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Enhancing Outcomes in a More Person-Centered and Inclusive Clinical Trials Enterprise

This segment of the four-part workshop focused on transforming the clinical trials enterprise to be more person-centered, inclusive, and equitable by 2030. Participants discussed priorities and actions for achieving this goal and how to engage stakeholders, including the public, in this effort.

THE ROAD TO 2030: PERSPECTIVES FROM THE FIELD

Frontline Experience: A Panel Discussion

In this session, panelists shared their perspectives on what is needed for the clinical trials enterprise to move toward greater person-centeredness over the coming decade. Panelists included Eliseo Pérez-Stable, director of the National Institute on Minority Health and Health Disparities (NIMHD) at the National Institutes of Health (NIH); Richardae Araojo, associate commissioner for Minority Health and director of the Office of Minority Health and Health Equity (OMHHE) at FDA; and Megan O’Boyle, principal investigator of the Phelan-McDermid Syndrome Registry. The session was moderated by Esther Krofah.

The Rationale for Inclusiveness

Pérez-Stable said increasing the inclusiveness of clinical trials is a priority. “Having a diverse sample in a clinical research trial is good science,” Pérez-Stable said. “There are questions that will be left unanswered if we stay with the easiest-to-recruit participants in a clinical research trial.” Furthermore, some groups experience disproportionate burdens from particular diseases. For example, he said that because COVID-19 is disproportionately impacting African American and Latino individuals, it may make sense to oversample them for related clinical trials. He acknowledged the challenge of achieving balanced representation in every study, but said that having at least some diverse participation in studies can lead to new discoveries and treatment approaches.

Araojo mentioned the 2012 FDA Safety and Innovation Act (FDASIA), which directed the agency to study the participation and analysis of demographic subgroups in clinical trials and to create a plan to support inclusive clinical trials.¹ One product of FDASIA was the FDA Center for Drug Evaluation and Research’s Drug Trials Snapshots program. Araojo shared some specific findings from a recent Snapshots report summarizing participant demographics in clinical trials of new products from

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¹ See https://www.fda.gov/regulatory-information/food-and-drug-administration-safetyand-innovation-act-fdasia/fdasia-section-907-inclusion-demographic-subgroups-clinical-trials (accessed July 1, 2021).

2015 through 2019.² Of the nearly 300,000 trial participants during this time frame (from both U.S. and ex-U.S. trial sites), 76 percent were white, 11 percent were Asian, 7 percent were Black or African American, and about 1 percent were American Indian or Alaska Native (5 percent were designated as other). When only participants enrolled at U.S. sites were assessed (about 102,000), the demographics shifted to 16 percent Black or African American, 2 percent Asian, and 1 percent American Indian or Alaska Native (FDA, 2020b).

O’Boyle observed that there is little diversity in clinical trials for rare diseases, unless the disease is known to be highly prevalent in a particular ethnic or racial group. She suggested that diversity in rare disease trials could be improved if providers referred more patients for genetic testing.

The Role of the U.S. Food and Drug Administration

OMHHE at FDA “works to protect and promote the health of racial and ethnic minority, underrepresented, and underserved populations [through] research, outreach, and communication that works toward addressing health disparities,” Araojo said.³ Activities within the agency’s current authorities include, for example, supporting intramural and extramural research; implementing culturally and linguistically appropriate strategies, tools, programs, initiatives, and campaigns; and issuing guidance documents. Improving diversity in clinical trials is a key priority for FDA, and Araojo referred participants to a recently released guidance for industry, Enhancing the Diversity of Clinical Trial Populations—Eligibility Criteria, Enrollment Practices, and Trial Designs.⁴ The guidance addresses methods to improve trial recruitment so those enrolled reflect those who will ultimately use the product being studied.

Araojo said FDA is committed to “consistent, continued, bidirectional community engagement” to advance inclusiveness and to help overcome the barriers to more representative participation of racial and ethnic minority populations in clinical trials. The agency will continue its efforts to reduce the burden of trial participation, and will apply the lessons learned, as appropriate, during the COVID-19 response about the use of new technologies, tools, techniques, real-world data, and other advances.

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² For the 2015–2019 Drug Trials Snapshots Summary Report, see https://www.fda.gov/media/143592/download (accessed July 1, 2021).

³ For more information about OMHHE at FDA, see https://www.fda.gov/about-fda/office-commissioner/office-minority-health-and-health-equity (accessed July 1, 2021).

⁴ See https://www.fda.gov/regulatory-information/search-fda-guidance-documents/enhancing-diversity-clinical-trial-populations-eligibility-criteria-enrollment-practices-and-trial (accessed July 1, 2021).

Engaging the Community to Reduce Barriers to Enrollment

O’Boyle spoke from her perspective as the principal investigator of the Phelan-McDermid Syndrome Registry and as the parent of a child with a rare genetic syndrome. She described the current clinical trials enterprise as out of alignment with the way people use and share personal data in 2021. IRBs are providing protections that participants do not necessarily want, and required consents often do not allow participants to share identified data, she said. Furthermore, much of what is included in consent forms is unnecessary, frightening to participants, or redundant. Forms are lengthy and seem to be written for the benefit of corporate lawyers rather than patients, O’Boyle said. “Short, concise, honest” forms are needed, and patient groups should be approached to review consent forms before IRB approval. O’Boyle said that sponsors should also seek input from patient groups on protocols and schedules (e.g., Would treatment be better tolerated before or after a meal? Will travel to appointments be a financial burden?). Patient input can inform the development of more patient-centered trials, which can lead to better participant retention.

O’Boyle said that time, money, and lives are being wasted as a result of “overprotection” by IRBs and a lack of inclusion of diverse patients in studies. She stressed that this message must come from patients and research advocacy organizations. Any effort by the researchers to address this would appear self-serving. Patient communities need education about clinical trials so they are empowered to speak up and communicate to trial sponsors their interests and concerns about protocols and consent forms.

Pérez-Stable emphasized the importance of understanding patient needs when designing trial procedures and the consent form. He agreed that consent forms need to be more user-friendly for participants, not just in length and content, but also language and reading level. Protocols or consent forms that are not acceptable or understandable to patients can be a barrier to recruiting a diverse population, and experience has shown that community engagement is an effective method for developing long-term connections with diverse populations.

Araojo highlighted the need for engaging patient advocacy groups to learn how to make trials less burdensome with regard to trial design, logistics, recruitment, and retention; engaging cultural ambassadors, faith-based organizations, and trusted leaders in the community; and eliminating language barriers. Community engagement is not just informing the community, it is also understanding their needs with regard to trial participation.

Another barrier to recruitment is a general lack of awareness of the availability of clinical trials, Araojo said. With COVID-19, the public was

aware that trials were being launched and that volunteers were needed, and that diverse participation was especially needed due to the disproportionate impact of COVID-19 in racial and ethnic minority communities. A lesson from the response to the COVID-19 pandemic is that inclusivity in trials can be achieved when there is appropriate attention to overcoming barriers through early-stage planning, appropriate site selection, and community engagement.

Krofah asked panelists to comment on the concerns that including patients in discussions of trial design and working to increase diverse participation slows the process and adds expense. O’Boyle said that failure to recruit or retain trial participants slows the process and increases expense. “If you do not design [trials] with the patients and families in mind, then you are not going to retain them,” she said. She advocated for engaging with patients and their caregivers even earlier, prior to selecting a target and defining product attributes or delivery mechanisms, to understand what their most pressing disease-related concerns and quality-of-life issue are.

Best Practices

Pérez-Stable said that planning for representative trial participation should be done early, and he suggested that experts might reach out to the contract research organizations recruiting in areas with minority populations to motivate them. He shared his experience working with the Operation Warp Speed⁵ leadership to increase their outreach to diverse communities. One of the COVID-19 vaccine sponsors, for example, created a website for people to register their interest in joining the trial. However, participants from all demographics did not visit the website in proportionate numbers right away, he said, and recruiters initially failed to reach out to many people from diverse communities who did register. The sponsor did understand the importance of diverse enrollment, he continued, and ultimately paused recruitment of white participants to achieve better representation of minority populations.

Pérez-Stable emphasized the importance of finding the right messenger to reach out to diverse communities. Too often recruiters believe that minorities are not interested in clinical trials, he said, or that they will only participate in trials if a religious leader or a celebrity or athlete endorses the trial. The most powerful messengers are actually local doctors, nurses, and community leaders who can speak in plain language to community members. The panelists also emphasized the importance of investing in

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⁵ Operation Warp Speed was a public–private partnership to accelerate the development of vaccines for COVID-19.

culturally tailored and linguistically competent messages and literature to share with patients from many backgrounds.

Krofah noted that community outreach and efforts to educate the public about clinical trials are chronically underfunded. Pérez-Stable observed that academic clinical research has been gradually moving toward early community engagement and said that industry has come to understand the value of investing in community engagement. He cited the COVID-19 Prevention Network (CoVPN)⁶ run by the National Institute of Allergy and Infectious Diseases as one positive example of COVID-19 clinical trials recruiting diverse participants. Pérez-Stable added that the NIH Community Engagement Alliance (CEAL) Against COVID-19 Disparities⁷ is investing community engagement, providing resources, and partnering with communities to develop and disseminate accurate information about COVID-19 disease, clinical trials, and vaccination.

THE ROAD TO 2030: VISIONS OF WHAT IS POSSIBLE

In this session, Silas Buchanan, chief executive officer of the Institute for eHealth Equity, shared several examples of how the Institute for eHealth Equity is working to create equitable partnerships with community organizations. Marilyn Metcalf, senior director of patient engagement at GlaxoSmithKline, discussed the potential for technology tools to improve patient outcomes and reduce patient burdens. Margaret Anderson, consulting managing director of strategy and analytics at Deloitte, described lessons from health movements of the past that can be brought forward to effect change for the future. The session was moderated by Luther Clark, deputy chief patient officer and global director for scientific, medical, and patient perspective at Merck & Co.

Forging Equitable Partnerships with Community-Based Organizations⁸

“A more inclusive clinical trials enterprise in 2030 will largely be defined by the number of equitable partnerships … created with underserved, faith, and community-based organizations,” Buchanan began.

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⁶ For more information about CoVPN, see https://coronaviruspreventionnetwork.org (accessed August 3, 2021).

⁷ For more information about the NIH CEAL program, see https://covid19community.nih.gov (accessed July 20, 2021).

⁸ This presentation is based on a blog post titled Driving Towards a More Inclusive Clinical Trials Enterprise by 2030: Action Without Strategy Is Aimless and Strategy Without Action Is Powerless, available at https://www.healthaffairs.org/do/10.1377/hblog20210503.43985/full (accessed July 1, 2021).

This strategy for inclusiveness depends on building trusted relationships with the leaders of these organizations, whom he said serve as conduits between underserved community members and the health care system. These types of community-based organizations have a wealth of experience addressing social determinants of health, Buchanan noted. Religious organizations, for example, have a long history of addressing food insecurity and other personal needs (e.g., soup kitchens, food pantries, clothing drives, transportation to health care appointments, daycare and after-school programs, adult education/GED classes). As discussed by workshop speaker Terris King, formerly of CMS (see Chapter 2), conversations about health also take place at barbershops and beauty salons, which are trusted community institutions where people feel safe discussing their personal concerns. Buchanan observed that the clinical trials enterprise has long underestimated the importance of equitably partnering with these trusted organizations as emissaries to the community. He cautioned, however, that it is not as simple as just reaching out to a church for a particular clinical trial. These relationships need to be nurtured over time.

As an example of how to begin reaching out to underserved communities, Buchanan described launching a Healthy Eating Active Living (HEAL) campaign. With a grant from the Aetna Foundation, the Institute for eHealth Equity partnered with five churches in Atlanta, Georgia; Dallas, Texas; and Columbus, Ohio, to co-create a HEAL campaign. Buchanan emphasized that they did not arrive with a fully developed campaign and tell the community what to do. Rather, decisions about aspects such as content, language, and images were community-driven, and endorsed by the participating faith-based organizations.

The campaign was facilitated by SMS text messaging. After the pastor spoke briefly to the congregation about health, they could text “healthy” to a short code phone number and begin answering a series of demographic and health-related questions. The HEAL campaign then messaged the 2,500 participating community members three times each week with additional information and questions. Over the course of 6 months, the response rate to the questions was 43 percent and, importantly, Buchanan said, no one left the program. Key elements of success, he said, were having each pastor’s blessing to launch the campaign, and gathering feedback and discussing next steps in weekly private meetings with the health ministry teams. Buchanan noted that about 35 percent of African Methodist Episcopal (AME) churches have a health ministry team, which usually includes members of the congregation who are current and retired nurses and doctors. Decisions about the HEAL campaign were driven by them as the experts on their community.

The relationships built through the HEAL campaign led to a partnership with the AME Church to launch AMECHealth.org,⁹ which Buchanan said is now the official channel for dissemination of health information for the AME Church. The website includes both publicly available information and a password-protected social network for the leadership of AME congregations, which the Institute for eHealth Equity uses to facilitate equitable collaboration and data sharing for health campaigns. He noted that many major health programs designed to reach African Americans through faith-based organizations collect data, but do not share data back in a lay format that the organizations can use (e.g., to apply for grants).

The Institute for eHealth Equity is also launching Our Healthy Community teams, a social network for community-based organizations designed to “shorten the distance” between the community and the clinical trials enterprise, health care providers, payers, and other stakeholders in health. Buchanan added that the Institute for eHealth Equity was recently selected by the Morehouse School of Medicine to participate on the National Advisory Board for the National COVID-19 Resiliency Network. They are developing a co-branded campaign with Morehouse, again working directly with faith-based organizations to ensure their input is included.

“What we are most interested in,” Buchanan concluded, “is helping to equitably connect all stakeholders, helping recruit more principal investigators of color, and building something that acknowledges the past while moving together toward the future.”

Achieving Improved Outcomes While Reducing Patient Burden

Metcalf shared a vision for health care in 2030 developed in collaboration with Rob Weker, a patient advocate, and based on input from patients.¹⁰ In this vision, a patient’s well-being would be monitored, to the extent they desired, making use of artificial intelligence and digital networking to provide comprehensive, proactive health services to the patient and the caregiver. Health care would ideally encompass early detection of disease, shared decision making about options, psychosocial support, expert medical care, and financial support, Metcalf said, with the goal of “achieving optimal outcomes with as little added burden to the patient as possible.”

The technical capabilities to achieve this vision exist or are being developed, Metcalf said. She pointed out, however, that patients who

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⁹ For more information, see http://amechealth.org (accessed July 20, 2021).

¹⁰ This presentation is based on a blog post titled Transforming Clinical Trials: A New Vision for 2030, available at https://www.healthaffairs.org/do/10.1377/hblog20210503.897529/full (accessed July 1, 2021).

have access to specialty medical facilities and have comprehensive insurance coverage are most likely to benefit. It is important to “consider the patients who are not well insured, who do not have physicians, or whose physicians do not have familiarity with or access to clinical trials and cutting-edge therapies,” Metcalf said.

Issues such as infrastructure, access, equity, and privacy are systemic issues that cannot be addressed effectively by one segment of the health system in isolation, she added. Furthermore, technical capability alone will not achieve this vision for 2030. Technologies are tools, and their accessibility and appeal to patients varies.

Achieving this vision requires an integrated health care system with shared purpose and shared information. The translation of research into clinical practice can be supported by prioritizing patient involvement in drug R&D and regulatory decision making when it comes to early disease detection, disease management, and treatment, Metcalf said.

Metcalf referred participants to a prior workshop of the National Academies’ Forum on Drug Discovery, Development, and Translation on Advancing the Science of Patient Input in Medical Product Research and Development¹¹ (NASEM, 2018). Discussions at that workshop highlighted the importance of designing trials from the start with patient needs and preferences in mind, and gathering input on participant trial experiences, including the experiences of participants who drop out of studies. Although some progress has been made in forging closer partnerships between patients and the health system, much work still needs to be done. “Creating an equitable person-centered health care system is not only possible, but absolutely necessary for the well-being of all people,” Metcalf concluded.

Advocating for Change: Learning from Past Movements That Changed Policy and Practice

Anderson reflected on how the current clinical trials system was formed by events of the past. In particular, she described how unethical practices in medical research, such as the U.S. Public Health Service Syphilis Study at Tuskegee, and the widespread use of Henrietta Lacks’s cells without her knowledge or consent, led to mistrust of the medical research system. Citing work by the Pew Research Center, Anderson shared data showing how public trust in the scientific community has remained fairly stable since the 1970s, while public trust in government

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¹¹ For more information on this workshop, see https://www.nationalacademies.org/ourwork/advancing-the-science-of-patient-input-in-medical-product-rd-towards-a-researchagenda--a-workshop (accessed July 20, 2021).

has declined steeply in the same time period.^12,13 Moving forward, she said that stakeholders across the clinical trials enterprise need to better explain the usefulness and value of clinical research for the practice of medicine and clinical care. In doing so, it is important to remember that “there is deep pain throughout the research system. These are real people, real lives, real diseases. It is important for us to honor that,” Anderson said.

Understanding the past is necessary to develop the solutions needed for the future, and Anderson described several examples of movements that changed policy and practice in health care. In the late 1980s, during the early days of the HIV/AIDS epidemic, activists took an “outside/inside” approach to effecting change by staging large public demonstrations outside that demanded attention from government, while also working with science and policy experts to propose specific policy changes from inside organizations. This strategy was also deployed by the Society for Women’s Health Research in the mid-1990s to mandate the inclusion of women in clinical trials. For the inside component, they approached female members of Congress to call for a U.S. Government Accountability Office review of the status of inclusion of women and minorities in research, the results of which helped to facilitate policy changes.

Anderson suggested that a similar strategy could be used for achieving and maintaining accountability for more person-centered clinical trials. Building off the movements described above, a range of organizations and actions have been driving change toward patient-centric clinical trials over time, for example, venture philanthropy organizations and foundations that fund research (e.g., the Cystic Fibrosis Foundation), patient cohorts (e.g., PatientsLikeMe, All of Us), nonprofit research organizations (e.g., Patient-Centered Outcomes Research Institute [PCORI]), agencies (e.g., FDA), and legislation (e.g., the 21st Century Cures Act). Anderson pointed out that these activities moved forward while information and methodologies about patient-centricity were still emerging. There was the will to seek change, she said.

In closing, Anderson listed some of the lessons to take forward.

• Meeting people where they are.
• Taking action without fear (“passion plus fearlessness equals change”).
• Using an outside/inside strategy to exert pressure on the system.

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¹² See https://www.pewresearch.org/politics/2021/05/17/public-trust-in-government-1958-2021 (accessed April 13, 2022).

¹³ See https://www.pewresearch.org/fact-tank/2020/08/27/public-confidence-in-scientists-has-remained-stable-for-decades (accessed April 13, 2022).

• Gathering a coalition of the willing. “[Think] broadly about who else needs to be brought [into the clinical trials enterprise] and give them assignments,” she said.
• Using disruption as a wedge (e.g., leverage the lessons from technology use in the COVID-19 pandemic response).
• Providing appropriate resources and funding for those organizations that are doing the work and reaching out to communities.
• Developing a pipeline of diverse scientists, clinical researchers, and health care providers.

Short-Term Goals to Foster a More Person-Centered and Inclusive Clinical Trials Enterprise: Panel and Breakout Discussion Highlights

Following the panel discussion, workshop participants were divided into virtual Zoom breakout rooms to consider short-term, tangible, and measurable goals and actions that could help ensure a more person-centered and inclusive clinical trials enterprise, and to discuss technologies, tools, and techniques that could be used to enhance inclusiveness and equity in clinical trials. Upon reconvening in plenary session, Krofah and several participants reflected on the panel and breakout group discussions and highlighted the following themes:

• Investing in community outreach and engagement. Relationships with the community need to be cultivated and maintained. Participants discussed funding community-based organizations, providing education and training for community members and leaders, compensating community leaders and partners for their time, and returning value to the community, Krofah reported.
• Educating the community about clinical trial opportunities. Jacqueline Alikhanni, patient ambassador at PCORI and trial participant, suggested that many patients would participate in clinical trials if they were better informed about what clinical trials are, what opportunities are available, and how to enroll. Educating communities about trials, especially communities of color, would help to foster trust, she said, and could help to overcome reservations about participating in trials that have resulted from a long history of negative experiences with the medical establishment.
• Engaging patients at the beginning of the trial process to ensure that participation is meaningful. For example, patients should be asked to provide input on therapeutic targets and outcomes of importance to them, and on the acceptability of elements of protocols and consent forms, Krofah reported. The need to balance what

• is meaningful to trial participants versus what is legally required in consent forms was noted.
• Rethinking data-sharing practices. Krofah summarized a point made by O’Boyle that, in some cases, trial participants might prefer to have informed consent agreements that permit sharing of identifiable information. Information not related to health is being shared constantly, such as with social media or streaming services. Some trial participants might choose to similarly share their health information if it could help develop meaningful treatments more efficiently.
• Clearly defining what is meant by community. “Different stakeholders define community differently,” Krofah said. Identifying trusted leaders in the defined community who can be partners and spokespersons is also important.
• Identifying appropriate metrics to assess progress in establishing a more person-centered and inclusive clinical trials enterprise. End-to-end visibility is needed with regard to diverse patient enrollment across trials while still preserving patient privacy and conforming to regulations, Krofah said.
• Mentoring principal investigators. Elena Rios of the National Hispanic Medical Association said that physicians involved in research need to serve as mentors to the next generation of clinical trial investigators. She added that many experienced investigators are associated with academic health centers while many new investigators are community based.
• Considering social determinants of health in inclusiveness. Barbara Bierer, director of the Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard University, said that information on social determinants of health is needed to inform diverse trial enrollment efforts, and that good, quick indicators of social determinants of health are needed for use in data collection (versus extensive, often uncomfortable, questioning of patients). Krofah agreed and added that “the full experience of an individual [influences] whether or not they even have the opportunity to understand and participate in clinical trials and research.”
• Developing a national, cooperative effort to educate stakeholders about inclusivity in clinical trials. Bierer suggested that the National Academies consider which aspects of improving inclusivity might be addressed cooperatively, at a national level, rather than organizations developing uncoordinated, individual efforts to educate about inclusivity.

Long-Term Goals to Foster a More Person-Centered and Inclusive Clinical Trials Enterprise: Panel and Breakout Discussion Highlights

Workshop participants considered longer-term, tangible, and measurable goals and actions that could ensure a more person-centered and inclusive clinical trials enterprise, and discussed technologies, tools, and techniques that could be used to enhance inclusiveness and equity in clinical trials. Upon reconvening in plenary session, Clark reflected on the panel and breakout group discussions and highlighted the following themes:

• Acting with urgency. Although the breakout discussants were charged with discussing actions and goals for the next 10 years, Clark reported that several breakout discussants emphasized that the importance of the issues warranted quick actions to begin making progress toward the stated goal and meeting any interim milestones as soon as possible.
• Focusing on earlier, broader, and consistent community engagement. The importance of community engagement was a key theme of the discussions, Clark said, including the benefits for both researchers and participants of engaging communities earlier in the clinical trial process. It was suggested that local health equity initiatives are an underused resource for continuous community engagement, and that clinical trials should be connected to these groups.
• Moving the clinical trials enterprise into health care settings. Breakout participants discussed the importance of developing robust clinical trial networks within communities, and investing in community-based trial infrastructure for the long term. Approaches might include: establishing sustainable funding models for community health workers, providing training opportunities for individuals working across the health care team to help patients make more informed decisions about trial participation, and sustaining long-term relationships between community leaders and the health care teams.
• Advancing the consent process. Participants discussed ways in which technology could be used to make consent forms and the consent process more interactive, more meaningful and patient friendly, and potentially virtual.
• Collecting information on social determinants of health. In addition to the usual demographic information, better information is needed on the social determinants of health impacting potential

• trial participants. Collecting the latter information should not create additional burden for the trial participant, Clark said, and breakout discussants suggested that the clinical trials enterprise connect with community partners that are already collecting this information as they work to address these issues.
• Identifying the problems that could be solved with technology. Breakout participants discussed how technology could help overcome many of the barriers to more person-centered and inclusive clinical trials, Clark reported. There was discussion of the need to address the “digital divide” and to ensure that patients with limited access to technology or technology literacy are not excluded. General areas discussed in which digital health technologies could help improve patient-centeredness and inclusiveness included
• Raising awareness about clinical trials. Technology can be an effective tool to disseminate reliable, high-quality, credible information about clinical trials, Clark said. It was also noted that technology can be leveraged to help foster trust in the clinical trials enterprise if trusted community voices are delivering the messages.
• Increasing access to clinical trials. Breakout participants discussed how to leverage technology to decentralize clinical trials and expand the population that can participate. Mobile technologies can be used to reach those living in rural and remote areas and others who face barriers to traveling to a clinical trial site, Clark said. Cell phones, for example, are now widely available even in the most remote parts of the world.

REFLECTIONS ON ACHIEVING PERSON-CENTERED AND INCLUSIVE TRIALS

Steven Galson and Krofah noted that an underlying theme throughout this part of the workshop was that the issues of person-centeredness and inclusivity in clinical trials have been discussed for decades and the time has come to take action, employ new approaches, and make progress. To close this part of the workshop, they summarized some of the key messages they heard during the discussions.

Person-Centeredness and Inclusiveness

• “If the patients are not at the center of our work, then who is?” Galson said, paraphrasing O’Boyle. To move forward, it is necessary to understand where and why there has been resistance to engaging patients and their caregivers in the clinical trials process.

• Communities need information about what clinical trials are and the advantages of participating, Galson summarized. It is a misperception that certain minority populations do not want to participate in clinical trials.
• Ease of recruitment often drives who is recruited for a given clinical study and, as discussed by Pérez-Stable, this does not represent the best science.
• The population enrolled in a trial does not necessarily reflect the population most burdened by the disease under investigation, Galson said. A summary of participant demographics that was discussed by Araojo showed that 16 percent of trial participants in the United States were Black or African American and 2 percent were Asian.
• There is also a need to engage and prepare a more diverse clinical research workforce, especially at the physician/principal investigator level. Participants discussed the “failure of medical education to significantly increase the diversity [of the] physician workforce in the United States,” Galson said.

Envisioning and Effecting Systemic Change

• There is optimism that change is possible and already taking place. “Now, like never before, this issue has risen to the top, not just within the medical research community, but within the public discourse at large,” Krofah said.
• A systemic, enterprise-level, cooperative approach is needed to improve inclusivity in clinical trials, Galson reported, rather than the many disparate efforts by individual organizations that are currently occurring. There are models to scale and best practices to be shared, Krofah added. The clinical trials enterprise needs to move beyond “islands of excellence” to “a whole ecosystem of excellence for all people,” she said.
• How to effect change was a topic across breakout group discussions, including the roles of mandates and enforcement, incentives, investments and capacity building, accountability, and the will to make change. More discussion is needed on the role of FDA in advancing inclusiveness in clinical trials, Galson suggested, such as the extent to which the agency has the authority to mandate changes.
• Learning from the ongoing response to the COVID-19 pandemic was a recurring topic of discussion. For example, Krofah asked, how can the infrastructure, networks, and collaborations be sustained and expanded to address other disease conditions that disproportionately affect particular communities?

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