Determining the best treatment for an individual is a fundamentally different problem from determining the best treatment, on average, in a population. This is not just because individuals have their own values and preferences, but also because they vary from one another in important characteristics that influence the risks and benefits of treatment. Drawing direct inferences for individuals based on average effects measured in groups of patients who differ from one another in important characteristics represents a fundamental flaw of logic.
The broad theme of this project is: “Evidence and the Individual Patient: Understanding Heterogeneous Treatment Effects for Patient-Centered Care.” A new methodological model is necessary to develop more patient-centered evidence, and focused and sustained attention to the issues of heterogeneous treatment effect (HTE) is required. This project will address fundamental conceptual/methodological issues, matters of practical implementation, and knowledge transfer of HTE to the broader patient community by: hosting an online webinar series for patient communities to address the concept of HTE as it pertains to the individual patient and explore its impact through facilitated discussions; and hosting a one-and-a-half-day conference for researchers, clinicians, and other stakeholders to improve the understanding of the inherent limitations of using group data to guide treatment decisions for individuals, explore concepts of HTE as they pertain to personalized decision making and precision medicine, elucidate the needs for modifying care in light of more personalized evidence, and identify the highest policy and research priorities to facilitate translation.
The project team anticipates that patient webinar participants will gain a more thorough understanding of the major problems with the analysis of HTE and how it relates to their individual care, and conference participants will gain a more thorough understanding of the major problems with the analysis of HTE and some proposed solutions. Opportunities for further research collaborations will be identified. Stakeholders will help identify various means to translate findings into the real world. Manuscripts will be generated based on the key findings from the patient-centered webinar series and methodologic conference.
Project stakeholders include: Bray Patrick-Lake, MFS, Co-chair at NIH Advisory Committee to the Director Working Group on the Precision Medicine Initiative, Director of Stakeholder Engagement at Clinical Trials Transformation; Sandra Ackerman, Freelance Writer/Contributing Editor at American Scientist; Robert M Golub, MD, Deputy Editor at JAMA; Walter Kernan, MD, General Internist, Clinical Trialist, Vice Chair for the AHA’s Stroke Scientific Statements Oversight Committee; Ben Nowell, PhD, Co-Principal Investigator for AR-PoWER PPRN; William Crown, PhD, Chief Scientific Officer, Optum Labs; John Spertus, MD, MPH, Chief Medical Officer, Director of Health Outcomes Sciences LLC; Thomas W. Concannon, PhD, Policy Researcher, the RAND Corporation. Stakeholders will take on various roles, including: webinar/conference planning, patient participant recruitment, translation, and dissemination. Patient participants will be engaged during the webinar series, and thereafter, several will be selected to fully participate in the methods conference.