Demonstrating Similar or Greater Health Benefits Based on Indirect Evidence: A Review of NICE Evaluations That Include a Cost-Comparison Approach

OBJECTIVES: The National Institute for Health and Care Excellence (NICE) cost-comparison pathway (formerly fast-track appraisal pathway) is an expedited reimbursement route for interventions that demonstrate similar/greater health benefits, at similar/lower costs, than technologies already recommended for the same indication. Indirect treatment comparisons (ITCs) are increasingly used to meet these criteria but can be associated with high uncertainty. We reviewed all NICE evaluations using cost-comparison analyses to understand the indirect evidence used and accepted by NICE to demonstrate similar/greater health benefits.

METHODS: NICE evaluations containing cost-comparison analyses with final guidance published between April 2017 and May 2023 were identified. Details of the evidence used to demonstrate similar/greater health benefits for the intervention were extracted, including the ITC methodology and volume of evidence presented.

RESULTS: 19 cost-comparison and 11 single-technology appraisal (STA) evaluations with cost-comparison analyses were identified. 26/30 (86.7%) demonstrated similar/greater health benefits between the intervention and at least one relevant comparator using an ITC; 20/26 (76.9%) of these used a network meta-analysis. Considering the primary clinical endpoint, 3/26 (11.5%) demonstrated statistically significant superiority for the intervention versus comparator(s), 9/26 (34.6%) demonstrated no statistically significant difference, and 5/26 (19.2%) described ‘comparable’ results. Results were redacted for the remainder (34.6%). 2/26 (7.7%) evaluations demonstrated equal efficacy using one endpoint, 5/26 (19.2%) using two, 3/26 (11.5%) using three, and 16/36 (61.5%) using four or more. 18/26 (69.2%) and 2/26 (7.7%) evaluations also demonstrated similar/greater safety and health-related quality of life outcomes through an ITC, respectively.

CONCLUSIONS: There was considerable variation in the indirect evidence used and accepted to demonstrate similar/greater benefits for the intervention. A greater understanding of the threshold of evidence required to support these criteria is needed, particularly with the introduction of NICE’s proportionate approach, which may lead to increasing use of the cost-comparison pathway to expedite patient access whilst reducing required resource.