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

描述(由申请人提供)：30年的癌症研究已经产生了深刻的见解，极大地降低了某些形式癌症的发病率和死亡率，并具有取得更大成功的巨大潜力(1)。开发有针对性的预防和治疗癌症的方法主要是对基础科学和转化临床试验的研发(R&D)进行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，以告知创新和补偿政策，并系统地确定、确定和设计未来的临床试验(8，9，10，11，12，13)。营利性生物制药公司在过去几十年中在研发方面的投资增长(14)突显了对公众资源进行深思熟虑的评估和规划的挑战，以及在进行未来研发时需要严格和系统的证据基础。 经济学理论提供了一个严格的框架，可能有助于将基础科学的进步转化为临床实践(15)。已经开发了方法来对临床试验(16、17、18、19、20、21)进行回顾评估和前瞻性识别、优先排序和设计。到目前为止，很少有工作调查这些方法如何应用于肿瘤学的独特背景下，为公共决策提供系统的经验基础。我的长期研究议程集中在应用经济方法来解决肿瘤学临床试验和实践的融资和组织中的关键政策问题，并最终产生实用工具，供公共决策者用来确定研发投资的优先顺序。 我申请这一职业发展奖是为了让我有机会获得关键领域的培训，这是我追求长期目标所必需的。我的计划的中心方面是利用先进的经济方法进行三个研究项目，旨在揭示三个关键的政策问题。这些项目的经验性追求需要关注最近肿瘤学研发的具体例子，并采用成本效益分析、成本效益分析、信息价值方法和净现值技术。 尽管将这些方法应用于具体的例子不能确切地回答广泛的问题，但这些项目的完成将为我(希望还有其他人)未来的工作做好准备，旨在为肿瘤治疗创新的融资和组织方面的公共决策建立严格的经验基础。我希望这些项目的结果将立即引起决策和学术界的关注。该奖项对于将我确立为一名拥有肿瘤学和转化医学经济学专业知识的经济学家，以及为回应公共政策制定者的需求而产生关键成果至关重要。

DESCRIPTION (provided by applicant): 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.