OBJECTIVES: Regulatory bodies offer expedited approvals based on single-arm-trial evidence for new medicines that address important unmet clinical needs. This in turn means that non-comparative data explored within a trial might then be used in reimbursement applications. Hence, we aimed to understand the type of analyses conducted in technology appraisals (TAs) to demonstrate indirect comparative efficacy, and themes in NICE decision-making when based on single-arm-trial data.

METHODS: We manually searched the NICE website for TAs in the previous year (May 2017 – May 2018) to identify submissions that included at least one single-arm trial and no RCT evidence to support the technology. Details for each TA that met our inclusion criteria were extracted and assessed.

RESULTS: Among the submissions for the 77 NICE-TAs, 11 used single-arm trial data without any additional RCT evidence. Of these 11 TAs (nine for oncology indications), seven did not perform population-adjusted indirect comparisons (PAIC; three included an unadjusted indirect comparison and four provided a narrative summary only). Of the four with PAICs, three were matching-adjusted indirect comparisons (MAICs) and one, a simulated treatment comparison (STC). Ultimately, NICE recommended ten (of 11) technologies, including eight (of the nine) oncology drugs, seven of which satisfied end-of-life criteria; and five treatments were made available via the Cancer Drug Funds (CDF). Of the oncology technologies recommended outside of the CDF two satisfied end-of-life criteria and performed an MAIC; one did not perform a PAIC but was considered innovative. Across all indications, NICE considered seven (of 10) technologies to be innovative.

CONCLUSIONS: The study highlights situations in which NICE has been prepared to recommend a technology based on non-comparative, single-arm trial data, despite the associated clinical uncertainty. These include technologies considered innovative, and in the case of oncology indications, because they extend otherwise severely reduced life expectancy in a small population.