OBJECTIVES: To identify and assess early stage anti-PD1/PD-L1 type immunotherapy (IO) indications appraised by NICE, whether treatment effect waning (TEW) was applied, what considerations/circumstances were listed and what methods were preferred during the appraisal process. In early-stage settings, where the goal of treatments is often cure, the considerations around treatment waning assumptions are different from the traditional metastatic setting.

METHODS: A targeted literature review was undertaken using the NICE website. The NICE technology appraisal (TA) committee papers were reviewed to identify the company’s preferred approach, the Evidence Assessment Group’s (EAG’s) preferred approach and the committee’s preferred approach to TEW. Completed NICE TAs of anti-PD1/PD-L1 type IOs to treat early-stage cancers before, during and/or after treatment with curative intent (e.g. surgery or radiotherapy) were included.

RESULTS: A total of 9 TAs met the final inclusion criteria. Of those, 8 included treatment stopping rules of 1 year. Of the original company submissions, 7 did not include TEW assumptions, 1 assumed TEW started 5 years after treatment initiation and 1 assumed TEW started 10 years after treatment initiation. The EAG requested TEW scenarios in 4 of the 9 TAs. In response to those requests, 2 provided further rationale why TEW was inappropriate and declined to provide scenarios. According to the final draft guidance (FDG) documents, TEW was not explicitly discussed by the committee in 5 of the 9 TAs. Of the FDGs which reported a discussion, the committee acknowledged the uncertainty, and their preferred assumption was often unclear.

CONCLUSIONS: This review provides insights into the assumptions used to model long-term treatment effect in early stage anti-PD1/PD-L1 type IO indications appraised by NICE. These results show that TEW assumptions are not routinely imposed by NICE in the early-stage settings. Characterizing the current precedent is important as precedent often informs approaches to analysis and decision-making in NICE appraisals.