Presentation (CTI)

OBJECTIVES: This study investigates how patient perspectives expressed through committee meeting contributions, written submissions, and public consultation comments from individual patients and patient groups are reflected in the decision-making processes of the National Institute for Health and Care Excellence (NICE) for rare disease technology appraisals. It aims to unpack the nature, frequency, and influence of patient input in shaping final recommendations.
METHODS: A retrospective document analysis was conducted on NICE technology appraisals for rare diseases appraised via the Single Technology Appraisal (STA) route and published between 2022 and 2025. Data sources include committee meeting papers, draft guidance documents, and final guidance. Patient input was thematically coded to identify recurring themes and their influence on appraisal outcomes.
RESULTS: Preliminary findings suggest that patient perspectives are frequently cited in appraisal documents, particularly in relation to treatment burden, unmet need, and acceptability. However, the extent to which these perspectives shape final recommendations varies. Patient perspectives are particularly important for rare diseases that don’t qualify for Highly Specialised Technology (HST) routing, where data are sparse but the technologies are still subject to the same willingness-to-pay threshold as non-rare diseases. Thematic analysis will reveal patterns in how such input is operationalised within NICE’s evolving decision-making framework.
CONCLUSIONS: This study offers an empirical, document-based analysis of patient involvement and how their input is incorporated into decision making in rare disease HTA. One of the challenges of individual patient input is that it may not represent the overall patient population. Findings will inform recommendations for enhancing transparency, consistency, prioritisation of patient input, and meaningful patient engagement in HTA decision-making, particularly for rare conditions.