Department of Epidemiology, Erasmus University Medical Center, Rotterdam, The Netherlands.
Department of Radiology and Nuclear Medicine, Erasmus University Medical Center, Rotterdam, The Netherlands.
Med Decis Making. 2024 Jul;44(5):512-528. doi: 10.1177/0272989X241255047. Epub 2024 Jun 3.
The COVID-19 pandemic underscored the criticality and complexity of decision making for novel treatment approval and further research. Our study aims to assess potential decision-making methodologies, an evaluation vital for refining future public health crisis responses.
We compared 4 decision-making approaches to drug approval and research: the Food and Drug Administration's policy decisions, cumulative meta-analysis, a prospective value-of-information (VOI) approach (using information available at the time of decision), and a reference standard (retrospective VOI analysis using information available in hindsight). Possible decisions were to reject, accept, provide emergency use authorization, or allow access to new therapies only in research settings. We used monoclonal antibodies provided to hospitalized COVID-19 patients as a case study, examining the evidence from September 2020 to December 2021 and focusing on each method's capacity to optimize health outcomes and resource allocation.
Our findings indicate a notable discrepancy between policy decisions and the reference standard retrospective VOI approach with expected losses up to $269 billion USD, suggesting suboptimal resource use during the wait for emergency use authorization. Relying solely on cumulative meta-analysis for decision making results in the largest expected loss, while the policy approach showed a loss up to $16 billion and the prospective VOI approach presented the least loss (up to $2 billion).
Our research suggests that incorporating VOI analysis may be particularly useful for research prioritization and treatment implementation decisions during pandemics. While the prospective VOI approach was favored in this case study, further studies should validate the ideal decision-making method across various contexts. This study's findings not only enhance our understanding of decision-making strategies during a health crisis but also provide a potential framework for future pandemic responses.
This study reviews discrepancies between a reference standard (retrospective VOI, using hindsight information) and 3 conceivable real-time approaches to research-treatment decisions during a pandemic, suggesting suboptimal use of resources.Of all prospective decision-making approaches considered, VOI closely mirrored the reference standard, yielding the least expected value loss across our study timeline.This study illustrates the possible benefit of VOI results and the need for evidence accumulation accompanied by modeling in health technology assessment for emerging therapies.
新冠疫情凸显了新型治疗方法审批和进一步研究决策的关键性和复杂性。本研究旨在评估潜在的决策方法，这对于完善未来公共卫生危机应对至关重要。
我们比较了四种药物审批和研究决策方法：美国食品和药物管理局的政策决策、累积荟萃分析、前瞻性价值信息（VOI）方法（使用决策时可用的信息）和参考标准（使用事后可用信息的回顾性 VOI 分析）。可能的决策包括拒绝、接受、提供紧急使用授权或仅在研究环境中允许新疗法的使用。我们使用住院新冠患者使用的单克隆抗体作为案例研究，分析了 2020 年 9 月至 2021 年 12 月的证据，并重点关注每种方法优化健康结果和资源配置的能力。
我们的研究结果表明，政策决策与参考标准（回顾性 VOI 分析）之间存在显著差异，预期损失高达 2690 亿美元，这表明在等待紧急使用授权期间，资源使用不理想。仅依靠累积荟萃分析进行决策会导致最大的预期损失，而政策方法的损失高达 160 亿美元，前瞻性 VOI 方法的损失最小（高达 20 亿美元）。
我们的研究表明，在大流行期间，价值信息分析的纳入可能对研究优先级和治疗实施决策特别有用。虽然在本案例研究中倾向于前瞻性 VOI 方法，但需要进一步的研究来验证各种情况下的理想决策方法。这项研究的结果不仅增强了我们对健康危机期间决策策略的理解，还为未来的大流行应对提供了潜在框架。
本研究回顾了在大流行期间，参考标准（回顾性 VOI，使用事后信息）和 3 种可能的实时研究治疗决策方法之间的差异，表明资源利用不理想。在所考虑的所有前瞻性决策方法中，VOI 最接近参考标准，在我们的研究时间线上产生的预期价值损失最小。本研究说明了 VOI 结果的可能益处以及在新兴疗法的卫生技术评估中需要伴随证据积累和建模。
