OBJECTIVES: The NICE Health Technology Assessment (HTA) procedure has established the use of indirect treatment comparisons (ITC) to derive efficacy estimates in the absence of direct comparisons. We assessed the use and Evidence Review Groups (ERGs) critique of various ITCs in the NICE technology appraisals (TAs) for haematologic cancers (HC).

METHODS: Final appraisal document, manufacturer submission and ERG report for all HC TAs published by NICE between Jan 2019 to Jun 2023 were reviewed to understand the use of ITC methods, clinical data considered, and ERGs critiques. Terminated appraisals were excluded.

RESULTS: Out of 42 HC TAs appraised by NICE, 48% included a PAIC and 19% included a network meta-analysis (NMA). Of the total submissions that included a PAIC, majority were for multiple myeloma (19%) followed by chronic lymphocytic leukaemia and follicular lymphoma (15% each). Further, 90% of the PAIC were unanchored while the remaining 10% used an anchored approach. Simulated treatment comparison was used in one submission only, however, was not preferred by ERG. Forty-three percent of the HC submissions including a PAIC in 2019 to 60% in 2022. Of all submissions with a PAIC, main reason for conducting a PAIC was study/population heterogeneity (50%) or availability of single arm trial data only (35%). While ERG acknowledged the inherent uncertainty of PAIC approach, it highlighted insufficient matching for effect modifiers in majority of the submissions and did not recommend 20% of these submissions due to high level of uncertainty in the clinical effectiveness results.

CONCLUSIONS: A trend was observed with increasing use of PAICs over the last 5 years in HC TAs. This is likely due to the submissions being based on single arm trial data, increasing complexity of study design and population heterogeneity. Improvement in methodology employed in the PAICs could improve chances of a positive recommendation by NICE.