Office of Health Economics, London, UK.
Pharmacoeconomics. 2010;28(2):93-102. doi: 10.2165/11314080-000000000-00000.
This article examines performance-based risk-sharing agreements for pharmaceuticals from a theoretical economic perspective. We position these agreements as a form of coverage with evidence development. New performance-based risk sharing could produce a more efficient market equilibrium, achieved by adjustment of the price post-launch to reflect outcomes combined with a new approach to the post-launch costs of evidence collection. For this to happen, the party best able to manage or to bear specific risks must do so. Willingness to bear risk will depend not only on ability to manage it, but on the degree of risk aversion. We identify three related frameworks that provide relevant insights: value of information, real option theory and money-back guarantees. We identify four categories of risk sharing: budget impact, price discounting, outcomes uncertainty and subgroup uncertainty. We conclude that a value of information/real option framework is likely to be the most helpful approach for understanding the costs and benefits of risk sharing. There are a number of factors that are likely to be crucial in determining if performance-based or risk-sharing agreements are efficient and likely to become more important in the future: (i) the cost and practicality of post-launch evidence collection relative to pre-launch; (ii) the feasibility of coverage with evidence development without a pre-agreed contract as to how the evidence will be used to adjust price, revenues or use, in which uncertainty around the pay-off to additional research will reduce the incentive for the manufacturer to collect the information; (iii) the difficulty of writing and policing risk-sharing agreements; (iv) the degree of risk aversion (and therefore opportunity to trade) on the part of payers and manufacturers; and (v) the extent of transferability of data from one country setting to another to support coverage with evidence development in a risk-sharing framework. There is no doubt that--in principle--risk sharing can provide manufacturers and payers additional real options that increase overall efficiency. Given the lack of empirical evidence on the success of schemes already agreed and on the issues we set out above, it is too early to tell if the recent surge of interest in these arrangements is likely to be a trend or only a fad.
本文从理论经济学的角度考察了药品的基于绩效的风险分担协议。我们将这些协议定位为证据开发覆盖的一种形式。新的基于绩效的风险分担可能会通过调整上市后的价格来反映结果，并结合新的方法来收集上市后证据成本，从而实现更有效的市场均衡。为此，必须由最有能力管理或承担特定风险的一方来承担。承担风险的意愿不仅取决于管理风险的能力，还取决于风险厌恶程度。我们确定了三个相关框架，这些框架提供了相关的见解：信息价值、实物期权理论和退款保证。我们确定了四种风险分担类别：预算影响、价格折扣、结果不确定性和亚组不确定性。我们得出结论，信息价值/实物期权框架可能是理解风险分担成本和收益最有帮助的方法。有几个因素可能在决定基于绩效或风险分担的协议是否有效以及未来是否会变得更加重要方面发挥关键作用：（i）上市后证据收集的成本和可行性与上市前相比；（ii）在没有预先约定如何使用证据来调整价格、收入或使用的情况下，进行证据开发覆盖的可行性，其中围绕额外研究回报的不确定性会降低制造商收集信息的动力；（iii）编写和监管风险分担协议的难度；（iv）支付方和制造商的风险厌恶程度（因此有交易的机会）；以及（v）从一个国家环境向另一个国家转移数据的程度，以支持风险分担框架下的证据开发覆盖。毫无疑问，原则上，风险分担可以为制造商和支付方提供额外的实物期权，从而提高整体效率。鉴于已经商定的计划的成功案例和我们上面列出的问题缺乏经验证据，现在判断最近对这些安排的兴趣热潮是否可能成为一种趋势还是仅仅是一种时尚还为时过早。
