1

Introduction¹

As stated in the 2022 National Academies of Sciences, Engineering, and Medicine (the National Academies) report Improving Representation in Clinical Trials and Research: Building Research Equity for Women and Underrepresented Groups, “An equitable clinical research enterprise would include trials and studies that match the demographics of the disease burden under study. However, we remain far from achieving this goal” (NASEM, 2022). Despite decades of work and ongoing efforts on the part of policymakers, patient groups, nonprofits, and industry sponsors to improve racial and ethnic diversity in clinical trials, there has been little change over time (Dzau et al., 2022). Furthermore, inconsistencies in data collection and reporting make it difficult to track progress of demographic group participation in the United States for clinical trials. Given the urgent need, there have been calls for collective action across sectors and organizations “to align on goals for system-wide sustainable change” (Bierer et al., 2024).

On May 20, 2024, the National Academies Forum on Drug Discovery, Development, and Translation, in collaboration with the National Cancer Policy Forum, convened a public workshop to explore opportunities to improve racial and ethnic diversity with a focus on system-level change and collective efforts across organizations and sectors that no one entity

___________________

¹ The planning committee’s role was limited to planning the workshop, and the Proceedings of a Workshop has been prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants and are not necessarily endorsed or verified by the National Academies of Sciences, Engineering, and Medicine, and they should not be construed as reflecting any group consensus.

can effectively take on alone (see Box 1-1). The workshop builds on meetings hosted by the Clinical Trials Transformation Initiative (CTTI) in June 2023, the Multi-Regional Clinical Trials (MRCT) Center of Brigham and Women’s Hospital and Harvard University in September 2023, and FasterCures of the Milken Institute in November 2023.

TOWARD A NATIONAL ACTION PLAN

To better align goals and drive collective, sustained efforts, members of four organizations with established leadership in increasing representation in clinical trials—CTTI, Milken Institute FasterCures, MRCT, and the Forum on Drug Discovery, Development, and Translation—coordinated a series of convenings to address the following aims as outlined by Barbara

Bierer, faculty director of the MRCT Center and professor of medicine at Harvard Medical School and Brigham and Women’s Hospital and member of the Forum on Drug Discovery, Development, and Translation:

• “Align on domains for improving diversity, equity, and inclusion in clinical trials that, if effectively addressed, would promote system-level change within the clinical trials enterprise.
• Describe common goals for each domain and key collective actions necessary to achieve those goals.
• Inspire organizations to work together toward common goals and commit to taking collective actions.
• Support organizations as they develop metrics to assess progress over time individually and collectively.
• Learn together.
• Drive accountability.”

Based on input from over 200 individuals representing academia, patient groups, federal agencies, industry, and communities; conversations at three convenings individually hosted by CTTI, MRCT, and FasterCures; information from questionnaires; and a literature analysis, a report coauthored by CTTI, the Milken Institute, and MRCT, Toward a National Action Plan for Achieving Diversity in Clinical Trials (Bierer et al., 2024),² was released in May 2024.³

The report published by the three organizations is ordered around eight domains or priority areas. Bierer shared that each domain includes goals, 2–6 collective actions to achieve those goals, and steps that “organizations can do now, can do with collaborators now, to advance the field” and promote systems-level change (see Box 1-2 for domains and goals). She pointed out that the some of the action steps overlap between domains and that the domains should not be thought of as “different silos.”

Morgan Hanger, executive director of CTTI and member of the Forum on Drug Discovery, Development, and Translation, stated that the publication is intended to serve as a first step toward collective action—more is needed to pressure-test the ideas and see what will work. Bierer noted that the action plan is focused initially on equitable representation of racially and ethnically minoritized populations in clinical trials, with the intention

___________________

² See https://mrctcenter.org/wp-content/uploads/2024/05/Toward-a-National-Action-Plan-for-Achieving-Diversity-Clinical-Trials-240502_FINAL.pdf (accessed September 9, 2024).

³ The content of this workshop was informed by the previous convenings hosted by MRCT, CTTI, and FasterCures. This fourth convening provided an opportunity for the clinical trials community to weigh in on themes that overlap with those found within the national action plan. The workshop was developed independent of the report.

that lessons could be applied toward improving inclusion of other frequently underrepresented populations.

Public Awareness and Communication

Bierer emphasized the need to raise public awareness about clinical trials and increase public understanding of the importance of representative participation in research. The public, including those who have been historically underrepresented in clinical research, should be involved in developing and implementing a sustainable national public awareness campaign. “The first time people hear about clinical trials should not be when they’re asked to volunteer,” she said. Approaches may include an inventory and understanding of ongoing initiatives and sharing best practices across organizations that have experience engaging different communities on health-related topics to better “understand the context in which people find reliable and trustworthy information.” An affirmative approach to national messaging could “[include] narratives of lived experiences.” There will not be a one-size-fits-all solution, but sustainable ways to scale up successful dissemination efforts will be critical, she added.

Community Engagement and Investment

Community engagement and investment are essential to develop effective messaging and communication strategies and support clinical research that is of value to the community, Bierer stated. It may be challenging for organizations to establish enduring community partnerships and shift investment models from one-off project-specific funding to sustainable long-term investments. She suggested that organizations should explore approaches for investing in communities and having community partners involved at every stage in the process, from defining the research that matters to communities, to communicating results of the trials, to implementation. One component of building those relationships is establishing trust, which might be done through transparent communication about trial results, including returning results to participants in a way that is actionable.

Site Enablement

Hanger discussed the value of enabling the conduct of clinical trials at more research sites, including community practices. Recognizing that conducting research in some settings (e.g., primary care) can be difficult, she laid out some considerations for how to make it easier for sites that may not have the infrastructure and resources of a traditional academic clinical research center. Helping organizations better partner with and support

a breadth of possible sites can include focusing on building on what sites already have, enabling access to peer experts and technology, and considering their strengths, such as community trust and engagement. Another consideration is sustainable funding models that avoid underbudgeting.

Workforce Diversity

Hanger recognized that work is already ongoing to support a more diverse and representative clinical trials workforce and the importance of starting early with efforts to increase public awareness and understanding of clinical research as a professional field. Additionally, she pointed to the need to establish a workforce pipeline, which includes training opportunities that underrepresented populations can access. There are examples of these types of programs, but Hanger pushed for organizations to work together to assess what is working, measure progress over time, and figure out approaches for running successful programs at scale. She added that sustainable change for improving workforce diversity will also require ongoing support for those who are already in the clinical trials workforce within and across organizations.

Trial Participation and Access

Bierer noted that designing trials for equitable access may depend more on the actions of individual organizations than on collaborative efforts. She mentioned the importance of accessible participant-facing materials on trials (including gender-neutral language that has been user tested and translated as necessary), better optimization of decentralized clinical trial elements to improve accessibility and reduce participant burdens and ensuring reimbursement for transportation, childcare, and other expenses that may be needed to cover the cost of participation. Another consideration is a “fiduciary commitment” to a participant’s well-being as they transition out of trial. Additionally, there may be system-level approaches to better connect patients and their clinicians to available trials that meet their needs and interests.

Funding, Resources, and Support

Funding, resources, and support “undergirds every other piece” of the plan, Bierer said. Resource allocation for particular study activities has been shown to increase trial representation. She pointed to the role of insurance coverage for trial participation, compensation for community partners, and direct funding for community health centers to distribute as they see fit as approaches to consider for facilitating and sustaining more representative clinical trials.

Comprehensive Data

Hanger highlighted the need for interoperable and accountable systems for the collection, dissemination, and analysis of data on trial participation. This should include data standards and consistent terminology so that information is consistent and comparable across studies and systems. She also pointed to the important role for comprehensive epidemiological data. At the same time, she acknowledged the need to know “what good looks like” to ensure that trial enrollment accurately reflects the populations most impacted by diseases and conditions. For this reason, the action plan focuses on upstream considerations for achieving comprehensive and consistent data—understanding the variables for measuring and achieving representative trials (e.g., where these data come from, how they are collected and shared across systems).

Accountability

Accountability across all the domains is essential, Hanger said, to garner community support and ensure that commitments to communities are met. She stated that organizations need agreement on what they are working toward, how they intend to get there, and whether organizations live up to commitments they make toward achieving agreed-upon goals. Bierer encouraged participants to take collective action, hold each other accountable, and “carry the collective work forward.”

ORGANIZATION OF THE PROCEEDINGS

This Proceedings of a Workshop summarizes the presentations and discussions during the public workshop held on May 20, 2024. Chapter 2 considers strategies for equitable participation in clinical trials, including how to scale existing efforts. In Chapter 3, panelists discuss defining, collecting, and sharing condition-specific demographic clinical trial data to facilitate continuous learning and accountability. Chapter 4 considers how to better enable sites to conduct more inclusive and representative trials, including discussions of funding and workforce development. In Chapter 5, speakers from the Centers for Medicare & Medicaid Services (CMS), U.S. Food and Drug Administration (FDA), and National Institutes of Health (NIH) highlight federal agency efforts to encourage diverse clinical trials and opportunities for collaboration and harmonization toward this common goal. Chapter 6 concludes with panelist and participant comments on practical approaches to collaboration across sectors to advance more equitable and representative participation in trials across the clinical trial ecosystem.

This page intentionally left blank.