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

基本信息

批准号：
9977782
负责人：
DANA P GOLDMAN
金额：
$ 54.69万
依托单位：
UNIVERSITY OF SOUTHERN CALIFORNIA
依托单位国家：
美国
项目类别：
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/9977782
关键词：
Academy Address Affect Aging Alzheimer&apos s Disease Alzheimer&apos s disease model Alzheimer&apos 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

项目摘要

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.

项目摘要/摘要反对阿尔茨海默氏病和相关痴呆症（ADRD）的进展非常缓慢，但有效治疗可能会出现。考虑到ADRD的规模及其成本，甚至延迟的效率适中进步可能具有重大的社会利益。但是，ADRD问题的规模也提出了融资挑战。我们如何为大量ADRD患者和那些人负担新的疗法症状前？因疾病阶段而变化的可能性加剧了这个问题，基因型或其他未知因素。付款人不愿支付每剂量高价的疗法，尤其是当这些好处可能会在以后的生活中积累时。我们将汇集经济学专家，神经科学和ADRD关心确定和量化替代性报销策略，以确保更好使用权。首先，使用Rand/UCLA适当方法的变体，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益处，并评估是否在患者亚组中，好处可能会有所不同。其次，我们将绘制用于临床试验端点当前的ADRD管道，以实现更广泛的生活质量结果 - 例如功能状况，疾病事件和死亡风险 - 可用于估计社会后果。第三，借鉴经济理论和出口文献，我们将开发新颖的报销机制，用于ADRD疗法，并表征如何他们将是结构化的。第四，我们将适应未来的老年模型（FEM），这是一个验证的微观仿真模型，研究新Adrd技术的长期健康和经济后果，并研究如何这些后果会随不同的定价机制而变化。我们将研究平均结果和替代技术和定价方案的ADRD患者之间差异的变化。第五，我们将使用研究技术和定价方案对健康和经济的影响的微观仿真模型 ADRD患者的非正式护理人员的结果。最后，我们将召集一个研讨会来传播我们的对主要市场和政策利益相关者的发现，并为潜在的下一步进行讨论。拟议的研究将共同制定和评估创新策略，以确保当前和未来 ADRD患者可以使用新的疗法。