Evaluating the "return on patient engagement initiatives" in medicines research and development: A literature review.

Lidewij Eva Vat,Robert A Kroes,Nick Fahy,Claudia Hey,Ana Diaz,Mathieu Boudes,Teresa Finlay,Gabor Purman,Elisa Ferrer,Jacqueline E.W Broerse,Tjerk Jan Schuitmaker‐Warnaar,Virginie Hivert,Marie‐Laure Kürzinger,Paul Robinson

doi:10.1111/hex.12951

Abstract

BackgroundShowing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. However, it is unclear what methods can best be used to evaluate patient engagement. This paper is focused on ways to evaluate patient engagement at three decision‐making points in the medicines research and development process: research priority setting, clinical trial design and early dialogues with regulators and health technology assessment bodies.ObjectiveOur aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods.Search strategy and inclusion criteriaWe undertook a scoping literature review using a systematic search, including academic and grey literature with a focus on evaluation approaches or outcomes associated with patient engagement. No date limits were applied other than a cut‐off of publications after July 2018.Data extraction and synthesisData were extracted from 91 publications, coded and thematically analysed.Main resultsA total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement.Discussion and conclusionsCurrently available indicators and methods are of some use in measuring impact but are not sufficient to understand the pathway to impact, nor whether interaction between researchers and patients leads to change. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures.

Highlights

This review identified a range of benefits, costs and challenges that patient engagement can have on research and development (R&D) and describes several indicators associated with their monitoring and evaluation
For patient engagement in the development of medicines to become standard practice at the key decision‐making points of priority setting, clinical trial design and regulatory and Health Technology Assessment (HTA) processes, benefits need to be demonstrable to all stakeholders
The approaches to evaluation we found are largely qualitative, and our review suggests that there are few quantitative tools and no standardized approaches to assessing the outcomes and impact

Summary

Introduction

There is increasing consensus among stakeholders that patient engagement in research and development (R&D) of medicines provides benefits for patients, researchers, industry, regulatory bodies, payers and policy makers.[1,2,3] The case for patient engagement is often presented in ethical and political terms referring to fairness, transparency and accountability.[4,5] Methodological arguments consider the experiential knowledge of patients—acquired by their personal experience of a condition—as valuable to improving the quality and relevance of the research.[6,7,8] The inclusion of patients in decision making about the development of new innovative medicines is a substantial change, requiring time and (financial) commitments from researchers, industry and patients.[2,4] Despite efforts to promote and support patient engagement in research, the prevalence of patient engagement in medicines research and development remains low.[9,10] Patient engagement has not been fully embedded in the health research system, partly because it is not yet clear to all involved what the added value is.[11]. Showing how engagement adds value for all stakeholders can be an effective motivator for broader implementation of patient engagement. It is unclear what methods can best be used to evaluate patient engagement. Objective: Our aim was to review the literature on monitoring and evaluation of patient engagement, with a focus on indicators and methods. Main results: A total of 18 benefits and 5 costs of patient engagement were identified, mapped with 28 possible indicators for their evaluation. Several quantitative and qualitative methods were found for the evaluation of benefits and costs of patient engagement. We suggest that the impacts of patient engagement can best be determined not by applying single indicators, but a coherent set of measures

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