COMPARING EUROPEAN AVAILABILITY METRICS WITH REAL-WORLD PATIENT ACCESS FOLLOWING NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE (NICE) RECOMMENDATION IN THE UK
Author(s)
Ella Barker, Ug1, Sam Large, BA, MSc1, Eddy Tye, Ug1, Phillip O'Neill, BA2, Saskia van Dijk, BSc3.
1Pfizer, Tadworth, United Kingdom, 2Office of Health Economics, London, United Kingdom, 3IQVIA, Amsterdam, Netherlands.
1Pfizer, Tadworth, United Kingdom, 2Office of Health Economics, London, United Kingdom, 3IQVIA, Amsterdam, Netherlands.
OBJECTIVES: The European Federation of Pharmaceutical Industries and Associations (EFPIA) Patients Waiting to Access Innovative Therapies (W.A.I.T.) Indicator 2025 Survey reports 61% of new medicines are available (positive NICE decision) in the UK, with 45% having limited availability. However, core W.A.I.T. metrics focus on new medicines and may miss licence extensions that capture additional availability. This study evaluated how availability metrics compare with UK patient access using M-scores (an established method measuring patient access in NICE Technology Appraisals (TAs)) and assessing license extensions.
METHODS: A retrospective review of 92 NICE TAs published January to December 2025 was conducted. M scores measure patient access as the proportion of the licensed population eligible under the NICE recommendation, derived by comparing the UK licensed indication with NICE restrictions using resource impact templates (RITs). Higher scores indicate broader access. UK licence extension status was also assessed.
RESULTS: 32 (51%) of 63 positive TAs were optimised. The mean M-score for optimised TAs was 42%, resulting in a M-score of 79% across all TAs. Licence extensions comprised 54% of TAs and had higher mean M-scores (58% versus 39% for new medicines), suggesting less restricted access. Data availability limited analysis, with only 18/32 optimised TAs containing extractable M-score data.
CONCLUSIONS: Although the W.A.I.T Indicator reports 61% UK availability, this does not reflect true patient access. Among medicines with limited availability (45%), the average M-score (42%), demonstrated the majority do not have access under limited availability. As licence extensions account for the majority of TAs (54%) and M-scores are excluded from current metrics, there is potentially a significant gap between reported versus actual availability. Findings align with long-term patterns from previous Office of Health Economics studies, however results are limited by incomplete NICE RIT reporting. Overall, incorporating license extensions and M-score metrics are essential to accurately compare medicine availability.
METHODS: A retrospective review of 92 NICE TAs published January to December 2025 was conducted. M scores measure patient access as the proportion of the licensed population eligible under the NICE recommendation, derived by comparing the UK licensed indication with NICE restrictions using resource impact templates (RITs). Higher scores indicate broader access. UK licence extension status was also assessed.
RESULTS: 32 (51%) of 63 positive TAs were optimised. The mean M-score for optimised TAs was 42%, resulting in a M-score of 79% across all TAs. Licence extensions comprised 54% of TAs and had higher mean M-scores (58% versus 39% for new medicines), suggesting less restricted access. Data availability limited analysis, with only 18/32 optimised TAs containing extractable M-score data.
CONCLUSIONS: Although the W.A.I.T Indicator reports 61% UK availability, this does not reflect true patient access. Among medicines with limited availability (45%), the average M-score (42%), demonstrated the majority do not have access under limited availability. As licence extensions account for the majority of TAs (54%) and M-scores are excluded from current metrics, there is potentially a significant gap between reported versus actual availability. Findings align with long-term patterns from previous Office of Health Economics studies, however results are limited by incomplete NICE RIT reporting. Overall, incorporating license extensions and M-score metrics are essential to accurately compare medicine availability.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA157
Topic
Health Policy & Regulatory, Health Technology Assessment, Study Approaches
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas