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：
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
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/9789172
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.

项目概要/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，但很有效考虑到 ADRD 的规模及其成本，治疗方法可能很快就会出现。可能会产生巨大的社会效益。然而，ADRD 问题的规模也呈现出一个严重的问题。融资挑战。我们如何为大量 ADRD 患者和那些患有 ADRD 的患者提供新的治疗费用？临床获益随疾病阶段而变化的可能性会加剧这个问题，基因型或其他未知因素，付款人不愿意为每剂治疗支付高价，尤其是。当这些好处可能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报销策略，以确保更好首先，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方法。 ADRD 干预措施，量化其成功的可能性和预期的临床效益，并评估这些干预措施是否有效其次，我们将绘制临床试验中使用的终点。当前的 ADRD 管道可实现更广泛的生活质量结果，例如功能状态、疾病发生率和死亡风险——可用于估计社会后果。第三，利用经济理论和经济理论。现有文献中，我们将为 ADRD 治疗开发新的报销机制，并描述如何第四，我们将采用未来老年人模型（FEM），这是一种经过充分验证的微观模拟。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因不同的定价机制而异。我们将研究平均结果和结果。 ADRD 患者对替代技术和定价方案的差异变化第五，我们将使用。研究技术和定价方案对健康和经济影响的微观模拟模型最后，我们将召开一个研讨会来宣传我们的成果。向主要市场和政策利益相关者提供调查结果，并就潜在的后续步骤进行讨论。拟议的研究将共同制定和评估创新战略，以确保当前和未来 ADRD 患者可以获得新疗法。