Brown School of Public Health, Providence, RI, USA.
Harvard Graduate School of Arts and Sciences, Cambridge, MA, USA.
Value Health. 2022 Jul;25(7):1141-1147. doi: 10.1016/j.jval.2021.11.1375. Epub 2022 Feb 24.
New health technologies are often expensive, but may nevertheless meet standard thresholds for cost effectiveness, a situation exemplified by recent hepatitis C cures. Currently, cost-effectiveness analysis (CEA) does not supply practical means of weighing trade-offs between cost-effectiveness and affordability, particularly when costs and benefits are temporally separated and in health systems with multiple payers, such as the United States. We formally characterized disagreements in CEA theory and identified how these trade-offs are presently addressed in practice.
We surveyed 170 health economics researchers.
When presented with a hypothetical cost-effective drug therapy in the United States that would require 20% of a state's Medicaid budget over 5 years, 34% of survey respondents recommended that policy makers fund the drug for all patients and 26% for a subset. By contrast, 26% recommended against funding the drug. We found additional disagreement regarding whether the willingness-to-pay threshold should be based on the budget (42%) or societal preferences (41%) and identified 4 approaches to weighing cost-effectiveness and affordability. A total of 61% of respondents did not believe that the threshold used in their last article (most often 1×-3× per capita gross domestic product) represented either the budget or societal willingness-to-pay threshold.
We use these findings to recommend metrics that can inform translation of CEA theory into practice. By contextualizing cost and value, researchers can provide more actionable policy recommendations.
新的医疗技术通常很昂贵，但仍可能符合成本效益的标准门槛，最近的丙型肝炎治愈案例就是一个例证。目前，成本效益分析（CEA）并没有提供实用的方法来权衡成本效益与可负担性之间的权衡，特别是当成本和效益在时间上分离且在多个付款人（如美国）的医疗体系中。我们正式描述了 CEA 理论中的分歧，并确定了目前在实践中如何解决这些权衡。
我们调查了 170 名卫生经济学研究人员。
当在美国提出一种需要在 5 年内使用 20%的州医疗补助预算的假设性成本效益药物治疗时，34%的调查受访者建议决策者为所有患者和 26%的患者子集提供药物资金。相比之下，26%的受访者建议不资助该药物。我们还发现了关于是否应根据预算（42%）或社会偏好（41%）来确定支付意愿阈值的额外分歧，并确定了 4 种权衡成本效益和可负担性的方法。共有 61%的受访者认为他们在上一篇文章中使用的阈值（通常是人均国内生产总值的 1×-3×）既不是预算也不是社会支付意愿的阈值。
我们利用这些发现来推荐可以将 CEA 理论转化为实践的指标。通过使成本和价值具体化，研究人员可以提供更具可操作性的政策建议。
