Ensuring Access to Novel Alzheimer’s and Dementia Treatments: Evaluating Innovative Payment Approaches

项目摘要/摘要 治疗阿尔茨海默病和相关痴呆症(ADRD)的进展缓慢得令人沮丧，但很有效 治疗方法可能即将出现。考虑到ADRD的规模和成本，即使是在拖延时间方面微不足道的效果 进步可能会带来巨大的社会效益。然而，ADRD问题的规模也呈现出一个 融资挑战。我们怎样才能为大量的ADRD患者和那些 症状前期？临床益处可能因疾病阶段而不同而加剧了这一问题， 基因或其他未知因素。付款人不愿为每剂疗法支付高昂的价格，尤其是 当这些好处可能在以后的生活中积累起来的时候。我们将汇聚经济学专家， 神经科学和ADRD关怀，以确定和量化替代报销战略，以确保更好地 进入。首先，使用兰德/加州大学洛杉矶分校适当性方法的变体，我们将确定最有希望的 ADRD干预措施，量化其成功的可能性和预期的临床益处，并评估这些 不同的患者亚组的福利可能有所不同。第二，我们将绘制用于 目前ADRD正在努力获得更广泛的生活质量结果--例如，功能状态、疾病发病率和 死亡风险--可以用来估计社会后果。第三，借鉴经济理论和 现有文献，我们将为ADRD疗法开发新的报销机制，并描述如何 它们将是有组织的。第四，我们将采用经过充分验证的微观模拟工具--未来老年人模型 模型，以研究新的反兴奋剂技术的长期健康和经济后果，并研究如何 这些后果会因不同的定价机制而有所不同。我们将研究平均结果和 在替代技术和定价方案下，ADRD患者之间的差异变化。第五，我们将使用 研究技术和定价情景对健康和经济影响的微观模拟模型 ADRD患者非正式照顾者的结果。最后，我们将召开一个研讨会，传播我们的 向主要市场和政策利益攸关方提供调查结果，并就下一步可能采取的措施展开讨论。 总之，拟议的研究将开发和评估创新战略，以确保当前和未来 ADRD患者可以获得新的治疗方法。

Project Summary/Abstract Progress against Alzheimer's disease and related dementias (ADRD) has been frustratingly slow, but effective treatments may be on the horizon. Given the scale of ADRD and its costs, even modest efficacy in delaying progression could have substantial social benefits. However, the scale of the ADRD problem also presents a financing challenge. How can we afford new therapies for large numbers of ADRD patients and those who are pre-symptomatic? This problem is exacerbated by the likelihood that clinical benefits will vary with disease stage, genotype, or other unknown factors. Payers are reluctant to pay high prices per dose for therapies, especially when these benefits may accumulate much later in life. We will bring together experts in economics, neuroscience, and ADRD care to identify and quantify alternative reimbursement strategies that ensure better access. First, using a variant of the RAND/UCLA appropriateness methods, we will identify the most promising ADRD interventions, quantify their likelihood of success and expected clinical benefit, and assess whether these benefits are likely to vary across patient subgroups. Second, we will map clinical trial endpoints used in the current ADRD pipeline to broader quality-of-life outcomes – e.g., functional status, disease incidence, and mortality risk – that can be used to estimate societal consequences. Third, drawing on economic theory and the exitant literature, we will develop novel reimbursement mechanisms for ADRD therapies and characterize how they would be structured. Fourth, we will adapt the Future Elderly Model (FEM), a well-validated microsimulation model, to study the long-term health and economic consequences of new ADRD technologies, and to study how these consequences would vary with different pricing mechanisms. We will study both average outcomes and changes in disparities across ADRD patients for alternative technology and pricing scenarios. Fifth, we will use the microsimulation model to study impacts of technologies and pricing scenarios on the health and economic outcomes of informal caregivers for ADRD patients. Finally, we will convene a workshop to disseminate our findings to key marketplace and policy stakeholders, and to generate discussion about potential next steps. Together, the proposed research will develop and evaluate innovative strategies to ensure that current and future ADRD patients gain access to novel therapies.