OBJECTIVES

Historically HTA submissions for oncology products have focused on the advanced/metastatic setting. There is now an increasing focus on earlier stage treatment, such as adjuvant therapy. Payers generally require overall survival (OS) data for the assessment of oncology treatments; however, adjuvant treatments are typically approved based on trials using surrogate survival endpoints (e.g. relapse-free survival, disease-free survival) due to the time required to collect OS data. This research aims to evaluate the acceptability of surrogate endpoints for adjuvant oncology treatments through recent HTA assessments.

METHODS

Respective websites were screened for publicly available assessments of adjuvant oncology therapies by HTA body (NICE, SMC, G-BA, HAS, PBAC, CADTH) from 1 Jan 2013 to 15 Jan 2019. Key data were extracted.

RESULTS

Eight assessments were identified that received at least draft guidance from an HTA body (NICE: 4; SMC: 1; G-BA: 1; PBAC: 1; CADTH: 1) across four therapies/therapeutic combinations. The pivotal trial primary endpoints were recurrence/relapse-free survival for 5/8 assessments and invasive disease-free survival for 3/8 assessments. Mature OS data were not provided for any submissions. 5/8 assessments were positive outcomes (NICE: 3; SMC: 1; G-BA: 1); 4/5 of these positive outcomes were restricted (e.g. Cancer Drugs Fund or discounts for NICE, or time-limited decision for G-BA). 2/8 assessments presented evidence provided by manufacturers for the correlation of the primary endpoint to OS (both assessments were positive outcomes: NICE: 1; SMC: 1).

CONCLUSIONS

Oncology therapies gaining regulatory approval for the adjuvant setting typically do so based on surrogate survival endpoints. Historically, surrogate endpoints that are sufficient for regulatory approval may not be sufficient to support a favorable HTA outcome. In this research, we show that use of surrogate endpoints data for adjuvant treatments does support positive HTA outcomes; however, most positive outcomes are restricted until more evidence is generated.