derived from analysis of RWD." McClellan said there is a common misconception that RWD simply means observational data.
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patient health status and/or the delivery of health care routinely collected from a variety of sources. RWE is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD (https://www.fda.gov/scienceresearch/specialtopics/ realworldevidence/default.htm, accessed December 17, 2018)
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Discerning which traditions are important and which can be let go, said Simon, is critical for moving scientific progress forward efficiently and effectively. FDA commissioner Scott Gottlieb concurred with this analogy in his keynote address, saying that expanding traditional notions of evidence generation to incorporate the use of RWE could help make the medical product development process more efficient and more cost-effective.
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However, FDA as a public health agency has a mandate to embrace the full continuum of evidence available from all sources along the entire life cycle of a product. "We can't allow our need for a point of regulatory accountability to prevent us from looking across the line we have to draw at practical information that's collected both before and after our point of demarcation when a product gains a license for initial market entry." RWE, said Gottlieb, could help FDA make more informed decisions along the continuum, from providing data about the benefit–risk profile of a product, allowing for early identification and a richer understanding of safety concerns.
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The legislation specifically requires FDA to hold workshops, evaluate the potential use of RWE, and issue draft guidance by the end of 2021. As part of these activities, FDA partnered with Duke-Margolis for the purpose of exploring the use of RWE for regulatory purposes and sponsored the threepart workshop series hosted by the Forum on Drug Discovery, Development, and Translation described in this proceedings.
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McClellan noted that regardless of the chosen method, it is unlikely that "one single real-world evidence study is going to be all the evidence that exists for most of these regulatory questions." FDA looks at the totality of the evidence when making regulatory decisions, and RWE can be a part of the overall picture. Another area of focus of the Duke-Margolis partnership with FDA, said McClellan, has been on the challenges of data.
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McClellan noted that some stakeholders -- health insurance companies in particular -- are making major investments in data infrastructure, with a goal of integrating data to support better decisions for patients. These types of efforts will be highly relevant to advancing the generation of RWE for regulatory purposes, he said, and will help to integrate the regulatory framework with a framework based on patient outcomes.
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