SickKids Research Institute and University of Toronto, Toronto, ON, Canada.
Pharmacoeconomics. 2012 Jun 1;30(6):447-59. doi: 10.2165/11592250-000000000-00000.
Previous application of value-of-information methods to optimal clinical trial design have predominantly taken a societal decision-making perspective, implicitly assuming that healthcare costs are covered through public expenditure and trial research is funded by government or donation-based philanthropic agencies. In this paper, we consider the interaction between interrelated perspectives of a societal decision maker (e.g. the National Institute for Health and Clinical Excellence [NICE] in the UK) charged with the responsibility for approving new health interventions for reimbursement and the company that holds the patent for a new intervention. We establish optimal decision making from societal and company perspectives, allowing for trade-offs between the value and cost of research and the price of the new intervention. Given the current level of evidence, there exists a maximum (threshold) price acceptable to the decision maker. Submission for approval with prices above this threshold will be refused. Given the current level of evidence and the decision maker's threshold price, there exists a minimum (threshold) price acceptable to the company. If the decision maker's threshold price exceeds the company's, then current evidence is sufficient since any price between the thresholds is acceptable to both. On the other hand, if the decision maker's threshold price is lower than the company's, then no price is acceptable to both and the company's optimal strategy is to commission additional research. The methods are illustrated using a recent example from the literature.
先前将信息价值方法应用于最佳临床试验设计主要采用了一种社会决策制定的视角，隐含地假设医疗保健成本由公共支出覆盖，并且试验研究由政府或基于捐赠的慈善机构资助。在本文中，我们考虑了社会决策者（例如英国的国家卫生与临床优化研究所 [NICE]）相关视角之间的相互作用，这些决策者负责批准新的健康干预措施以获得报销，并考虑了拥有新干预措施专利的公司之间的相互作用。我们从社会和公司的角度建立了最优决策，允许在研究的价值和成本与新干预措施的价格之间进行权衡。考虑到当前的证据水平，存在决策者可接受的最大（阈值）价格。提交的价格高于此阈值的申请将被拒绝。考虑到当前的证据水平和决策者的阈值价格，存在公司可接受的最小（阈值）价格。如果决策者的阈值价格超过公司的价格，那么当前的证据就足够了，因为两个阈值之间的任何价格都是可以接受的。另一方面，如果决策者的阈值价格低于公司的价格，那么两个阈值之间的任何价格都不能被双方接受，并且公司的最佳策略是委托进行额外的研究。该方法使用文献中的一个最近的例子进行了说明。
