OBJECTIVES: Single-arm trials (SATs) have been increasingly used to support oncology technical appraisals (TAs) by the National Institute for Health and Care Excellence (NICE), despite known limitations in providing comparative evidence. The objective of this study is to assess the frequency of SAT-based oncology NICE TAs, the approach to generating comparative evidence, and recommendations from the committee review.

METHODS: Oncology NICE TAs published in the past five years were reviewed to identify submissions using only SATs as clinical evidence. Full-text screening of the committee papers and TA guidance was conducted by a single investigator and the abstracted data were validated by a second investigator. Information on drug of interest, indication, trial design, external comparator data, and committee commentaries was extracted.

RESULTS: Of 191 oncology NICE TAs identified between May 2017 to May 2022, 32 (17%) used only SATs as primary clinical evidence. Comparative evidence was generated from various sources, including comparator trials, real-world evidence (RWE), and SATs (intra-patient comparison). More than one source or approach was applied in some submissions: 12 (38%) naïve comparison and 25 (78%) adjusted indirect treatment comparison (ITC), including 21 (66%) matching-adjusted indirect comparison (MAIC) and 4 (12%) synthetic control. Of submissions using MAIC, 13 used comparator trials, 7 used RWE, and 1 used both. In spite of the high uncertainty around treatment benefits generated from SATs, the committee has granted recommendation to 91% (29 of 32) of SAT-based TAs, while half were recommended for use within the Cancer Drugs Fund. The decisive factor is whether plausible incremental cost-effectiveness ratios can meet the conventional cost-effectiveness thresholds, which are determined by the relative treatment effect demonstrated through ITC. Unmet medical need and end-of-life criteria are also being considered.

CONCLUSIONS: SAT-based submissions to NICE have a high acceptance rate. Sensible ITC approaches can lead to positive reimbursement decisions.