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Introduction1
The conduct and design of clinical trials has changed considerably over the past decade since the 2011 Institute of Medicine workshop Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020 (IOM, 2012). The evolution of health care is expanding the possibilities for integration of clinical research into the continuum of clinical care; new approaches are enabling the collection of data in real-world settings; and new modalities, such as digital health technologies2 and artificial intelligence applications, are being leveraged to overcome challenges and advance clinical research. At the same time, the clinical research enterprise is strained by rising costs, varying global regulatory and economic landscapes, increasing complexity of clinical trials, barriers to recruitment and retention of research participants, and a clinical research workforce that is under tremendous demands.
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1 This workshop was organized by an independent planning committee whose role was limited to identification of topics and speakers. This Proceedings of a Workshop was prepared by the rapporteurs as a factual summary of the presentations and discussions that took place at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants and are not endorsed or verified by the National Academies of Sciences, Engineering, and Medicine, and they should not be construed as reflecting any group consensus.
2 Defined as ranging “from hardware—such as wearable devices and sensors—to software, such as mobile phone apps that enable consumers to monitor their own health and participate in studies; telemedicine platforms to connect patients with clinical providers; and artificial intelligence to support clinical decision making” (NASEM, 2020, p. 1).
Looking ahead to 2030, the Forum on Drug Discovery, Development, and Translation of the National Academies of Sciences, Engineering, and Medicine (the National Academies) convened a public workshop for stakeholders from across the drug research and development (R&D) life cycle to reflect on the lessons learned over the past 10 years and consider opportunities for the future. The agenda for the workshop was developed by an independent planning committee to address the established task (see Box 1-1).3 Specifically, the workshop was designed to consider goals and priority action items that could advance the vision of a 2030 clinical trials enterprise that is more efficient, effective, person-centered, inclusive, and integrated into the health care delivery system so that outcomes and experiences for all stakeholders are improved. The workshop was co-chaired by Esther Krofah, executive director at FasterCures, a center of the Milken Institute, and Steven Galson, senior vice president of Research and Development at Amgen Inc.
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3 The agendas for the four parts of the workshop can be found in Appendix C.
Originally intended to be held in person in Washington, DC, over the course of 1.5 days, the workshop was redesigned as a four-part virtual event spanning 5 months due to the COVID-19 pandemic and the need to hold the workshop virtually. At the time of the first meeting in January 2021, more than 25 million people in the United States had been infected with COVID-19, and more than 400,000 had died as a result.4 “We have witnessed a tremendous response from the medical research community in accelerating the development of therapeutics and vaccines,” said Krofah. In January 2021, two vaccines had already shown sufficient evidence of safety and efficacy in clinical trials to be authorized for emergency use by the U.S. Food and Drug Administration (FDA). At the same time, however, the pandemic resulted in the disruption of new and existing clinical trials for a range of diseases. “It’s clear that we have an urgent need, now more than ever, to advance a clinical trials enterprise that is more efficient, effective, person-centered, inclusive, and integrated into the health delivery system,” Krofah said.
ORGANIZATION OF THE WORKSHOP AND PROCEEDINGS
This Proceedings of a Workshop summarizes the presentations and discussions that took place during the four-part virtual public workshop held on January 26, February 9, March 24, and May 11, 2021.5 All four parts of the workshop, including interactive breakout group discussions, were facilitated remotely via Zoom and webcast live. Participants were also able to submit comments and questions throughout the workshop via the webcast comment window or within a dedicated Slack workspace.
Chapter 2 provides an overview of some changes in communities and clinical research as a result of the COVID-19 pandemic, lessons learned from the positive and negative effects of those changes, and ways in which an envisioned 2030 clinical trials enterprise could differ from the current system. It introduces some of the key challenges and opportunities for achieving that vision. Chapter 3 summarizes workshop discussions focused on achievable goals to enhance person-centeredness and inclusivity throughout the clinical trials enterprise and ways to improve public engagement and partnership. Chapter 4 covers discussions on how the thoughtful and deliberate use of new technologies could enhance the clinical trials enterprise. Chapter 5 recaps the workshop discussions in
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4 This information is from the Johns Hopkins University Coronavirus Resource Center. For more information and updated counts, see https://coronavirus.jhu.edu/us-map (accessed September 9, 2021).
5 Archived webcast videos and additional meeting materials are available on the National Academies website. See https://www.nationalacademies.org/our-work/envisioning-a-transformed-clinical-trials-enterprise-for-2030-a-workshop (accessed July 19, 2021).
which participants considered approaches to building a more resilient, sustainable, and transparent clinical trials enterprise, including the integration of clinical research and clinical practice. Throughout Chapters 3, 4, and 5, many individual workshop speakers and participants drew examples from their experiences and observations during the COVID-19 pandemic, and discussed the potential for some of those lessons learned to inform an envisioned transformation of the clinical trials enterprise.
Over the course of the workshop, three current and former FDA officials shared their personal perspectives on the state of the clinical trials enterprise, changes made as a result of the COVID-19 pandemic, examples of positive changes made that potentially could be continued post-pandemic, and what it may take to realize the 2030 vision. These discussions are summarized in Chapter 6, which also includes closing remarks from the workshop co-chairs. A series of Health Affairs blog posts were written by select workshop speakers to complement the workshop discussions (see Appendix A).6 Participants were encouraged to share their insights and observations about the workshop on Twitter using the hashtags #ClinicalTrials2030 and #DrugForum.
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6 See https://www.healthaffairs.org/topic/ss170 (accessed July 1, 2021).
