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

确保获得新型阿尔茨海默病和痴呆症治疗方法：评估创新的支付方式

• 批准号: 9789172
• 负责人: DANA P GOLDMAN
• 金额: $ 61.65万
• 依托单位: UNIVERSITY OF SOUTHERN CALIFORNIA
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-30 至 2022-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9789172
• 关键词: Academy; Address; Affect; Aging; Alzheimer' s Disease; Alzheimer' s disease model; Alzheimer' s disease related dementia; American; Americas; Amyloid beta-Protein; Area; Biological Markers; Brain; Cardiovascular Diseases; Caregivers; Cerebrospinal Fluid; Clinical; Clinical Trials; Cognitive; Communicable Diseases; Consensus; Costs and Benefits; Dementia; Disease; Disease Progression; Dose; Early treatment; Economic Models; Economics; Educational workshop; Elderly; Ensure; Family Caregiver; Formularies; Future; Genotype; Goals; HIV/HCV; Health Policy; Health Services Accessibility; Impaired cognition; Incentives; Incidence; Insurance Carriers; Intervention; Life; Literature; Longitudinal Studies; Malignant Neoplasms; Manufacturer Name; Maps; Measures; Mediation; Medical; Medical Technology; Medicare/Medicaid; Medicine; Methods; Modeling; Monitor; Neurobehavioral Manifestations; Neurosciences; Outcome; Patients; Pharmaceutical Preparations; Policies; Policy Maker; Policy Research; Population; Prevalence; Price; Privatization; Process; Quality of life; Reimbursement Mechanisms; Reporting; Research; Savings; Structure; System; Technology; Testing; Time; Variant; Work; alternative treatment; base; behavioral health; caregiving; clinical Diagnosis; clinical practice; cost; dementia care; design; drug candidate; economic outcome; effective intervention; effective therapy; functional status; health economics; informal caregiver; innovation; meetings; mortality risk; neuroimaging; novel; novel therapeutics; optimism; patient subsets; payment; physical conditioning; population health; social; success; tau Proteins; tau aggregation; tau-1; theories; trend

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.

项目总结/摘要 阿尔茨海默病和相关痴呆症（ADRD）的进展缓慢得令人沮丧，但有效 治疗方法可能就在眼前。考虑到ADRD的规模及其成本，即使是在延迟治疗方面的适度疗效， 进步可以带来巨大的社会效益。然而，ADRD问题的规模也提出了一个 融资挑战。我们如何为大量的ADRD患者和那些 症状前这个问题由于临床益处将随疾病阶段而变化的可能性而加剧， 基因型或其他未知因素。支付者不愿意为每剂治疗支付高价， 这些好处可能会在以后的生活中积累起来。我们将召集经济学专家， 神经科学和ADRD护理，以确定和量化替代报销策略，确保更好地 access.首先，使用兰德/加州大学洛杉矶分校的适当性方法的变体，我们将确定最有前途的 ADRD干预措施，量化其成功的可能性和预期的临床获益，并评估这些措施是否 受益可能因患者亚组而异。其次，我们将绘制临床试验终点图， 目前ADRD管道更广泛的生活质量结果-例如，功能状态、疾病发生率，以及 死亡风险-可用于估计社会后果。第三，借鉴经济理论， 现有文献，我们将开发新的ADRD治疗报销机制，并描述如何 他们将被组织起来。第四，我们将调整未来老年人模型（FEM），这是一个经过充分验证的微观模拟 模型，研究新的ADRD技术的长期健康和经济后果，并研究如何 这些后果会因定价机制不同而有所不同。我们将研究平均结果， ADRD患者在替代技术和定价方案方面的差异变化。第五，我们将使用 微观模拟模型，研究技术和定价方案对健康和经济的影响 ADRD患者的非正式照顾者的结局。最后，我们将举办一个研讨会， 调查结果提供给主要市场和政策利益相关者，并就潜在的后续步骤展开讨论。 总之，拟议的研究将制定和评估创新战略，以确保当前和未来 ADRD患者可以获得新的治疗方法。