Oncology R&D: Expanding the Role of Economics to Aid Public Policy

三十年的癌症研究已经产生了深刻的见解，这些见解大大降低了某些癌症的发病率和死亡率，并且具有取得更大成功的巨大潜力(1)。有针对性的预防和治疗癌症的方法的发展主要是基础科学和转化临床试验研究与开发（R&D）的大量公共投资的结果(2)。面对高昂且不断增长的治疗成本（3,4），不断增加的试验失败率和支出(5)，以及地方和联邦预算赤字（6,7），资助临床试验的公共机构需要这些投资价值的经验证据，以便为创新和报销政策提供信息，并系统地确定、优先考虑和设计未来的临床试验（8,9,10,11,12,13）。在过去的几十年里，营利性生物制药公司在研发方面投资的增长（14）突出了对公共资源进行深思熟虑的评估和规划的挑战，以及在追求未来的研发时需要一个严格和系统的证据基础。

Three decades of cancer research has produced insights that have dramatically reduced morbidity and mortality for some forms of cancer and has great potential to produce more success (1). The development of targeted approaches to prevent and treat cancer is primarily the result of substantial public investments in basic science and research and development (R&D) in translational clinical trials (2). In the face of high and growing treatment costs (3,4), increasing trial failure rates and spending (5), and local and federal budgetary deficits (6,7) public agencies that fund clinical trials need empirical evidence of the value of these investments to inform innovation and reimbursement policy and systematically identify, prioritize and design future clinical trials (8,9,10,11,12,13). The growth in investment by for-profit biopharmaceutical firms in R&D over the past decades (14) has highlighted the challenge of thoughtful evaluation and planning of the public's resources and the need for a rigorous and systematic evidence base in pursuing future R&D. Economic theory provides a rigorous framework that may aid the translation of advances in basic science into clinical practice (15). Methods have been developed to retrospectively evaluate and prospectively identify, prioritize and design clinical trials (16,17,18,19,20,21). To date, little work has investigated how these methods applied to the unique context of oncology may provide a systematic empirical base for public decision making. My long term research agenda is focused on applying economic methods to address key policy concerns in the financing and organization of oncology clinical trials and practice and ultimately produce practical tools that public decision makers may use to identify and prioritize R&D investments. I am applying for this career development award to afford me the opportunity to gain training in critical areas I require to pursue my long term agenda. The central aspect of my plan is the pursuit of three research projects using advanced economic methods aimed at informing 3 key policy questions. The empirical pursuit of these projects requires focus on specific examples of recent oncology R&D and employs cost benefit analysis, cost-effectiveness analysis, value of information methods and net present value techniques. Although the application of these methods to specific examples cannot definitively answer broad questions, the completion of these projects will prepare me (and hopefully others) for future work aimed at developing a rigorous empirical base for public decision-making in the financing and organization of oncology treatment innovation. I hope that the results of these projects will be of immediate interest to policy making and academic communities. The award is critical to establishing myself as an economist with expertise in the economics of oncology and translational medicine and to producing key results responsive to public policy maker needs.