The HTA Landscape of Orphan Products in the UK Between 2019 and 2024
Author(s)
Penelope Cervelo Bouzo1, Tina Wang, PhD2, Belen sola, MRes, PhD2, Neil McAuslane, BSc, MSc, PhD2.
1Research Analyst, CIRS, London, United Kingdom, 2Centre for Innovation in Regulatory Science, London, United Kingdom.
1Research Analyst, CIRS, London, United Kingdom, 2Centre for Innovation in Regulatory Science, London, United Kingdom.
OBJECTIVES: Approximately 300 million people, globally, are affected by one of the 7,000 estimated rare diseases. Regulatory agencies have provided incentives to address challenges caused by small patient populations. However, the high uncertainty around their data remains challenging for Health Technology Assessment (HTA) agencies. This study aims to investigate how UK HTA agencies assess orphan New Active Substances (NAS).
METHODS: Time and 1st outcome of HTA assessments for orphan and non-orphan NASs (UK-designated) were collected from NICE and SMC websites between January 2019 and December 2024. Additionally, SMC kindly provided their HTA submission dates.
RESULTS: We identified 52 NICE and 40 SMC assessments for orphan NASs between 2019 and 2024. In England, 48 received a positive or positive with restrictions outcome (92%), and 14 NASs of these underwent the highly specialised technology pathway (29%). In Scotland, 32 orphan NASs received a positive or positive with restrictions outcome (80%), and 22 underwent the orphan and ultra-orphan pathway (69%). In both England and Scotland, the HTA assessment time was longer for the orphan NASs compared to non-orphan products (England, 447 vs 331 days; Scotland, 242 vs 193 days). The Patient Access Scheme (PAS) was applied for the majority of the recommendations in England and Scotland (67% and 85% of orphan NASs, respectively).
CONCLUSIONS: A high proportion of NICE and SMC orphan NASs received a positive or positive with restrictions HTA outcome, predominantly enabled by the PAS. However, the assessment time for orphan NASs is longer than non-orphans which may be due to the need for different procedural processes.
METHODS: Time and 1st outcome of HTA assessments for orphan and non-orphan NASs (UK-designated) were collected from NICE and SMC websites between January 2019 and December 2024. Additionally, SMC kindly provided their HTA submission dates.
RESULTS: We identified 52 NICE and 40 SMC assessments for orphan NASs between 2019 and 2024. In England, 48 received a positive or positive with restrictions outcome (92%), and 14 NASs of these underwent the highly specialised technology pathway (29%). In Scotland, 32 orphan NASs received a positive or positive with restrictions outcome (80%), and 22 underwent the orphan and ultra-orphan pathway (69%). In both England and Scotland, the HTA assessment time was longer for the orphan NASs compared to non-orphan products (England, 447 vs 331 days; Scotland, 242 vs 193 days). The Patient Access Scheme (PAS) was applied for the majority of the recommendations in England and Scotland (67% and 85% of orphan NASs, respectively).
CONCLUSIONS: A high proportion of NICE and SMC orphan NASs received a positive or positive with restrictions HTA outcome, predominantly enabled by the PAS. However, the assessment time for orphan NASs is longer than non-orphans which may be due to the need for different procedural processes.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA318
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Rare & Orphan Diseases