NICE Has Embraced Single-Arm Trials in Technology Appraisals but Where Is the Guidance?
Author(s)
Eleni Karinou, PhD, Charlotte Mitchell, BSc, Kacey Rawson, MSc.
PPD Evidera Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom.
PPD Evidera Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom.
OBJECTIVES: Guidance and consistency in decision making by Health Technology Assessment (HTA) agencies on the use of single-arm trials (SAT) as part of appraisals is being outpaced by the deployment of SAT methodology beyond advanced stage cancer and rare disease. This study aims to identify published guidance by National Institute for Health and Care Excellence (NICE) on SAT use in reimbursement submissions and to evaluate the acceptance of technology appraisals (TA) that utilize evidence solely from SATs.
METHODS: We searched the NICE website for guidance on the use of SATs as sufficient evidence for decision-making. NICE TAs published between June 2021 and June 2025 were screened. We counted the number of indications with an SAT-based submission and assessed their indirect comparison methodologies.
RESULTS: No SAT guidance from NICE was identified. There were 385 submissions to NICE, 70 of which were withdrawn. Of the remaining 315 TAs, 16% (n=50) utilised evidence from SATs. Forty-four (88%) SAT-based TAs received a positive recommendation. While oncology remained the predominant area of all SAT-based submissions (72%), a number of TAs in other disease areas were identified, including genetic conditions (14%), blood disorders (10%) and kidney disease (4%). Nineteen (38%) SAT appraisals were in rare diseases. Matching-adjusted indirect comparison was the most common comparative evidence generation method (62%).
CONCLUSIONS: Our research highlights the high acceptance rate of SAT-based submissions by NICE in a wide range of therapy areas beyond oncology and rare disease. In many of the TAs receiving a positive recommendation, uncertainty in the comparative evidence methods was raised and often unresolved in publicly available sources. Equally, in TAs receiving a negative recommendation, uncertainty in the comparative evidence methods was often listed as the primary reason. Consistency and transparency in the appraisal process is not possible without guidance on acceptable methods for incorporating SAT evidence.
METHODS: We searched the NICE website for guidance on the use of SATs as sufficient evidence for decision-making. NICE TAs published between June 2021 and June 2025 were screened. We counted the number of indications with an SAT-based submission and assessed their indirect comparison methodologies.
RESULTS: No SAT guidance from NICE was identified. There were 385 submissions to NICE, 70 of which were withdrawn. Of the remaining 315 TAs, 16% (n=50) utilised evidence from SATs. Forty-four (88%) SAT-based TAs received a positive recommendation. While oncology remained the predominant area of all SAT-based submissions (72%), a number of TAs in other disease areas were identified, including genetic conditions (14%), blood disorders (10%) and kidney disease (4%). Nineteen (38%) SAT appraisals were in rare diseases. Matching-adjusted indirect comparison was the most common comparative evidence generation method (62%).
CONCLUSIONS: Our research highlights the high acceptance rate of SAT-based submissions by NICE in a wide range of therapy areas beyond oncology and rare disease. In many of the TAs receiving a positive recommendation, uncertainty in the comparative evidence methods was raised and often unresolved in publicly available sources. Equally, in TAs receiving a negative recommendation, uncertainty in the comparative evidence methods was often listed as the primary reason. Consistency and transparency in the appraisal process is not possible without guidance on acceptable methods for incorporating SAT evidence.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA254
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes