Assessing the Potential Impact of NICE’s Updated HST Routing Criteria on Ultraorphan Drugs
Author(s)
Jelena Sostar, MSc1, April Dominique Ocampo, BSc2, Andrea Bernardini, PhD3, Vishwas R. Agashe, PhD3.
1Certara Evidence and Access, Correggio, Italy, 2Certara Evidence and Access, Makati, Philippines, 3Certara Evidence and Access, London, United Kingdom.
1Certara Evidence and Access, Correggio, Italy, 2Certara Evidence and Access, Makati, Philippines, 3Certara Evidence and Access, London, United Kingdom.
OBJECTIVES: Highly Specialised Technology (HST) evaluations by the National Institute of Health and Care Excellence (NICE) in United Kingdom are designed to facilitate assessment of treatments for ultra-rare, debilitating conditions with their known challenges in evidence generation for very small patient populations. Recently, NICE made changes to the HST routing criteria to improve their clarity and consistency without affecting the number of therapies assessed via the HST programme. We applied the updated routing criteria to previously conducted HST appraisals to understand their potential impact on the eligibility of ultra-orphan therapies for this programme.
METHODS: All HST reports published by NICE till date were extracted and benchmarked against each of the four updated routing criteria to assess their current eligibility for the HST programme.
RESULTS: Benchmarking n=30 HST appraisals revealed that ~37% (11/30) of appraisals for 10 distinct therapies may not meet either n=1 (10/11) or n=2 (1/11) of the n=4 updated routing criteria, if interpreted strictly. Of these, 1/11 may not meet the ‘ultra-rare’ criterion, 3/11 the ‘innovation’ criterion, 2/11 the ‘narrow eligible population’ criterion, and 6/11 the ‘no existing effective treatment’ criterion; inclusive of 1/11 that may not meet the ‘innovation’ and ‘no existing effective treatment’ criteria. Illustratively, afemelanotide (HST27) may not qualify because the estimated 390 eligible patients in England would lie marginally above the specified ‘≤300 patients’ threshold.
CONCLUSIONS: Although the updated HST routing criteria may facilitate benchmarking of therapies against a set of better-defined eligibility metrics, inflexibility in their application could potentially make it harder for ultra-orphan drugs to access the HST pathway. Given notable variation in the number of HST assessments conducted annually, the real-world impact of these changes will take time to be established, which may raise perceived uncertainty about securing the HST designation among health technology developers.
METHODS: All HST reports published by NICE till date were extracted and benchmarked against each of the four updated routing criteria to assess their current eligibility for the HST programme.
RESULTS: Benchmarking n=30 HST appraisals revealed that ~37% (11/30) of appraisals for 10 distinct therapies may not meet either n=1 (10/11) or n=2 (1/11) of the n=4 updated routing criteria, if interpreted strictly. Of these, 1/11 may not meet the ‘ultra-rare’ criterion, 3/11 the ‘innovation’ criterion, 2/11 the ‘narrow eligible population’ criterion, and 6/11 the ‘no existing effective treatment’ criterion; inclusive of 1/11 that may not meet the ‘innovation’ and ‘no existing effective treatment’ criteria. Illustratively, afemelanotide (HST27) may not qualify because the estimated 390 eligible patients in England would lie marginally above the specified ‘≤300 patients’ threshold.
CONCLUSIONS: Although the updated HST routing criteria may facilitate benchmarking of therapies against a set of better-defined eligibility metrics, inflexibility in their application could potentially make it harder for ultra-orphan drugs to access the HST pathway. Given notable variation in the number of HST assessments conducted annually, the real-world impact of these changes will take time to be established, which may raise perceived uncertainty about securing the HST designation among health technology developers.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR30
Topic
Health Policy & Regulatory, Health Technology Assessment, Organizational Practices
Topic Subcategory
Reimbursement & Access Policy
Disease
Rare & Orphan Diseases