Evaluating Oncology Value-Based Frameworks in the U.S. Marketplace and Challenges in Real-World Application: A Multiple Myeloma Test Case

BACKGROUND: With the continuous rise in costs for oncology drugs, the American Society of Clinical Oncology (ASCO), the Institute for Clinical and Economic Review (ICER), the Memorial Sloan Kettering Cancer Center’s Drug Abacus (DrugAbacus), and the National Comprehensive Cancer Network (NCCN) have developed value-based frameworks (VBFs) to assist stakeholders in formulary and treatment decision-making processes. Since emerging VBFs have the potential to affect available treatment options for patients, it is important to understand the differences associated with these VBFs within various therapeutic areas. OBJECTIVES: To (a) compare VBFs across 3 therapeutic options for relapsed or refractory multiple myeloma (RRMM) and (b) identify challenges and limitations associated with real-world decision making using VBFs in the U.S. marketplace. METHODS: The values of regimens carfilzomib (CFZ), elotuzumab (ELO), and ixazomib (IX) were generated using the ASCO, NCCN, ICER, and DrugAbacus VBFs. These regimens, used for second- or third-line treatment of RRMM, shared a common comparator in clinical trials: lenalidomide + dexamethasone (LEN + DEX). ASCO’s 2016 VBF, which incorporated clinical benefit, toxicity, and bonus points, was used to generate a net health benefit score, along with the drug wholesale acquisition cost, for each regimen compared with LEN + DEX. Results of the 2016 NCCN Evidence Blocks for multiple myeloma and the ICER 2016 report of treatment options for RRMM were extracted to generate the value of CFZ, ELO, and IX. No output was generated from DrugAbacus because of the lack of regimens included in the test case. Shortcomings associated with running the test case in RRMM for each VBF were also identified. RESULTS: Among the 3 therapeutic agents, CFZ, in combination with LEN + DEX, was the most valued. ASCO and ICER VBFs suggested that CFZ + LEN + DEX may be the most valued, followed by ELO + LEN + DEX and IX + LEN + DEX. NCCN suggested that LEN + DEX may be the most valued followed by CFZ + LEN + DEX, IX + LEN + DEX, and ELO + LEN + DEX. A number of shortcomings were noted across each VBF, such as complexities of drug evidence evaluation with the ASCO VBF, the inability to adjust the ICER and NCCN VBFs to specific populations, and subjectivity associated with the NCCN VBF and DrugAbacus. CONCLUSIONS: Although the test case provided some consensus on treatment decisions, there is much nuance and limitations with the VBFs available for RRMM. Clearer objectivity and better adaptability to specific treatment decisions are warranted.