Envisioning a Transformed Clinical Trials Enterprise for 2030

A Perspective on the Use of Digital Health Technologies

Robert Califf from Verily Life Sciences reflected on the use of existing and emerging technologies for achieving the aspirations for a transformed 2030 clinical trials enterprise. He highlighted opportunities to advance a learning health system given improvements in the quality of electronic health records and claims data and the development of standards, common data models, and the automated curation methods. However, he cautioned that “digital technology can either be a rising tide that raises all boats if we make it equitable … or it can be used much like it is now in most of our health systems … to segment populations to optimize the situation for some people, particularly those who are already digitally enabled.”

A Perspective on the State of Clinical Trials in 2021

Elliott Levy of Amgen, Inc. suggested that a more patient- and society-focused clinical research enterprise aligns with a more efficient and productive for trial sponsors. Levy stated that, “clinical trial diversity can be increased. We already know how. What is most needed is simply the will and the discipline to systematically apply existing methods.” Improving trial efficiency can help reduce the burden of participation for patients and better support evidence-based decision-making.

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

Eliseo Pérez-Stable, National Institute on Minority Health and Health Disparities at NIH, pointed out that “having a diverse sample in a clinical research trial is good science… [T]here are questions that will be left unanswered if we stay with the easiest-to-recruit participants in a clinical research trial.”

Richardae Araojo, of the Office of Minority Health and Health Equity at FDA observed that the response to the COVID-19 pandemic has demonstrated that early-stage planning, appropriate site selection, and community engagement can enable more inclusive trials.

Megan O’Boyle, principal investigator of the Phelan-McDermid Syndrome Registry and parent of a child with a rare genetic syndrome, along with Pérez-Stable and Araojo, discussed the importance of engaging with patient advocacy groups to understand the needs and preferences of patients. Incorporating patient input when designing trial procedures and consent forms, finding the right messengers - local doctors, nurses, and community leaders who can speak in plain language to community members, and using culturally-tailored language, can enable more patient-centered trials and enhance participant recruitment and retention.

Forging Equitable Partnerships with Community-Based Organizations

Silas Buchanan, Institute for eHealth Equity, stated that “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.” “What we are most interested in,” Buchanan said, “is helping to equitably connect all stakeholders, helping recruit more principal investigators of color, and building something that acknowledges the past while moving together towards the future.”

For more information see: Driving Towards More Inclusive Clinical Trials by 2030: Action Without Strategy Is Aimless, Strategy Without Action is Powerless

Achieving Improved Outcomes While Reducing Patient Burden

Marilyn Metcalf, GlaxoSmithKline, shared a vision for health care in 2030 developed in collaboration with Rob Weker, a patient advocate. In their vision, artificial intelligence and digital networking would help provide comprehensive, proactive health services to the patient and the caregiver. Healthcare would encompass early detection of disease, shared decision-making, psychosocial services, expert medical care, and financial support. “Creating an equitable person-centered health care system is not only possible, but absolutely necessary for the well-being of all people,” Metcalf concluded.

For more information see: Transforming Clinical Trials: A New Vision for 2030

Understanding the Past to Inform Policy and Practice

Margaret Anderson, Deloitte, emphasized that understanding the past is necessary for developing future solutions. She described examples of past movements that have changed policy and practice in health care that took an “outside/inside” approach, such as staging large public demonstrations, while also working within organizations to develop and implement policy change. Anderson suggested that a similar approach could help achieve a more person-centered clinical trials enterprise. There is optimism that change is possible and already taking place. “Now, never like before, this issue has risen to the top, not just within the medical research community, but within the public discourse at large,” said Esther Krofah, FasterCures, Milken Institute.

Practical Applications for Technology to Enhance the Clinical Trials Enterprise

Jennifer Goldsack, Digital Medicine Society, said that an enhanced clinical trials enterprise for the future does not necessarily require more technology, but rather, more solved problems. Bradford Hirsch, SignalPath Research, suggested that technologies needed to drive change in the clinical trials enterprise already exist. What is lacking is coordination and an understanding of how to effectively use those technologies to advance clinical trials.

Tara Hastings, Michael J. Fox Foundation, pointed out that technology is “a two-way street” and it is not just about what trial participants may contribute to research, but also what the clinical trials enterprise can give back to participants and their providers. Digital health technologies can help inform patients about clinical trials and reduce the burden of participation, but it is not silver bullet solution for engaging more people in research. She emphasized the need for co-development of clinical trial technologies with the people the products are intended to serve. “People are much more willing to give their data when they have the feeling that they are getting something out of it” said Jan Benedikt Brönneke, health innovation hub of the German Federal Ministry of Health.

Collaboration in Action

Janice Chang, TransCelerate BioPharma, Inc. (TransCelerate) said there has been an “unparalleled willingness” by TransCelerate member companies to learn from each other and collaborate to identify solutions for trial continuity during the pandemic. Many of the tools, technologies, and processes that were implemented during the COVID-19 pandemic could be adopted more broadly across the clinical trials enterprise.

Technology-Enabled Decentralized Clinical Trials

Pamela Tenaerts, Medable, Inc. discussed applications for digital health technologies to enable decentralized clinical trials that ensure participant safety and patient-centricity; deliver reliable, actionable data to decision makers; and improve participant and site satisfaction with the clinical trial process. Additional data can help characterize the performance of technology-enabled decentralized clinical trials based on parameters such as participant safety, participant and site experience, data privacy, and data integrity.

Reflections on the Role of Technology in Clinical Trials

Andy Coravos, HumanFirst; Eric Perakslis, Duke Clinical Research Institute; and Sam Roosz, Crescendo Health; reflected on the thoughtful and responsible deployment of digital health technologies. Roosz pointed out that each interaction a patient has with the health system is an opportunity to foster trust in clinical trials, and identify potential areas of hidden bias or inaccessibility in these encounters. Coravos said that patients using digital health technologies should be able to trust that their information is secure, and a holistic approach to data governance should balance data security with usability of the technology and include nondiscrimination protections. Perakslis emphasized the importance of working with existing networks of doctors and community health workers, and emphasized the need for some overlap in the regulatory oversight of research and care. “To move clinical trials into the community, we have to create the right type of overlap so that we … have more fluency between research and care.”

For more information see: The Future of Clinical Trials: How Will New Technologies Affect the Lives of Participants?

Building a More Resilient, Sustainable, and Transparent Clinical Trials Enterprise

For the clinical trials enterprise to be sustainable it has to be worthwhile and practical for frontline health care providers who care for underrepresented populations to participate as investigators. Bárbara Segarra-Vázquez from the University of Puerto Rico added that the sustainability of the clinical trials enterprise requires informing the public about trials, listening to patients, and dispelling myths about the willingness of diverse populations to participate in trials. She said “we have to train our young investigators to trust patients, talk to patients, and listen to patients.” It will be impossible to achieve health equity until the representation of certain populations and groups in trials is improved, said Elizabeth Ofili from the Research Centers in Minority Institutions Coordinating Center.

Martin Landray from the University of Oxford discussed the Randomized Evaluation of COVID-19 Therapy (RECOVERY) Trial as an example to highlight the importance of conducting randomized clinical trials and to demonstrate how clinical trials can be a core component of clinical care. Beyond providing clear results on clinically important treatments for severe COVID-19, participating in the RECOVERY Trial inspired some junior doctors to become active in clinical research. “Clinical trial[s are] part of good quality clinical care; they are not an optional extra,” Landray said.

Tenaerts described the approach taken by the Clinical Trials Transformation Initiative to create frameworks and operational tools to support change and improve clinical trials. She suggested that simply issuing recommendations may not be sufficient to change behavior. However, providing operational tools can aid stakeholders in implementing policy and practice recommendations to improve clinical trials.

Building Trust

Brian Southwell, RTI International, said that clinical trials necessarily intersect with the public sphere, so developing a person-centered communication approach requires an understanding of what trust and transparency mean to different people. He described a future vision in which “trials are advancing science by enrolling people who are the most directly impacted by diseases.”

For more information see: A Future of Trusted Clinical Trials: Communication Strategies to Encourage Trust and Transparency

Dyan Bryson, Inspired Health Strategies, illustrated people’s relationships outside of the formal health care system, including how family, friends, coworkers, classmates, and others with whom they have trusted relationships most influence their health-related decisions and habits they adopt.

Freda Lewis-Hall, retired from Pfizer, observed that a fresh, unencumbered perspective combined with the purposeful use of existing resources and networks are both needed to build out a community-based clinical research infrastructure.

Opportunities for Transformation

Mark McClellan, Duke-Margolis Center for Health Policy, outlined two approaches for building community trust in the research enterprise: 1) simplifying existing trial networks to reduce the burden on community health care providers, and 2) leveraging capacity outside of the clinical trials enterprise, such as electronic registries that already track longitudinal patient data for quality improvement purposes. He suggested that there are opportunities to facilitate change in the clinical trials enterprise by building upon ongoing public health policy activities (e.g., PDUFA reauthorization).

Amy Abernethy, Verily Life Sciences, stated that, “2030 is now. Our vision for the clinical trials enterprise is unfolding in front of us in 2021 and going forward.” The clinical trials infrastructure exists to generate the clinical evidence needed to make decisions about health-related interventions, she said, and new innovations must work within the clinical trials ecosystem while still ensuring patient safety and data integrity. The technology industry can serve as a partner - not just a vendor – as stakeholders consider how the clinical trials enterprise should evolve in terms of technical and data capabilities.

In a public health emergency, the goal of the clinical trials enterprise should be to rapidly generate robust and actionable data that can be used to improve standards of care and disease outcomes, said Janet Woodcock, FDA. However, the response of the U.S. clinical trial ecosystem to the pandemic has been less than optimal. The creation of a community-based clinical trials network is as essential to pandemic preparedness as ensuring the availability of personal protective equipment, and as such, should be a government-supported activity. She stated that, “we need a national clinical trial capacity stockpile, just as we need a stockpile of medicine and equipment.”