Monitoring and Evaluation of Patient Engagement in Health Product Research and Development: Co-Creating a Framework for Community Advisory Boards

Authors

Sevgi E. Fruytier, Athena Institute, Vrije Universiteit AmsterdamFollow
Lidewij Eva Vat, Athena Institute, Vrije Universiteit AmsterdamFollow
Rob Camp, European Organisation for Rare Diseases (EURORDIS)Follow
François Houÿez, European Organisation for Rare Diseases (EURORDIS)Follow
Hilde De Keyser, CF EuropeFollow
Denise Dunne, Cystinosis IrelandFollow
Davide Marchi, Vertex Pharmaceuticals (Europe) LimitedFollow
Laura McKeaveney, Novartis AGFollow
Richard H. Pitt, Boehringer Ingelheim International GmbHFollow
Carina A.C.M. Pittens, Athena Institute, Vrije Universiteit AmsterdamFollow
Meagan F. Vaughn, Translate Bio, Inc.Follow
Elena Zhuravleva, F. Hoffmann–La Roche LtdFollow
Tjerk Jan Schuitmaker-Warnaar, Athena Institute, Vrije Universiteit AmsterdamFollow

Publication Date

1-17-2022

Keywords

patient engagement, patient participation, rare diseases, program evaluation, drug development

Abstract

Purpose: While patient engagement is becoming more customary in developing health products, its monitoring and evaluation to understand processes and enhance impact are challenging. This article describes a patient engagement monitoring and evaluation (PEME) framework, co-created and tailored to the context of community advisory boards (CABs) for rare diseases in Europe. It can be used to stimulate learning and evaluate impacts of engagement activities.

Methods: A participatory approach was used in which data collection and analysis were iterative. The process was based on the principles of interactive learning and action and guided by the PEME framework. Data were collected via document analysis, reflection sessions, a questionnaire, and a workshop.

Results: The tailored framework consists of a theory of change model with metrics explaining how CABs can reach their objectives. Of 61 identified metrics, 17 metrics for monitoring the patient engagement process and short-term outcomes were selected, and a “menu” for evaluating long-term impacts was created. Example metrics include “Industry representatives’ understanding of patients’ unmet needs;” “Feeling of trust between stakeholders;” and “Feeling of preparedness.” “Alignment of research programs with patients’ needs” was the highest-ranked metric for long-term impact.

Conclusions: Findings suggest that process and short-term outcome metrics could be standardized across CABs, whereas long-term impact metrics may need to be tailored to the collaboration from a proposed menu. Accordingly, we recommend that others adapt and refine the PEME framework as appropriate. The next steps include implementing and testing the evaluation framework to stimulate learning and share impacts.

Recommended Citation

Fruytier SE, Vat LE, Camp R, Houÿez F, De Keyser H, Dunne D, Marchi D, McKeaveney L, Pitt RH, Pittens CA, Vaughn MF, Zhuravleva E, Schuitmaker-Warnaar TJ. Monitoring and evaluation of patient engagement in health product research and development: co-creating a framework for community advisory boards. J Patient Cent Res Rev. 2022;9:46-57. doi: 10.17294/2330-0698.1859