Abstract
Patient organisations are increasingly involved in HTA. Given this, it is important to understand what these organisations contribute and how their voices are accounted for in the decision-making process. This study characterises inputs from patient organisations and/or their nominated patient experts in technology appraisals for ultra-rare diseases in England and Wales and seeks to understand how these are considered in NICE final recommendations. We thematically analysed all HST appraisals completed between January 2022 and August 2024 (N = 15). We appraised inputs from patient organisations' and experts' written submissions, the novelty of patient inputs, as well as financial ties between contributing organisations and the manufacturer of the technology being appraised. We compared themes identified with those found in the Final Evaluation Determination documents to understand how and to what extent patients' inputs were considered in NICE final recommendations. We found that patient submissions mainly focused on disease aspects (54%). Patients raised concerns on access challenges, caregiver burden, and mental health impacts. Most patient themes overlapped with manufacturers' submissions (82%) and doctors' testimonies (45%), with most novel insights focusing on access issues and mental health. Patient organisations reported receiving funding from the technology manufacturer in most appraisals, with amounts ranging from £5,000 to £74,113. Approximately half of patient inputs were explicitly mentioned in NICE final decision documents, with some considerations being neglected despite being raised by patients. While NICE incorporates many issues of importance to patients, there is room for improvement to ensure all aspects patients deem important are captured. Further research could pinpoint optimal areas for patient contributions and assess their impact.