Cancer medicines : clinical impact, economics, and value

Abstract

Background and Importance: There has been much debate recently over rapidly growing drug expenditures. Cancer medicines, in particular, have driven new brand spending over recent years, and US oncological expenditures have risen faster than for many other disease areas, in part because of rapidly growing drug prices, as well as increased rates of use. Objective: In the face of ongoing debates on how to reasonably control growth in pharmaceutical spending, while also providing patients with the best possible care, this thesis sets out to help address the question of whether growing pharmaceutical expenditures are providing value-for-money to patients and society. Novelty and Empirical Contributions: This thesis is based in part on a systematic review with narrative synthesis of English-language HTA appraisals of the comparative clinical risks and benefits of new cancer medicines, as well as on the novel use of methods to generate comparative evidence on their use and cost. Adapting established methods, these data are then used to examine existing questions over whether growing expenditures are worth the cost to patients and society. This thesis makes five major contributions to the literature on value-based spending on cancer medicines: 1) approximately one in three newly licensed cancer medicines provide no known overall survival benefit, while one in five provide no known overall survival, quality of life, or safety benefit; 2) novel use of methodologies to model treatment course and duration reveals that cancer drug use and costs vary greatly between individual medicines, and across Australia, France, the UK, and the US; 3) the monetized value of survival gains attributable to cancer drug innovation, net of growth in cancer drug spending, varies across individual medicines, and, at a country-level, remains unambiguously positive in Australia, France, and the UK, but negative in the US; 4) spending on new cancer medicines is often only weakly associated with their clinical benefits; and 5) the strength of this association nevertheless varies across countries, with the UK demonstrating the strongest evidence of value-based spending on new cancer medicines. Clinical and Policy Implications: Findings from this thesis provide a resource for valuebased clinical decision-making by patients and physicians. Moreover, growing expenditures on cancer medicines may only weakly be associated with meaningful clinical benefits, though the extent to which this is true differs across countries. These findings highlight the important role that health policy can have in encouraging valuebased cancer drug spending. In particular, it is argued that managed access schemes promoting access and evidence development, as well as the use of value-based spending policies, can help expedite access to new treatments, incentivize the development of clinically meaningful medicines, and rationalize growing cancer drug expenditures. Future Research Directions: The comparative clinical risks and benefits from new cancer medicines using real-world data, and how they compare with trial-based results; how evidence on the comparative impact from new treatments is measured, weighted, and rewarded in decision-making by regulators and payers; and how it is effectively linked through policy and regulation to cancer drug spending.