Department of Public Health Sciences, University of Alberta, Edmonton, Alberta, Canada.
Pharmacoeconomics. 2010;28(2):113-42. doi: 10.2165/11530820-000000000-00000.
As tensions between payers, responsible for ensuring prudent and principled use of scarce resources, and both providers and patients, who legitimately want access to technologies from which they could benefit, continue to mount, interest in approaches to managing the uncertainty surrounding the introduction of new health technologies has heightened. The purpose of this project was to compile an inventory of various types of 'access with evidence development' (AED) schemes, examining characteristics of the technologies to which they have been applied, the uncertainty they sought to address, the terms of arrangements of each scheme, and the policy outcomes. It also aimed to identify issues related to such schemes, including advantages and disadvantages from the perspectives of various stakeholder groups. A comprehensive search, review and appraisal of peer-reviewed and 'grey' literature were performed, followed by a facilitated workshop of academics and decision makers with expertise in AED schemes. Information was extracted and compiled in tabular form to identify patterns or trends. To enhance the validity of interpretations made, member checking was performed. Although the concept of AED is not new, evaluative data are sparse. Despite varying opinions on the 'right' answers to some of the questions raised, there appears to be consensus on a 'way forward'--development of methodological guidelines. All stakeholders seemed to share the view that AEDs offer the potential to facilitate patient access to promising new technologies and encourage innovation while ensuring effective use of scarce healthcare resources. There is no agreement on what constitutes 'sufficient evidence', and it depends on the specific uncertainty in question. There is agreement on the need for 'best practice' guidelines around the implementation and evaluation of AED schemes. This is the first attempt at a comprehensive analysis of methods that have been used to address uncertainty concerning a new drug or other technology. The analysis reveals that, although various approaches have been experimented with, many of them have not achieved the ostensible goal of the approach. This article outlines challenges related to AED schemes and issues that remain unresolved.
随着支付方(负责确保审慎和有原则地使用稀缺资源)与医疗服务提供者和患者(他们希望获得可能使他们受益的技术)之间的紧张关系持续加剧,人们对管理引入新医疗技术所带来的不确定性的方法越来越感兴趣。本项目的目的是编制各种类型的“有证据开发的准入”(AED)计划清单,研究它们所应用的技术特征、它们试图解决的不确定性、每个计划的安排条款以及政策结果。它还旨在确定与这些计划相关的问题,包括从各个利益相关者群体的角度来看的优缺点。对同行评议和“灰色”文献进行了全面的搜索、审查和评估,然后对具有 AED 计划专业知识的学者和决策者进行了专题研讨会。以表格形式提取和编制信息,以确定模式或趋势。为了提高所作解释的有效性,进行了成员检查。尽管 AED 的概念并不新鲜,但评估数据却很少。尽管对一些提出的问题的“正确”答案存在不同意见,但似乎在“前进”的方法上达成了共识——制定方法学指南。所有利益相关者似乎都认为 AED 有可能促进患者获得有前途的新技术,并鼓励创新,同时确保有效利用稀缺的医疗资源。对于什么构成“充分证据”没有达成一致意见,这取决于具体的不确定性。对于 AED 计划的实施和评估,围绕“最佳实践”指南的必要性达成了一致意见。这是首次对用于解决新药或其他技术不确定性的方法进行全面分析。分析表明,尽管已经尝试了各种方法,但其中许多方法并没有达到方法的预期目标。本文概述了与 AED 计划相关的挑战以及尚未解决的问题。