Role and cost-effectiveness of biomarker testing in metastatic cancer treatment : policy implications

Abstract

Background Cancer is the top cause of death in Hong Kong and consumes a considerable amount of resources in treatment. The presence of biomarkers as a tool in selecting patients who are likely to benefit from cancer therapy present a challenge to local treatment protocol and policy as there are no specific guideline for biomarker testing. This paper aims to review the evidence of biomarker guided treatment and their cost-effectiveness using lung cancer as an example and recommend appropriate use of these test and relevant policy recommendation for use in cancer treatment.

Method A systematic review was done using PubMed for relevant Phase III efficacy trials on Epidermal Growth Factor Receptor (EGFR), Anaplastic Lymphoma Kinase (ALK) mutation in first-line setting and cost-effectiveness studies on testing for these two mutations.

Findings For the efficacy review, 9 out of the 10 papers or articles met its efficacy primary endpoint. Progression-free survival (PFS) was the mostly used primary endpoint and all biomarker directed therapy favors longer PFS compared with chemotherapy comparator. None of those studies met the primary endpoint/secondary endpoint in improving overall survival. For the cost-effectiveness testing review, EGFR testing was found to be cost-effective in the Eastern-Asia context. EML4-ALK testing was not cost effective.

Conclusion Targeted therapy towards EGFR mutation and EML4-ALK rearrangement was found to improve PFS in patients harboring these genetic abnormalities. Cost-effectiveness analysis show coupling EGFR testing with therapy is a promising approach whereas the test for EML4-ALK rearrangement in all lung cancer patients is not cost-effective. It reminded a policy question to screen for rare mutation like EML4-ALK depending on the ethical perspective taken by the health system. In such case, risk sharing and alternative financing model for rare biomarker can be considered. A reimbursement list for biomarker testing should be in place within Hospital Authority to increase transparency for patients.