Presentation (CTI)

OBJECTIVES: Patient engagement in HEOR and HTA enhances relevance and utility for decision-makers. We previously identified published frameworks or guidance to capture the patient voice. Our review highlighted limited assessment of their quality in an HEOR or HTA context. Here, we critically appraise the frameworks and guidance identified for patient engagement in HEOR or HTA.
METHODS: Frameworks or guidance for patient engagement in priority setting (n=3), evidence synthesis (n=2), and HTA (n=5) were appraised using relevant domains of the Appraisal of Guidelines for REsearch & Evaluation (AGREE) II. Relevant signalling questions from 5/6 applicable domains in AGREE II were used: scope and purpose, stakeholder involvement, rigor of development, clarity of presentation and applicability.
RESULTS: Priority‑setting frameworks or guidance, such as the James Lind Alliance Handbook, Pratt’s engagement model and Pollock’s engagement model score highly for scope and process and stakeholder involvement however are heterogenous in clarity of presentation. Evidence‑synthesis frameworks like RIPPLES and ACTIVE scored highly across domains with the exception of applicability. HTA frameworks from Abelson, EUPATI, EUnetHTA, CDA and NICE provide comprehensive guidance with clear scope and purpose however, development of the guidance was not consistently described. The quality of guidance for implementation and accessibility to patients, researchers and sponsors varied greatly across the guidance and frameworks assessed. Overall, limited detail was given on resource implications of proposed approaches.
CONCLUSIONS: While frameworks outlined valuable approaches, methodological rigor, accessibility and implementation guidance varies. Future frameworks and guidance should transparently report development processes and integrate protocols for the evaluation of their use and impact on HEOR or HTA. This will enable informed selection for use in patient-centric research and continual improvement.