By Elisabeth M. Oehrlein, PhD, MS, Assistant Vice President, Research and Programs 

Why is this important? 

Interest in real-world data (RWD) and real-world evidence (RWE) among health care stakeholders, including patients, regulators, and value assessors has grown over the past two decades. In parallel, interest in patient engagement and other stakeholder engagement in research has grown substantially. A comprehensive understanding of patients’ lived experiences can assist researchers in ensuring that studies reflect as closely as possible “real-world” patient experiences and health care as it is delivered. However, there are currently no recommended methods or standards to leverage patient input that can be applied when designing RWD studies. Without a set of guiding principles to support and encourage patient engagement in RWE, the status quo is unlikely to change. 

What did we do? 

Guided by a multi-disciplinary Advisory Board and insights from numerous experts, the National Health Council (NHC) developed a set of recommendations that provide guidance for how patient input, gathered through meaningful patient engagement, can be identified and incorporated into the design, conduct, and translation of real-world research that reflects patients’ lived experiences. 

What did we learn? 

High-quality RWD studies and RWE evaluations are not conducted by RWD researchers in silos. Instead, they are a product of meaningful engagement of relevant key stakeholders that contribute various sources of knowledge, insights, and expertise throughout the design, execution, and reporting of studies.  

The output includes 13 consensus recommendations, organized into three themes: 

• Developing a Patient-Centered Research Question and Study Design  
• Disseminating Research Findings 
• General Considerations 

Click here to read the full report and recommendations.

You can also learn more about this work at our upcoming webinar on July 30 from 10 -11 a.m. ET. Sign up for our webinar mailing list at NHCprograms@nhcouncil.org.

Acknowledgements: This work was supported by a grant from the PhRMA Foundation. Special thanks to our Advisory Board: 

• Wendy Smith Begolka, MBS, Vice President, Scientific and Clinical Affairs, National Eczema Association 
• Jennifer Bright, MPA, Executive Director, Innovation and Value Initiative 
• Mehmet Burcu, PhD, MS, Principal Scientist & Director, Center for Observational & Real-World Evidence (CORE), Merck & Co. 
• Angeline M. Carlson, BS, RPh, PhD, Principal, DataIQ 
• Robert W. Carlson, MD, Chief Executive Officer, National Comprehensive Cancer Network® 
• Susan dosReis, BSPharm, PhD, Professor and Vice Chair for Research, Pharmaceutical Health Services Research Dept., University of Maryland School of Pharmacy 
• Julia McBeth, Director of Research and Advocacy, Celiac Disease Foundation 
• Debbe McCall, MBA, Patient PI on Health eHeart Alliance Patient-powered Research Network 
• C. Daniel Mullins, PhD, Professor and Chair, Pharmaceutical Health Services Research Dept., University of Maryland School of Pharmacy 
• Chris L. Pashos, PhD, Independent Consultant and Expert Advisor 
• Prasun Subedi, PhD, Senior Director, Center for Health Systems Innovation and Leadership, Pfizer 
• Patrick Wildman, Vice President, Advocacy & Government Relations, Lupus Foundation 
• Richard Willke, PhD, Chief Science Officer, International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 
• Marcus D. Wilson, PharmD, Chief Analytics Officer, Anthem, Inc 

Project Team 

• Elisabeth Oehrlein, PhD, MS, Assistant Vice President, Research & Programs, NHC 
• Eleanor M. Perfetto, PhD, MS, Executive Vice President, Strategic Initiatives, NHC 
• Silke Schoch, Manager, Research & Programs, NHC 
• Laura Elisabeth Gressler, PhD, MS, Graduate Research Assistant, UMB 
• Joey Mattingly, PharmD, MBA, PhD, Associate Professor, UMB 
• T. Rosie Love, MPH, PhD Candidate, UMB