OBJECTIVES: This study assessed the clinical benefit of oncology regimens for an ultra-rare tumor using concepts from European Society of Medical Oncology (ESMO) and American Society of Clinical Oncology (ASCO) frameworks. It also enhanced clinical assessment of these frameworks by accounting for potential long-term benefits of a novel treatment.

METHODS: An enhanced value framework (EVF) was developed by combining ESMO and ASCO frameworks with well-validated approaches for projecting long-term survival. Published single-arm data for chemotherapies and patient-level data from recently approved immuno-oncology (IO) agent for treating aggressive ultra-rare tumor in metastatic setting was used. Under the ESMO Magnitude of Clinical Benefit Scale (MCBS), form 2a was selected due to lack of sufficient follow-up data on potential curative benefits in terms of overall survival (OS) at the time of approval. Along with OS benefit (1 to 4, indicating increasing OS benefit), MCBS also valued improvements in toxicity and/or quality of life (QoL) (1 for significant improvement in either). The ASCO value framework calculated incremental net health benefit (NHB) by combining scores attributable to clinical benefit, toxicity, and bonus benefits.

RESULTS: The projected mean OS over life-time for IO was 42.5 months in the base case compared with 4.2 months for chemotherapies, driven mostly by improvements in progression-free survival (35.0 vs 2.5 months). Under ESMO MCBS version 1.1, IO scored 3 points higher than chemotherapies for efficacy (4 over 1) as well as 1 for improvement in both toxicity and QoL. Under the ASCO framework, the NHB of IO vs chemotherapies ranged from 212-218, with 193 attributable to clinical benefit, 0-5 to toxicity, and 20 bonus points.

CONCLUSIONS: From HTA perspective, the IO agent for ultra-rare tumor in this case study was determined to provide considerable clinical benefit, irrespective of value framework used to assess benefits associated with the product.