Using evidence to set priorities in health: an analysis of decisions of the Pharmaceutical Benefits Advisory Committee

澳大利亚率先采用严格的临床和经济证据来评估药物，然后再资助我们的国家补贴药物福利计划。在引入药品在获得补贴之前必须证明成本效益的要求后的十年里，一直没有对该系统进行正式的独立评估来评估其绩效。该项目将审查药品福利咨询委员会在过去十年中的建议，并考虑解释这些决定的因素。有时有人断言，这些决定是武断的，或基于不适当的考虑，例如政府的财政成本或当时的政治，而不是有关药物的物有所值。我们将审查反对以具有成本效益的方式提供改善生命的药物这一目标的决定背后的原因。我们会看一种药物是否被推荐或拒绝上市的关键决定因素。一个关键的焦点将是，就这些决定因素是否符合计划的目标而言，这些决定因素是否可以被描述为合法的。例如，拒绝的主要原因是缺乏关于有效性的高质量证据——成本效益，还是仅仅是因为政府的高财政成本等因素。该项目将为1992-2004年向PBAC提交的所有材料建立一个数据库，使我们能够探讨决策过程在更广泛地使用关于保健方面的有效性和成本的证据方面以及针对PBS的证据方面的有效性的若干问题。在强调证据及其解释的一些问题时，总体目标是提高未来决策过程的质量。

Australia has pioneered the use of rigorous clinical and economic evidence in the evaluation of drugs prior to funding on our nationally subsidised Pharmaceutical Benefits Scheme. In the ten years since the introduction of the requirement that drugs demonstrate cost effectiveness prior to subsidy being granted there has been no formal independent evaluation of the system to assess its performance. This project will examine the recommendations of the Pharmaceutical Benefits Advisory Committee in the last decade and consider the factors that explain those decisions. At times it has been asserted that those decisions have been arbitrary or based on inappropriate considerations such as the financial cost to government or politics of the day rather than the value for money of the drug in question. We will examine the reasons behind the decisions against the objectives of providing access to life enhancing medicines in a cost effective manner. We will look at what are the key determinants of whether a drug is recommended for listing on the PBS or is rejected. A key focus will be on whether those determinants could be described as legitimate in terms of their consistency with the objectives of the scheme. For example whether the main cause of rejection is a lack of high quality evidence on effectiveness- cost effectiveness or simply because of factors such as the high financial cost to government. The project will create a database of all submissions to the PBAC 1992-2004 that will allow us to explore a number of questions about the effectiveness of the decision making process in using evidence on effectiveness and costs in health more broadly as well as those specific to the PBS. In highlighting some of the problems with the evidence and its interpretation the overall aim is to improve the quality of the decision making process in the future.