Advancing Clinical Trials at the Point of Care (ACT@POC)
Advancing Clinical Trials at the Point of Care (ACT@POC)
New from ACT@POC
Enhancing Integration of Clinical Research with Clinical Care:
Empowering Health Systems to Lead on Evidence Generation
America is experiencing a life-threatening, backward slide in health. Overall life expectancy in the United States declined by 2.7 years from 2019 to 2021, the largest two-year drop in almost a century. Addressing this sharp decline will require health systems to lead the next wave of clinical evidence generation for improving disease outcomes.
Gaps in the US clinical research evidence generation system leave the nation vulnerable to health emergencies while also impeding its ability to efficiently produce practical, relevant evidence. Advancing point-of-care trials enables randomized trials in the real world to answer priority research questions while affording patients and providers an experience aligned with normal clinical care.
While the U.S. Food and Drug Administration (FDA) leadership has recognized challenges in the current evidence generation system, overcoming many of the hurdles to widespread use of point-of-care trials—especially for regulatory decision-making—means addressing both real and perceived policy barriers.
In November 2021, leaders of health care organizations launched the Coalition for Advancing Clinical Trials at the Point-of-Care (ACT@POC) in response to the continuing large and avoidable gaps in timely evidence to inform patient care. Today, the work of this coalition continues. In an open letter, ACT@POC’s members describe our mission to support larger-scale, more efficient clinical trials. We outline ACT@POC’s principles for its operational priorities, including engaging practicing clinicians in a broader range of settings for clinical research, supporting the development of digital tools, and assuring that clinical trial designs are fit for purpose to surface real-time, real-world information about the effectiveness of available therapies.
ACT@POC’s recent actions to support its mission include:
- Providing technical assistance and recommendations to the Office of Science and Technology Policy, Advanced Research Projects Agency for Health, FDA and other policy leaders to advance point-of-care trial approach networks in important areas of evidence gaps
- Convening a major public workshop with health system leaders, Federal government stakeholders, frontline clinicians, and other organizations instrumental in achieving broader participation in clinical trials
- Launching Coalition working groups focused on addressing key challenges, such as regulatory barriers and data interoperability, to advance these issues and support dialogue with senior leaders throughout the health ecosystem
- Highlighting the Coalition’s work in white papers and a Health Affairs Forefront blog
Through our work, we have identified recommended actions for health systems to enact a culture that encourages and supports integrated clinical research at the point-of-care. Our coalition/ACT@POC will work to advance these recommendations, develop supporting resources for other clinical research stakeholders, and launch and leverage pilot point-of-care trials to improve evidence generation systems for patients, providers, health systems, and all stakeholders.
- Work with community leaders on research priorities and engage with patients to foster patient trust in clinical research. Patients may be reluctant to enroll and trust the results of trials that are disconnected from the health needs of the community.
- Permit direct contact with potential research participants to broaden awareness of and interest in clinical trial participation and enhance recruitment. Health system patients already have a relationship with our providers, and patient recruitment policies can be designed to both protect privacy and expand access to trials.
- Provide capacity and seek opportunities for external funding (e.g. from sponsors or payers) to support dedicated research time for providers to participate in clinically relevant research, along with opportunities to investigate research questions of mutual interest.
- Frontline providers often lack time to collect additional patient information and needed trial data as well as complete trial oversight and compliance activities. Dedicated research time should be paired with steps to simplify participation in research and streamline the ability to collect usable structured data at the point-of-care.
- Work with legal teams to adopt a risk-proportionality framework for indemnity agreements and Institutional Review Board (IRB) review based on considerations including the known safety data for the investigational therapeutic, the phase of the clinical trial, the unmet patient need, and the desired trial enrollment. Organizational risk management processes for research (e.g., IRB oversight, liability insurance) should be better aligned, simplified, and modularized with the risk proportional to the trial type and therapy in question.
- Support clinical research infrastructure and digital technologies that reduce burden on providers and create processes for generating evidence from data collected in point-of-care trials. Coordination between private and public stakeholders can provide sustainable funding for real-world evidence generation.
- Improve the interface between local and central/single IRBs through a timely and effective process to address potential concerns while improving efficiency. Multi-site trials that use a central or single IRB may face uncertainties around mutual decision-making between local health system IRBs and the central/single IRB.
- Work with trial sponsors and supporting organizations to help define staff responsibilities and roles for engaging in research. Staff providing routine care should be able to support point-of-care trials without being classified as research personnel and having the added burdens that result. Recent FDA draft guidance on decentralized trials distinguishes local health care providers from study investigators. However, additional considerations may still be needed to minimize staff and site burden. Health systems have a critical perspective to shape those considerations.
- Establish policies that address ethical challenges for staff supporting point-of-care research. Combining clinical research and care poses new challenges that require a rethinking of traditional research ethics. Patients and providers need clear expectations surrounding trial participation activities, including during informed consent. Clear guidelines for the ethical conduct of point-of-care research can maintain and build patient trust. Such ethical conduct guidelines could build off existing conflict of interest policies for providers and should not add additional burdens for them.
As health care leaders, our patient care mission demands better evidence. We are committed to this mission. Creating a culture of participation is only a first step in facilitating a necessary transformation to better integrate clinical care and clinical research, but it is a critical foundation for further progress. Better coordination and integration between care and research has the potential to close evidence gaps for patients with chronic and rare diseases as well as address key questions related to value in the provision of health care. It can also help achieve much needed increased representative enrollment in clinical trials and increased patient trust in clinical research through better engagement of their primary providers. Our goal is the formation of a learning health system that provides better care for all patients by generating practical evidence on how to improve disease outcomes that matter to patients and help reverse the downward trends in U.S. life expectancy.
The Advancing Clinical Trials at the Point of Care (ACT@POC) coalition will generate quality clinical research evidence in real time to better evaluate treatments and therapeutics, including those to treat COVID-19. The Coalition will engage a broader, more diverse group of patients and providers and develop digital health tools that make clinical trials simpler to run and more accessible to patients.
Currently, the complexity and cost of traditional clinical trials pose obstacles to patient and provider participation, identification of effective treatments for diseases, and the acceleration of new clinical insights and knowledge. This multi-stakeholder coalition aims to drive implementation of large-scale clinical trials at the community level—in the doctor’s offices and care facilities where most of the U.S. population receives care.
“The vast majority of patients and their providers do not participate in clinical trials,” said Dr. Mark McClellan, director of the Duke-Margolis Center for Health Policy. “Through its more accessible, cost-effective, and inclusive approach, the Advancing Clinical Trials at the Point of Care coalition will help the clinical trial enterprise answer priority research questions for COVID-19, prepare for future public health emergencies, and address common diseases where there is long-standing unmet medical need including, cardiovascular and neurological diseases.”
Founding members of the coalition detail their collective commitment to this effort in an open letter. Founding ACT@POC members include: Duke-Margolis Center for Health Policy; MITRE; CURE Drug Repurposing Collaboratory (C-Path + NCATS+ FDA); Emory-Morningside Center for Innovative and Affordable Medicine; Intermountain Healthcare; Mayo Clinic; University of California, Irvine; Duke University Health System; and Broad Institute of MIT and Harvard. Its growing membership will include health systems, community-based care organizations, health research organizations, and other collaborators.
“Coalitions bring together multidisciplinary experts to solve the hardest problems,” said John Halamka, M.D., president, Mayo Clinic Platform. “We have assembled leaders in clinical trials, informatics, and public policy to accelerate cures using real world evidence and advance patient care.”
The consequences of inefficient, low-value COVID-related clinical research made the need for a coalition to support inclusive and efficient clinical trials apparent. One analysis found that, of 2,610 trials of existing drugs repurposed as COVID-19 therapeutics, only five percent had sufficient enrollment and key design features to yield actionable evidence to combat COVID-19. Further, only a tiny fraction of Americans diagnosed with COVID-19 participated in those trials.
“COVID-19 has required health systems and public health experts to rely on data for decision-making to quickly gain insights that could deliver better patient outcomes,” said Dr. Brian Anderson, MITRE, chief digital health physician. “The answers to many research questions can be found in the data and allow for engagement of patients in underserved communities who are often left out of traditional clinical research trials. By working with clinicians who are the trusted care givers of underserved communities, we hope to more fully engage and reach people through this collaboration to help yield new answers to many public health diseases.”
The coalition’s agenda and action steps aim to substantially augment the evidence generation capacity of the current clinical trial enterprise by working with patient groups, community hospitals and health centers, medical practices, research organizations, and biotechnology companies. In its work, the Coalition will target:
- Engaging clinicians in a broader range of care settings
- Developing and adopting effective data collection tools
- Collaborating with clinical trial design leaders, regulators, funders, and other stakeholders to assure that clinical trial design features are fit for purpose
- Enrolling diverse trial participants for broader participation in effective community-level trials
- Addressing unmet medical needs by reaching a critical mass of participation in existing and new clinical trials
- Improving technology supports and capabilities to conduct such studies over time
For more information about ACT@POC, please visit: Coalition for Advancing Clinical Trials (actpoc.org).
Additional Coalition Materials
The Broad Institute
The Broad Institute of MIT and Harvard is a research organization that convenes a community of researchers from across many disciplines and partner institutions—MIT, Harvard, and Harvard-affiliated hospitals.
Based in Cambridge, Massachusetts, the Broad Institute was founded in 2004 to fulfill the promise of genomic medicine—three years after completion of the Human Genome Project, which Broad scientists helped create and lead. Our origins are rooted in genomics, and as biomedical research and knowledge have expanded since 2004, so have we. Our researchers are deeply collaborative, nimbly launching innovative, high-risk projects at every scale, inventing new technologies, building and implementing computational tools, developing new therapeutics to advance into the clinic, mentoring and training the next generation of scientists, and sharing our data and tools openly to enable breakthroughs anywhere.
Clinical Trials Transformation Initiative (CTTI)
At its most fundamental level, the Clinical Trials Transformation Initiative (CTTI) is a group of individuals and organizations that want to improve the quality and efficiency of clinical trials. It was co-founded by Duke University and the U.S. Food and Drug Administration in 2007 in an effort to identify and address challenges to well-designed, properly executed clinical trials, and offer recommendations to improve and modernize research.
Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.
Duke Clinical Research Institute
The Duke Clinical Research Institute, part of the Duke University School of Medicine, is the world’s largest academic clinical research organization. We conduct innovative research to deliver on our mission to share knowledge that improves health around the world. DCRI projects are led by physician scientists whose grounding in patient care helps to inform their research, and supported by staff who have deep expertise in operationalizing global studies. Learn more at dcri.org.
Duke Health is committed to conducting innovative basic and clinical research, rapidly translating breakthrough discoveries to patient care and population health, providing a unique educational experience to future clinical and scientific leaders, improving the health of populations, and actively seeking policy and intervention-based solutions to complex global health challenges. Underlying these ambitions is a belief that Duke Health is a destination for outstanding people and a dedication to continually explore new ways to help our people grow, collaborate and succeed. Learn more at Duke Health.
Duke-Margolis Center for Health Policy
The mission of the Duke-Margolis Center for Health Policy at Duke University is to improve health, health equity, and the value of health care through practical, innovative, and evidence-based policy solutions. For more information, visit healthpolicy.duke.edu and follow us on Twitter @DukeMargolis.
Morningside Center for Innovative and Affordable Medicine
The Morningside Center for Innovative and Affordable Medicine (Morningside Center) is an interdisciplinary unit within the Woodruff Health Sciences Center at Emory University. Extending the vision of GlobalCures Inc., a non-profit medical research organization, the Morningside Center was established at Emory in 2019 through a gift from the Morningside Foundation to promote research, education, and advocacy for effective and affordable medical treatments.
Located in Utah, Idaho, and Nevada, Intermountain Healthcare is a nonprofit system of 25 hospitals, 225 clinics, the Intermountain Medical Group with some 2,700 employed physicians and advanced care practitioners, a health plans division called SelectHealth, Homecare, and other health services. Helping people live the healthiest lives possible, Intermountain is committed to improving community health and is widely recognized as a leader in transforming healthcare by using evidence-based best practices to consistently deliver high-quality outcomes and sustainable costs.
Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news. For information on COVID-19, including Mayo Clinic's Coronavirus Map tracking tool, which has 14-day forecasting on COVID-19 trends, visit the Mayo Clinic COVID-19 Resource Center.
Medable is on a mission to get effective therapies to patients faster by transforming clinical drug development with disruptive technologies. The company’s digital platform streamlines design, recruitment, retention and data quality for decentralized trials, replacing siloed systems with integrated digital tools, data and interfaces to accelerate trial execution. Medable connects patients, sites and clinical trial teams to improve patient access, experience and outcomes. Medable’s software has been named a Leader in the industry by both Everest Group and IDC. Medable is a privately held, venture-backed company headquartered in Palo Alto, California.
MITRE’s mission-driven teams are dedicated to solving problems for a safer world. Through our public-private partnerships and federally funded R&D centers, we work across government and in partnership with industry to tackle challenges to the safety, stability, and well-being of our nation.
Quantum Leap Healthcare Collaborative
Quantum Leap Healthcare Collaborative is a 501C(3) charitable organization established in 2005 as a collaboration between medical researchers at University of California, San Francisco and Silicon Valley entrepreneurs. Our mission is to integrate high-impact research with clinical processes and systems technology, resulting in improved data management and information systems, greater access to clinical trial matching and sponsorship, and greater benefit to providers, patients and researchers. Our goal is to improve and save lives. Quantum Leap provides operational, financial, and regulatory oversight to the I-SPY Trials. For more information, visit www.QuantumLeapHealth.org.
University of Massachusetts Department of Medicine
The Department of Medicine’s vision is to be the national leader in the delivery of innovative and high-quality patient care, education programs and in transformative basic and clinical research.
UCI Health Affairs
Founded in 2017 with a $200 million gift from Orange County philanthropists Susan and Henry Samueli, UCI Health Affairs is a first of its kind health sciences enterprise and is unique to Orange County, Calif. Together, the Susan & Henry Samueli College of Health Sciences and UCI Health form UCI Health Affairs, led by Vice Chancellor Steve Goldstein, MA, MD, PhD, FAAP. Susan & Henry Samueli College of Health Sciences is translating our tripartite mission to “discover, teach, heal,” into the diverse healthcare workforce of the future and groundbreaking scientific discoveries. Creating a first-of-its-kind alliance across health disciplines, the college comprises the School of Medicine, the Sue & Bill Gross School of Nursing, the recently launched School of Pharmacy & Pharmaceutical Sciences, the future School of Population & Public Health (scheduled to be accredited in 2021), the Susan Samueli Integrative Health Institute, and research units focused on cancer, clinical translational science, precision health and stem cells. The college shares a robust synergy with UCI Health, the only academic health system in Orange County. UCI Health Affairs is transforming education, discovery, and patient care to benefit the region, state and nation.
Vanderbilt University Medical Center
Managing more than 2 million patient visits each year, Vanderbilt University Medical Center (VUMC) is one of the largest academic medical centers in the Southeast, and is the primary resource for specialty and primary care in hundreds of adult and pediatric specialties for patients throughout Tennessee and the Mid-South. The School of Medicine’s biomedical research program is among the nation’s top 10 in terms of National Institutes of Health peer review funding, receiving more than $500 million in public and private awards during 2016. The Medical Center is the region’s locus of postgraduate medical education, with over 1,000 residents and fellows training in more than 100 specialty areas. Vanderbilt University Adult Hospital and the Monroe Carell Jr. Children’s Hospital at Vanderbilt are recognized each year by U.S. News & World Report’s Best Hospitals rankings as national leaders, with 19 nationally ranked adult and pediatric specialties. Through the Vanderbilt Health Affiliated Network, VUMC is working with over 60 hospitals and 5,000 clinicians across Tennessee and five neighboring states to share best practices and bring value-driven and cost-effective health care to the Mid-South.
Through the exceptional capabilities and caring spirit of its people, Vanderbilt will lead in improving the healthcare of individuals and communities regionally, nationally and internationally. We will combine our transformative learning programs and compelling discoveries to provide distinctive personalized care.