PATIENT INVOLVEMENT IN RARE DISEASE HEALTH TECHNOLOGY ASSESSMENT (HTA): REAL-WORLD PRACTICES IN AUSTRALIA, SINGAPORE, AND TAIWAN
Author(s)
Rui Min Fung, BSc, Carol Kao, BSc.
Avalere Health, Singapore, Singapore.
Avalere Health, Singapore, Singapore.
OBJECTIVES: To assess the extent to which patient involvement in health technology assessment (HTA) reflects formal guidance in Australia, Singapore, and Taiwan, drawing on rare disease HTA reports published in 2025.
METHODS: We conducted a cross-jurisdictional analysis of all rare disease HTA reports published by Australia’s, Singapore’s, and Taiwan’s HTA bodies in 2025. Across reports, we extracted structured data on: (1) whether patients were involved; (2) mechanisms of involvement; (3) types of input collected; and (4) impact on outcomes. Findings were evaluated against jurisdictions’ patient involvement guidance, with within- and cross-jurisdictional trends identified.
RESULTS: We identified 62, 15 and 26 rare disease HTA reports from Australia, Singapore, and Taiwan, respectively. Documented patient involvement was near-universal in Australia (57/62, 92%), but substantially lower in Singapore and Taiwan. Singapore documented patient testimonials in 6/15 reports (40%); two further reports noted that no responses were received despite invitations. Only 5/26 Taiwanese reports (19%) included direct local patient input; 9/26 (35%) reports referenced patient evidence from foreign HTA agencies. Australia and Taiwan leveraged publicly accessible online platforms to collect input, whereas Singapore sourced testimonials through local patient and voluntary organizations. Across jurisdictions, patient evidence was predominantly descriptive, with common domains being treatment experience (90%), quality of life/ daily functioning (81%), and disease/ symptom burden (72%). Evidence of patient input influencing HTA outputs was limited.
CONCLUSIONS: Despite formal guidance and established mechanisms, substantial patient involvement gaps remain in Singapore’s and Taiwan’s rare disease HTAs. Australia has normalized open, public-facing submission processes, supported by a well-developed patient advocacy infrastructure that includes umbrella and disease-specific patient groups. In contrast, Singapore and Taiwan rely on narrower channels such as invitation-only processes or foreign patient evidence, limiting volume and applicability. However, findings may underestimate total patient involvement, as engagement occurring outside published reports (e.g., topic nomination) were not captured.
METHODS: We conducted a cross-jurisdictional analysis of all rare disease HTA reports published by Australia’s, Singapore’s, and Taiwan’s HTA bodies in 2025. Across reports, we extracted structured data on: (1) whether patients were involved; (2) mechanisms of involvement; (3) types of input collected; and (4) impact on outcomes. Findings were evaluated against jurisdictions’ patient involvement guidance, with within- and cross-jurisdictional trends identified.
RESULTS: We identified 62, 15 and 26 rare disease HTA reports from Australia, Singapore, and Taiwan, respectively. Documented patient involvement was near-universal in Australia (57/62, 92%), but substantially lower in Singapore and Taiwan. Singapore documented patient testimonials in 6/15 reports (40%); two further reports noted that no responses were received despite invitations. Only 5/26 Taiwanese reports (19%) included direct local patient input; 9/26 (35%) reports referenced patient evidence from foreign HTA agencies. Australia and Taiwan leveraged publicly accessible online platforms to collect input, whereas Singapore sourced testimonials through local patient and voluntary organizations. Across jurisdictions, patient evidence was predominantly descriptive, with common domains being treatment experience (90%), quality of life/ daily functioning (81%), and disease/ symptom burden (72%). Evidence of patient input influencing HTA outputs was limited.
CONCLUSIONS: Despite formal guidance and established mechanisms, substantial patient involvement gaps remain in Singapore’s and Taiwan’s rare disease HTAs. Australia has normalized open, public-facing submission processes, supported by a well-developed patient advocacy infrastructure that includes umbrella and disease-specific patient groups. In contrast, Singapore and Taiwan rely on narrower channels such as invitation-only processes or foreign patient evidence, limiting volume and applicability. However, findings may underestimate total patient involvement, as engagement occurring outside published reports (e.g., topic nomination) were not captured.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
PCR19
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement
Disease
SDC: Rare & Orphan Diseases