Transforming Clinical Trials: A New Vision For 2030
Editor’s Note
This post inaugurates the Health Affairs Blog short series, “Envisioning A Transformed Clinical Trials Enterprise For 2030.” The series explores ideas for advancing a clinical trials enterprise that is more efficient, effective, person-centered, inclusive, and integrated into the health delivery system of 2030 than is currently the case. The series is being published in conjunction with a four-part public workshop, also titled Envisioning a Transformed Clinical Trials Enterprise in 2030 and convened under the auspices of the National Academies Forum on Drug Discovery, Development, and Translation. Posts in this series are published by Health Affairs Blog with the support of the National Academies, but they represent the opinions of the authors and do not necessarily represent the views of the Forum on Drug Discovery, Development, and Translation, the National Academies, or any other organization; these posts do not constitute reports or products of the National Academies.
The year is 2030. My wearable device detects a measurement that could signal a health problem. It immediately notifies me and my primary care physician. All possible associated health problems are searched, assessed according to my history, and ranked probabilistically using artificial intelligence. A timely diagnosis determines my medical condition.
My primary care physician and I explore possible treatment options and together decide that a clinical trial may be best suited for me. A top medical specialist and health system are identified to meet my health requirements and preferences (e.g., type of treatment, location of study site). I obtain a second opinion from another specialist regarding my diagnosis and clinical options.
My medical team, caregiver, and I agree on a treatment plan. I understand my course of action. A nurse practitioner coordinates and integrates my medical care. Any medications I take outside of my study visits are automatically supplied directly to me (as appropriate). When I take the trial medicine it is automatically tracked with smart technology, e.g., automated uploading of weight from a digital scale or data from an insulin pen.
The study site takes a multidisciplinary approach to integrating my medical treatment considering, e.g., my other health conditions, nutrition, and psychological well-being. My entire medical team meets to discuss my case and develops a tailored, integrated treatment plan. My caregiver is also supported and integrated as a key player in my treatment.
Automated mechanisms are in place to capture my patient-reported data with detail and insight while mitigating the effort to report them. Regulators will use these patient-reported metrics as a key part of their deliberation and approval process. Researchers may also use this trove of data to identify important questions not addressed by existing trials.
A patient navigator—a health professional who works with me to coordinate my care—helps me manage financial resources; facilitates communication between myself, my family, and medical providers; and addresses my medical questions and psychosocial needs. My caregiver and I are connected to an experienced patient who has walked the journey for my disease. I receive frequent updates; the ‘burden’ on me and my caregiver is minimized as much as possible. At the end of treatment, my post-treatment plan is well documented, and understood by me, my caregiver, and my medical team. Follow up monitoring and ongoing communication is appropriate and based on my personal preferences.
Making This Future Attainable For All Patients
Timely integration of clinical trial information along with medical information and psychosocial considerations does not occur seamlessly. The data are dispersed and often the information is communicated to the patient by many experts. But the vision described above is achievable by better integrating systems and through capabilities already within reach. For patients who are well insured, have access to major health care facilities, and see health care providers who are aware of or participate in clinical trials, this is a plausible vision.
But how do we ensure this vision is inclusive of all patients? We must consider the patients who have limited access to health information, who are not well insured, who do not have health care providers or whose physicians do not have familiarity with or access to clinical trials or cutting-edge therapies. It is important that information, choices, and care are available for all patients, understandable to them, and accessible with or without internet connections and smart phones. Health care research and the access it offers to newer therapies needs to move further into local communities, partnering with trusted centers of communication. This cannot be a one-way broadcast of information. It needs to be a true conversation among patients, caregivers, community leaders, health care providers, and researchers. There are some models in place (e.g., the Center for Sustainable Health Care Quality and Equity—promoting sustainable healthy communities in every zip code), but they are not yet standard practice.
Advancing The Science Of Patient Input
Patients today face many challenges when it comes to finding the right diagnoses, treatments, and/or clinical trials. Patient input has aided tremendously in understanding and overcoming these challenges. For example, Fox Insight, an online research study for Parkinson’s diseases supported by the Michael J. Fox Foundation for Parkinson’s Research, enables study participants to contribute information about their family and medical history, current medical conditions, quality of life, and activities of daily living. This information helps researchers to build a natural history of each patient’s disease.
Better aligning priorities for patient involvement in medical product research and development with regulatory decision-making regarding early disease detection, disease management, and treatment would create a more direct translation of research to practice. Further, converting traditionally anecdotal patient input into rigorous, credible evidence for use by a broad range of stakeholders could improve patients’ experiences and outcomes in clinical research and care.
A 2018 workshop hosted by the Forum on Drug Discovery, Development, and Translation at the National Academies of Science, Engineering, and Medicine (the Forum) examined knowledge gaps and other barriers that hinder advancement of the science of patient input into medical product research and development. The list of barriers was further reviewed and prioritized by participants in the Advancing the Science of Patient Input Action Collaborative; this was an ad hoc activity associated with the Forum to identify and prioritize specific areas of research that—if effectively addressed—could lead to more evidence-based and effective patient input.
To promote scientific rigor, patients need to be involved from early research planning throughout the development of medicines, in clinical aspects and also nonclinical aspects such as the delivery of medicines through appropriate devices. More person-centered research is emerging through patients’ advice on protocols, informed consent forms, educational materials, and study logistics. This makes clinical trials more feasible and accessible for patients through improved information sharing that is meaningful and understandable (e.g., through more comprehensible informed consent forms); it lowers the hurdles patients must overcome (e.g., through reduced travel burdens to study sites via fewer procedures arranged to better fit patients’ schedules). Accessibility can be further enhanced by ongoing engagement with patients and sharing of insights among stakeholders throughout the drug research and development lifecycle.
A Path Forward
Ultimately the patient wants timely, transparent, understandable information to make decisions about participating in a study. To build a more relevant clinical trials enterprise, outside-the-box thinking needs to incorporate meaningful patient input throughout the research lifecycle.
For Patients Who Participated In Clinical Research, Provide Opportunities For Engagement, Ongoing Connection, And Long-Term Follow-Up
Involving patients long-term provides two advantages. First, regardless of that patient’s outcome, they are not left feeling that the research community is “done with me.” As a patient explained in an internal team meeting on clinical trial design, “I still have my life. Keep me informed. Don’t just drop me. Keep a psychological connection.” Second, a growing pool of knowledgeable contributors can be asked, under appropriate agreements, for their ideas and input into improving clinical research for both patients and providers.
Create And Support More Opportunities For Involvement In Clinical Research For The Network Of Researchers And Health Care Providers
Include more material about clinical research in the education of health care providers. Provide opportunities for patients to teach and learn. Create opportunities for practicing health care providers to participate in clinical research, e.g., as sub-investigators, through observational or pragmatic studies, or in other ways that allow broader reach into diverse and underserved communities. “Diversity” here can refer to race and ethnicity, economic factors, or rural versus urban; the idea is to address any combination of systemic inequalities fueled by lack of funding, infrastructure, and other resources that continues to widen gaps between patients’ health care access and outcomes.
Finally, support these opportunities by assessing and equitably implementing solutions that decrease the burden on health care providers, who often must balance research commitments against their existing clinical care. Use lessons from the recent COVID-19 pandemic to “decentralize” clinical trials. Allow local sites to collect study samples from patients and send them to a central screening or laboratory site for analysis, reducing variability in study test results while increasing participation of health care providers and patients in more distributed facilities. Ship clinical trial treatments to local sites or directly to patients and include their local health care providers in monitoring clinical trial patients’ health.
For Patients And Health Care Providers, Create An Open Dialogue To Address Questions And Concerns About The Differences Between Research And Clinical Care
Clinical research may involve randomization of patients by treatment groups to test new approaches that may or may not be better than the standard of care. Clinical care aims to provide each patient with the best available treatment for that individual. Establish a sense of trust and open dialogue with patients and providers to address their underlying questions and concerns. Involve them as partners in developing research agendas. Ensure that patients and providers have a clear understanding of what happens during a trial, the type of health care patients will receive, any costs related to enrolling in a trial, the benefits and risks associated with participation, and any potential long-term cost implications of trial participation and treatment, including coverage by payers. Finally, as part of maintaining an ongoing connection, share access to the collected data with the patient and inform them of findings from the trial.
The challenges before us are not new; we have discussed them for decades. Now it is surely time to act, to learn from the extraordinary circumstances and opportunities created by the COVID-19 pandemic, including the lessons of our connections as humans around the globe. In the following post in this series, Silas Buchanan (link live on Thursday, May 6) addresses the challenges of reaching out to underserved communities such as Black Americans who have particular reason to distrust the clinical trial enterprise.
Creating a person-centered health care system necessitates a more equitable health care system. Our human community should now require better cooperation among stakeholders, more concerted focus on health priorities at a global level, and more flexibility in meeting local health needs.
Authors’ Note
Marilyn Metcalf, PhD, is a Senior Director of Patient Engagement at GlaxoSmithKline, which manufactures prescription drugs that undergo clinical trials as part of the Food and Drug Administration’s approval process. Rob Weker is the Principal at Weker Advisors LLC, providing consulting services across the health care industry; he serves on the Hospital of the University of Pennsylvania Patient and Family Advisory Council and on GlaxoSmithKline’s Oncology Patient Council. The authors thank the staff of the National Academies Forum on Drug Discovery, Development, and Translation, including C. Shore, A. Wagner Gee, and J. Liao for contributing to the development of this blog piece. Additional thanks go to Alexandra McGregor, PhD, GlaxoSmithKline.