Clinical trials represent a critical pathway to innovative treatments, life-changing cures, and medical advances. But for all their scientific rigor, most studies miss one crucial element: input from the very people they are trying to help.
In the past, patients had little say in the design of clinical trials. Responsibility for study design typically lies with academics, clinicians, statisticians, and industry experts. However, a recent study by the Health Stories Project found that 97% of patients believe it is important for biopharmaceutical companies to incorporate patient feedback into the design of clinical trials. In addition, 83% of patients expressed interest in providing feedback. Unfortunately, most studies do not take the patient voice into account, resulting in 85% of clinical trials not retaining enough patients to complete. This lack of patient involvement has resulted in many studies providing minimal real-world benefit at best and harm at worst.
Fortunately, the landscape continues to evolve. Over the past decade, there has been increasing patient advocacy coupled with regulatory guidance that drives home the message: patients need and deserve a seat at the table.
Poorly designed trials often fail when patients review them or are under real-world pressure. For example, a treatment that requires daily doctor visits could be successful under ideal experimental conditions. However, when the same restrictions are imposed on patients who must balance work, family, and comorbidities, treatment adherence often drops to unsustainable levels.
Excluding patients from clinical trials can have a significant impact on engagement. Patients and doctors have different perspectives when it comes to treatment success and outcomes. Patients who live with their conditions on a daily basis are better able to determine which outcomes are most important compared to traditional experts. For example, while reducing mortality may be statistically significant for researchers, clinical treatments that have such effects can leave patients bedridden and unable to work or care for their families. By leveraging these experiences and examining the benefit-risk trade-offs, we can gain valuable insights for designing clinical trials that effectively meet patients’ needs.
Involve patients from the start
To optimize the validity and success of studies, researchers must collaborate from the beginning of study planning through authentic conversations with patients and caregivers who are themselves experts.
One way to ensure that a clinical trial is relevant to patients’ everyday lives is to conduct a comprehensive survey process. This can be achieved by organizing meetings between researchers and patients, where researchers can gain a deep understanding of patients’ individual stories and experiences with the condition under study. Additionally, researchers may benefit from self-completion exercises in which patients create their own timelines for their illness and explore techniques for expressing both the conscious and unconscious manifestations of their illness.
Patient involvement in clinical trials should not be limited to just taking part in the trial. Patients should also be included in the definition of study endpoints to capture their experiences during the study. Factors such as the impact of treatment on their functional limitations, emotional well-being, and social functioning may provide additional insight into the effectiveness of treatment. To ensure that clinical trials are accessible to a broader population and do not create barriers, clinicians can consider using decentralized technologies such as wearables and smartphone apps to allow patients to easily report data from any location.
looking ahead
In the future, clinical trial providers will make greater efforts to facilitate the connection of patients and healthcare providers to relevant clinical trials. It is important for healthcare professionals and patients to ensure continued access to various healthcare systems that share anonymized data to advance research and connect patients to appropriate studies. This will enable the acceleration of breakthrough discoveries and therapies by involving patients in the development of clinical trials.
Patients are looking for cures just as industry and science are looking for breakthroughs. Breakthrough advances can be achieved by integrating patient collaboration and priorities across the full spectrum of trial design. To lead clinical trials as partners, patients must be treated as equals to research teams and not as passive subjects. It is important for the industry to recognize that patients are both beneficiaries and creators of scientific knowledge.
Photo: 9amstock, Getty Images
Christopher P. Boone, Ph.D., is a global manager, strategist, professor and social scientist with a unique passion for revolutionizing healthcare and life sciences through clinical research informatics, real-world evidence and health equity. As Group Vice President of Life Sciences Research Services at Oracle Corporation, Chris leads the function responsible for conducting research that accelerates the discovery, development and implementation of extraordinary discoveries and therapies to transform healthcare and patients’ lives to improve. His academic contributions as an adjunct assistant professor at New York University’s Robert F. Wagner Graduate School of Public Service further enhance his influence.