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PP09-64: Key Themes from Comments Received to the Framework of the FDA’s Real-World Evidence Program

Poster Presenter

      Heejung Noh

      • ORISE Fellow, Office of New Drugs, CDER
      • FDA
        United States


The objective is to share high level themes of the public comments submitted to the docket for the Framework of the FDA Real-World Evidence Program.


Public comments submitted to the docket between December 7, 2018 and July 17, 2019 for the Framework of the FDA’s Real-World Evidence Program were collected. The collected comments were then reviewed and categorized for consideration by FDA subject matter experts.


34 stakeholders submitted comments to the docket (Docket No. FDA-2018-N-4000). The distribution of stakeholders who commented are pharmaceutical companies (n=7), professional and health policy organizations (n=16), healthcare system (n=1), academia (n=1), advocacy groups (n=5), and technology and clinical research organizations (n=4). Each submission was reviewed, and individual comments were then categorized into topic areas. Categories included: requests for greater clarity regarding timelines for RWE program items, providing additional considerations on the generation of RWE from RWD, clarifications in terminology, stakeholder engagement, data source, quality and standards, analytical methodology, study design, endpoints, MyStudiesApp, regulatory decisions, and regulatory considerations. Some categories were further classified into subcategories to identify more common explicit themes. For instance, comments that were categorized as study design were then grouped into observational studies, randomized controlled trials, hybrid designs, and external control arms. Among categorized topics, stakeholder engagement and study design categories yielded the greatest number of comments. Stakeholders provided comments in multiple categories. The majority of comments in the stakeholder engagement and study design categories were from pharmaceutical companies and professional and health policy organizations. Stakeholders from technology and clinical research organizations comments were primarily focused on data quality. Advocacy group’s comments were distributed among multiple categories. Only one healthcare system and one from academia provided comments; and, those comments were primarily related to study design. All stakeholders except for advocacy groups had at least one comment related to the need to clarify the terminology used in the FDA Framework and the need for consistency moving forward.


There was a wide distribution of stakeholders who provided comments to the docket. The analysis showed that stakeholder comments aligned with their perspectives and need to understand the potential for the use of RWE as it relates to their area of expertise. Stakeholder engagement, study design, and clarification of terminology were of greatest interest across all stakeholders. Stakeholders appreciated opportunities to engage the Agency and encouraged continuous engagement as the Agency continues to explore the potential use of RWE. Professional and health policy organizations as well as advocacy groups urged the Agency to increase opportunities so that patient perspectives are considered. Other notable stakeholder input addressed the types of situations in which observational studies and RWD/RWE-based comparator arms can be utilized to support regulatory decision-making. Stakeholders urged the Agency to continue its development of guidances on the suitability of RWD and to engage stakeholders throughout the process of determining when a study designs have the potential to generate RWE to help answer regulatory questions. Furthermore, stakeholders expressed the need for consistency in the use of “real-world” terminology to prevent confusion since many terms can have several interpretations. This is important for future guidance development. For example, stakeholders suggested the use of “primary” and “secondary” in describing data instead of “prospective” and “retrospective” because this context for which those terms are used differs. Overall, stakeholders expressed appreciation for the Agency’s efforts to understand the potential for RWE and supported the Agency’s outlook on how to move forward to implement the RWE program. FDA RWE subject matter experts reviewed all comments and were also appreciative of stakeholder feedback and perspectives. The Agency is committed to working with stakeholders to continue exploring the potential use of RWD to generate RWE.