A Comparison of the NICE HST Program With Health Technology Assessments of Ultra-Orphan Drugs From France and Germany
Author(s)
Georgia Hollier-Hann, PhD1, Audrey Elaine Fulthorp, PhD1, Steven Horsburgh, PhD1, Stephen Ralston, MSc2.
1Coronado Research, Newcastle upon Tyne, United Kingdom, 2Coronado Research, Guildford, United Kingdom.
1Coronado Research, Newcastle upon Tyne, United Kingdom, 2Coronado Research, Guildford, United Kingdom.
OBJECTIVES: The evaluation of ultra-orphan medicines for extremely rare conditions presents unique challenges for health technology assessment (HTA) bodies due to limited clinical evidence and high uncertainty around cost-effectiveness. NICE (England) addresses these challenges through its Highly Specialised Technologies (HST) programme. This research compares the HST programme with corresponding assessments by HAS (France) and G-BA (Germany).
METHODS: The NICE website was searched to identify HST final evaluations published before June 2025, with corresponding HTAs identified from the HAS and G-BA websites. Decision outcomes and conditions were evaluated. Where multiple HTAs were conducted for each medicine, or multiple decisions were reported within a single HTA (i.e., covering multiple indications), each HTA/decision is counted separately in the results.
RESULTS: Twenty-eight medicines were assessed by NICE, of which HAS and G-BA assessed 26 and 25, respectively. NICE conducted 30 HST assessments across the 28 medicines, of which 93% were recommended. Most positive recommendations required a commercial arrangement (89%) and 21% included a managed access agreement. HAS conducted 27 HTAs covering decisions on 34 indications, of which 82% were reimbursed. ASMR ranged from II to V, with most achieving ‘moderate’ (III; 30%) or ‘minor’ clinical added value (IV; 37%). Eighty-one percent of HTAs included requests for additional data collection, with reassessments planned within 1-5 years. G-BA conducted 26 HTAs, reporting decisions for 31 indications. Most indications had a ‘non-quantifiable added benefit’ (58%) or higher (26%), though 16% had ‘no/less added benefit’. Thirty-eight percent of HTAs had limitations on the duration of validity, with reassessments planned within 2-5 years.
CONCLUSIONS: Despite different methodologies and decision-making criteria, these major European HTA bodies have largely approved the same ultra-orphan medicines. Implementation of commercial arrangements with NICE and pricing negotiations based on HAS and G-BA clinical benefit ratings highlights the varying price of these HSTs.
METHODS: The NICE website was searched to identify HST final evaluations published before June 2025, with corresponding HTAs identified from the HAS and G-BA websites. Decision outcomes and conditions were evaluated. Where multiple HTAs were conducted for each medicine, or multiple decisions were reported within a single HTA (i.e., covering multiple indications), each HTA/decision is counted separately in the results.
RESULTS: Twenty-eight medicines were assessed by NICE, of which HAS and G-BA assessed 26 and 25, respectively. NICE conducted 30 HST assessments across the 28 medicines, of which 93% were recommended. Most positive recommendations required a commercial arrangement (89%) and 21% included a managed access agreement. HAS conducted 27 HTAs covering decisions on 34 indications, of which 82% were reimbursed. ASMR ranged from II to V, with most achieving ‘moderate’ (III; 30%) or ‘minor’ clinical added value (IV; 37%). Eighty-one percent of HTAs included requests for additional data collection, with reassessments planned within 1-5 years. G-BA conducted 26 HTAs, reporting decisions for 31 indications. Most indications had a ‘non-quantifiable added benefit’ (58%) or higher (26%), though 16% had ‘no/less added benefit’. Thirty-eight percent of HTAs had limitations on the duration of validity, with reassessments planned within 2-5 years.
CONCLUSIONS: Despite different methodologies and decision-making criteria, these major European HTA bodies have largely approved the same ultra-orphan medicines. Implementation of commercial arrangements with NICE and pricing negotiations based on HAS and G-BA clinical benefit ratings highlights the varying price of these HSTs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA3
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases