The cost of healthcare continues to rise, particularly for novel biologic oral oncolytic drugs, which have experienced an unprecedented rate of development in recent years. While these precision targeted therapies continue to expand the arsenal of treatment interventions available to providers, critics have described the data leading to many of their approvals as statistically significant but clinically meaningless. Imatinib remains the only precision therapeutic that has theoretically cured an advanced malignancy, and in the years since its FDA approval in May of 2001, the promise of oral biologic targeted small-molecule cancer drugs arguably has waned. Some have suggested that cancer care is at an inflection point. When therapies deliver only marginal clinical benefit, selection of the appropriate treatment for a cancer patient needs to include cost among the traditional factors of efficacy, safety, and patient preference. Overall, many agree that drug launch prices are not proportional to the health benefit they provide.1 Over the past several years frameworks have been developed by professional associations, cancer centers, and payers in the attempt to reconcile these factors in the context of value.
The value of any therapeutic intervention in its simplest sense includes the total costs associated with that intervention divided by the clinical benefit provided—a cost-to-benefit ratio in its purest form. The complication arises in taking that definition and determining what costs and what benefit should be considered in the calculation. The American Society of Clinical Oncology (ASCO), the National Comprehensive Cancer Network (NCCN), Memorial Sloan-Kettering Cancer Center (MSKCC), the Institute for Clinical and Economic Review (ICER), and others have developed frameworks or tools for calculating clinical benefit alongside cost in order to compare treatment options. A summary of these tools, the metrics they employ, and the audiences they are meant to target remains an ongoing topic of debate.2 These frameworks and tools are widely diverse in their approaches, presentation of data, and the insights that can be gleaned, and as such, there has been a renewed focus by other organizations, such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), to generate a consistent paradigm that seeks to leverage the advantages of each model.3