How are patient inputs considered in HTA? A thematic document analysis of NICE ultra-rare disease appraisals
File(s)
Published version
Author(s)
Gentilini, Arianna
Rana, Alina
Type
Journal Article
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.
Date Issued
2024-08-01
Date Acceptance
2024-11-28
Citation
European Journal of Health Economics, 2024, 26 (6), pp.945-968
ISSN
1618-7598
Publisher
Springer
Start Page
945
End Page
968
Journal / Book Title
European Journal of Health Economics
Volume
26
Issue
6
Copyright Statement
© The Author(s) 2024 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
License URL
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/39725821
PII: 10.1007/s10198-024-01748-1
Subjects
Business & Economics
Economics
GROUP SUBMISSIONS
Health Care Sciences & Services
Health Policy & Services
HEALTH TECHNOLOGY-ASSESSMENT
HTA
INVOLVEMENT
Life Sciences & Biomedicine
NICE
ORPHAN
PARTICIPATION
Patient organisations
Rare diseases
Science & Technology
Social Sciences
Publication Status
Published
Coverage Spatial
Germany
Date Publish Online
2024-12-27