OBJECTIVES:

Patient engagement (PE) in health technology assessment (HTA) is vital to understanding the unmet needs and impact of a disease and its treatment and informs healthcare resource allocation. With current guidelines for PE in early development, there is significant variability in PE among various HTA bodies. We aimed to identify the challenges perceived by patients and patient organizations for involvement in the HTA process in Europe.

METHODS:

We performed a targeted literature review in PubMed to identify qualitative studies published from 2012 to 2022 assessing the challenges reported by patients and their representatives for active participation in HTA activities in Europe. We also reviewed guidance published by prominent HTA bodies in EU4 and the UK that address these challenges.

RESULTS:

Twelve cross-sectional studies reporting patient-, caregiver-, and patient-organization-perceived challenges in HTA engagement were identified. Commonly reported barriers were overlooking patient inputs (n=8), limited disease-specific patient representation (n=7), time constraints (n=6), inadequate communication (n=5), conflicting vision (n=3), preconceptions about patient inputs (n=2), disease-associated stigma (n=2), and limited PE knowledge (n=1). Further, the lack of information (n=8), resources (n=6), training (n=6), transparency (n=5), and remuneration (n=4) substantially limited PE in HTA. The National Institute for Health and Care Excellence (NICE) and the Institute for Quality and Efficiency in Health Care (IQWiG) ensure that the lived experience of patients regarding the technology being assessed is adequately captured early in the HTA process, whereas PE in HTA is still in the early stages in France, Spain, and Italy.

CONCLUSIONS:

Methodological challenges and lack of concrete guidelines limit meaningful patient contributions to the HTA process. It is important for HTA bodies to work toward a shared vision ensuring timely patient participation at every stage of the HTA process, as PE supports equity, relevance, accountability, and credibility of the decision-making process.