Patient Preference Studies in HTA for Rare Diseases: A Cross-Country Analysis of EU-4, UK, and Canada
Author(s)
Puneet Kumar, M.Pharm1, Mohit Joshi, M.Pharm2, Vaibhav Sheth, PhD2, Samridhi Johri, M.Pharm2.
1Syneos Health, London, United Kingdom, 2Syneos Health, Gurugram, India.
1Syneos Health, London, United Kingdom, 2Syneos Health, Gurugram, India.
OBJECTIVES: Patient preference studies (PPS) are being recognized as valuable tool by Health Technology Assessment (HTA) bodies to evaluate patient-valued attributes and associated trade-offs particularly among rare diseases where clinical evidence is limited. However, integration of PPS in technology appraisals remains unclear across the globe. This review aimed to explore the adoption of PPS in HTAs in rare diseases across EU-4, UK and Canada.
METHODS: Technology appraisals for rare diseases published in the last 5 years (2020 to present) by NICE, SMC, IQWiG, HAS, AIFA, AEMPS, and CDA were reviewed to assess the use of PPS and the extent to which they support/influence appraisal recommendations. Additionally, committee papers were examined to understand each agency’s stance on the use of PPS in the evaluation of technologies for rare diseases.
RESULTS: Among the reviewed HTAs, only NICE demonstrated explicit use of patient preference methods such as discrete choice experiment (DCE), time-trade-off and standard gamble in technology appraisals of rare diseases, including lipodystrophy, AADC deficiency, AHUS, NDM and PNH. These methods were primarily utilized for deriving utility values or for systematic assessment of patient attributes and trade-offs. In contrast, SMC and CDA acknowledged patient preferences through qualitative methods. SMC employed the patient and clinician engagement (PACE) program, which gathers structured patient insights to inform technology appraisals in rare diseases, while CDA considered patient interviews/surveys to support HTA evaluations. No evidence of PPS use or other qualitative methods was observed in France, Germany, Italy and Spain in technology appraisals for rare diseases.
CONCLUSIONS: The findings of this analysis suggests that the use of PPS in rare disease technology appraisals remains limited and inconsistent across the EU-4, UK, and Canada. To support greater adoption of PPS in rare diseases where patient trade-offs are complex, enhanced collaboration and shared learning among HTA bodies in these regions is warranted.
METHODS: Technology appraisals for rare diseases published in the last 5 years (2020 to present) by NICE, SMC, IQWiG, HAS, AIFA, AEMPS, and CDA were reviewed to assess the use of PPS and the extent to which they support/influence appraisal recommendations. Additionally, committee papers were examined to understand each agency’s stance on the use of PPS in the evaluation of technologies for rare diseases.
RESULTS: Among the reviewed HTAs, only NICE demonstrated explicit use of patient preference methods such as discrete choice experiment (DCE), time-trade-off and standard gamble in technology appraisals of rare diseases, including lipodystrophy, AADC deficiency, AHUS, NDM and PNH. These methods were primarily utilized for deriving utility values or for systematic assessment of patient attributes and trade-offs. In contrast, SMC and CDA acknowledged patient preferences through qualitative methods. SMC employed the patient and clinician engagement (PACE) program, which gathers structured patient insights to inform technology appraisals in rare diseases, while CDA considered patient interviews/surveys to support HTA evaluations. No evidence of PPS use or other qualitative methods was observed in France, Germany, Italy and Spain in technology appraisals for rare diseases.
CONCLUSIONS: The findings of this analysis suggests that the use of PPS in rare disease technology appraisals remains limited and inconsistent across the EU-4, UK, and Canada. To support greater adoption of PPS in rare diseases where patient trade-offs are complex, enhanced collaboration and shared learning among HTA bodies in these regions is warranted.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA266
Topic
Health Technology Assessment, Patient-Centered Research, Study Approaches
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
Rare & Orphan Diseases