RAPID: Shared decision models to guide drug allocation during a pediatric chemotherapy shortage

RAPID：共享决策模型在儿科化疗短缺期间指导药物分配

• 批准号: 2010487
• 负责人: Jessica Malpass
• 金额: $ 13.35万
• 依托单位: University of Virginia Main Campus
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-01-15 至 2021-12-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=2010487&HistoricalAwards=false
• 关键词: RAPID; Shared; decision; models; guide

Drug shortages require decisions on the allocation of essential drugs that are in limited supply. Vincristine is a chemotherapeutic drug that is central to many treatment regimens involving the most common pediatric cancers. Currently, we are experiencing a vincristine shortage in the United States expected to last through 2020 and there is no drug substitution that has equal efficacy or mode of action. Children have already been impacted by the current vincristine shortage and it is expected that cancer drug shortages will continue to increase at an alarming rate given the lack of profitability and fragility of the supply chain. Clinicians experience uncertainty in how best to allocate drugs in limited supply, and communicate status updates across institutions. Additionally, it is difficult to assess the health and economic consequences of various shortage scenarios. This study fills an important scientific gap in understanding how decisions regarding drug shortages are made in real time. Modeling and simulating shared decision models have implications for not only the current vincristine drug shortage, but future drug shortages at large, including those represented by pandemic situations. Modeling the health and economic impacts may also have policy implications for regulations supporting the supply chain requirements of critical drugs without substitutions. This project advances the knowledge of assessments of risk and trade-offs in the context of a drug shortage. Additionally, this project identifies information critical to drug shortage communication among clinicians, hospital systems, patients and families impacted by the shortage, drug manufacturers, regulators, and society at large. Tis project advances the development of scientific methods necessary to promote national health and welfare.By testing shared decision models necessary to guide drug allocation, the investigators will develop a decision framework that can also be applied in practice. Specifically, this project uses a mixed-modal approach of immersive qualitative methods followed by decision analytic modeling and Monte Carlo simulation to (1) evaluate current decision-making processes for drug allocation given the vincristine pediatric chemotherapy shortage and (2) use stakeholder-guided decisions and real-world data to model and simulate the clinical and economic outcomes of various drug allocation shortage scenarios. While the lens of this award is focused on the current vincristine shortage, the methods and principles of this work are novel in terms of understanding how decision making and risk communication processes are occurring in the context of uncertainty and lack of alternative treatment strategies. This methodological approach is also used to develop best practices for future decision analysis of drug shortages.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.

药品短缺需要就供应有限的基本药品的分配做出决定。长春新碱是一种化疗药物，是涉及最常见儿科癌症的许多治疗方案的核心。目前，美国正面临长春新碱短缺，预计将持续到 2020 年，并且没有具有同等功效或作用方式的药物替代品。 儿童已经受到当前长春新碱短缺的影响，鉴于缺乏盈利能力和供应链脆弱，预计抗癌药物短缺将继续以惊人的速度增加。临床医生在如何最好地分配有限供应的药物以及跨机构传达状态更新方面遇到不确定性。此外，很难评估各种短缺情况对健康和经济的影响。这项研究填补了理解如何实时做出有关药物短缺决策的重要科学空白。建模和模拟共享决策模型不仅对当前的长春新碱药物短缺有影响，而且对未来整个药物短缺（包括大流行情况下的药物短缺）都有影响。对健康和经济影响进行建模还可能对支持无替代关键药物供应链要求的法规产生政策影响。该项目增进了药物短缺情况下风险评估和权衡的知识。此外，该项目还确定了对临床医生、医院系统、受短缺影响的患者和家庭、药品制造商、监管机构和整个社会之间的药品短缺沟通至关重要的信息。该项目推动了促进国民健康和福利所需的科学方法的发展。通过测试指导药物分配所需的共享决策模型，研究人员将开发一个也可应用于实践的决策框架。具体来说，该项目使用沉浸式定性方法的混合模式方法，然后进行决策分析建模和蒙特卡罗模拟，以（1）评估当前长春新碱儿科化疗短缺的药物分配决策过程，以及（2）使用利益相关者指导决策和真实世界数据来建模和模拟各种药物分配短缺场景的临床和经济结果。虽然该奖项的重点是当前的长春新碱短缺问题，但这项工作的方法和原则在理解不确定性和缺乏替代治疗策略的背景下如何进行决策和风险沟通过程方面是新颖的。这种方法还用于开发未来药品短缺决策分析的最佳实践。该奖项反映了 NSF 的法定使命，并通过使用基金会的智力价值和更广泛的影响审查标准进行评估，被认为值得支持。