The NHC recently received a PhRMA Foundation Research Award in Value Assessment (VA) for our project, Methods Principles for Using Patient-Provided Information to Improve Real-World Evidence for Patient-Centered Value Assessment.
The objective of this project is to develop research-methods, good-practice principles on how and at which study-design stage, patient-provided information (e.g., experiences, preferences, perspectives, desired outcomes, etc.) should be used by researchers. Specifically, we seek to identify how to use patient-provided information to inform methods decisions (e.g., meaningful outcomes) to improve the rigor of real-world evidence (RWE) for use in patient-centered value frameworks and assessments.
The new principles, to be established through multi-stakeholder collaboration, will help researchers transparently apply patient-provided information to their research designs. This will improve the rigor of RWE because design decisions will be based on patients’ lived experiences instead of researcher or clinician assumptions. Patient-informed RWE will contribute to a patient-centered evidence base to further inform patient-centered VA.
We expect our principles, which will be finalized in summer 2019, to be useful for researchers as they seek to adhere to the ISPOR-ISPE Special Task Force on RWE in Health Care Decision Making and other recommendations for stakeholder engagement.
The project originated from patient input on the topics of both value assessment and RWE. For the project, we will leverage the vast knowledge of the NHC membership, especially the NHC Value Work Group. In 2016, the NHC formed the Work Group, which currently includes 27 staff members from 18 patient advocacy organizations that have had or expect to have interactions with value framework developers regarding treatment-specific value assessments.
In member discussion on RWE, patients recommended that researchers should co-develop RWE with patients. In the patients’ view, information they provide directly is more “real world” than data from claims and electronic health records, two primary sources of real-world data. They expressed those data are only “real” to an extent and may not reflect the diversity of disease experiences, preferences, or outcomes. Participants recommended RWE should aggregate these data with additional real-world sources, including patient-provided and patient-generated data (e.g., patient-organization registries).
The NHC team working on this project includes: Dr. Eleanor M. Perfetto, Senior Vice President, Strategic Initiatives and Dr. Elisabeth M. Oehrlein, Senior Director, Research and Programs. For this project, the NHC is teaming with experts from the University of Maryland, Baltimore.
For more information about this project, contact NHC Senior Director of Research and Programs Dr. Elisabeth Oehrlein.