A Long and Winding Road: An Evaluation of NICE HST Appraisals Taking Longer Than Two Years for Guidance Publication
Author(s)
Daniela Gonçalves-Bradley, PhD, Christine Worsley, PGC.
STRATENYM Inc., Toronto, ON, Canada.
STRATENYM Inc., Toronto, ON, Canada.
OBJECTIVES: NICE’s Highly Specialised Technology (HST) programme evaluates technologies for ultra-rare, debilitating conditions, aiming to provide equitable access for underserved populations in a timely fashion. However, additional consultations in the HTA process can push back publication of final guidance. This study explored the critiques commonly presented during HST appraisals that experienced long delays.
METHODS: Publicly available HST appraisal data were collected from programme inception to May 2025. The duration of the appraisal process was calculated as the length of time from Final Scope (FS) until Final Evaluation Determination (FED). For those appraisals taking longer than 2 years, the main critiques from the committee and External Assessment Group were extracted and qualitatively analysed.
RESULTS: Thirty HSTs were published, of which two were not recommended. Mean duration of recommended HST appraisals was 81 weeks (range: 39 - 318), with six appraisals taking longer than 2 years from FS until FED. A number of common issues were observed in these most-delayed HST appraisals, including: (1) lack of coherence between the company’s strategy and the presented clinical trial evidence, (2) questionable selection and interpretation of outcome measures, (3) limited description of ancillary studies, (4) incomplete review of previous evidence, including inadequate searches to identify comparator data, (5) model assumptions that were not aligned with current clinical guidance, and (6) methodological limitations of the statistical analysis and/or the economic model.
CONCLUSIONS: Common themes raised during the six most-delayed HST appraisals included methodological limitations of the clinical and economic data, as well as inconsistencies between the company’s strategy and existing evidence. Early engagement with HTA bodies is vital to understand submission requirements, and to develop evidence generation plans that address gaps and support robust economic models with realistic assumptions, avoiding the need for additional consultations and subsequent delays.
METHODS: Publicly available HST appraisal data were collected from programme inception to May 2025. The duration of the appraisal process was calculated as the length of time from Final Scope (FS) until Final Evaluation Determination (FED). For those appraisals taking longer than 2 years, the main critiques from the committee and External Assessment Group were extracted and qualitatively analysed.
RESULTS: Thirty HSTs were published, of which two were not recommended. Mean duration of recommended HST appraisals was 81 weeks (range: 39 - 318), with six appraisals taking longer than 2 years from FS until FED. A number of common issues were observed in these most-delayed HST appraisals, including: (1) lack of coherence between the company’s strategy and the presented clinical trial evidence, (2) questionable selection and interpretation of outcome measures, (3) limited description of ancillary studies, (4) incomplete review of previous evidence, including inadequate searches to identify comparator data, (5) model assumptions that were not aligned with current clinical guidance, and (6) methodological limitations of the statistical analysis and/or the economic model.
CONCLUSIONS: Common themes raised during the six most-delayed HST appraisals included methodological limitations of the clinical and economic data, as well as inconsistencies between the company’s strategy and existing evidence. Early engagement with HTA bodies is vital to understand submission requirements, and to develop evidence generation plans that address gaps and support robust economic models with realistic assumptions, avoiding the need for additional consultations and subsequent delays.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA8
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas