GlaxoSmithKline R&D, Gunnels Wood Road, Stevenage, Hertfordshire, UK.
Stanford University, Stanford, California, USA.
J Glob Health. 2022 Jun 11;12:04045. doi: 10.7189/jogh.12.04045.
Partners from an NGO, academia, industry and government applied a tool originating in the private sector - Quantitative Decision Making (QDM) - to rigorously assess whether to invest in testing a global health intervention. The proposed NEWBORN study was designed to assess whether topical emollient therapy with sunflower seed oil in infants with very low birthweight <1500 g in Kenya would result in a significant reduction in neonatal mortality compared to standard of care.
The QDM process consisted of prior elicitation, modelling of prior distributions, and simulations to assess Probability of Success (PoS) via assurance calculations. Expert opinion was elicited on the probability that emollient therapy with sunflower seed oil will have any measurable benefit on neonatal mortality based on available evidence. The distribution of effect sizes was modelled and trial data simulated using Statistical Analysis System to obtain the overall assurance which represents the PoS for the planned study. A decision-making framework was then applied to characterise the ability of the study to meet pre-selected decision-making endpoints.
There was a 47% chance of a positive outcome (defined as a significant relative reduction in mortality of ≥15%), a 45% chance of a negative outcome (defined as a significant relative reduction in mortality <10%), and an 8% chance of ending in the consider zone (ie, a mortality reduction of 10 to <15%) for infants <1500 g.
QDM is a novel tool from industry which has utility for prioritisation of investments in global health, complementing existing tools [eg, Child Health and Nutrition Research Initiative]. Results from application of QDM to the NEWBORN study suggests that it has a high probability of producing clear results. Findings encourage future formation of public-private partnerships for health.
非政府组织、学术界、工业界和政府的合作伙伴应用了一种源自私营部门的工具——定量决策（QDM）——来严格评估是否投资于测试全球卫生干预措施。拟议的新生儿研究旨在评估在肯尼亚出生体重非常低（<1500 克）的婴儿中使用葵花籽油进行局部保湿治疗是否会与标准护理相比显著降低新生儿死亡率。
QDM 过程包括事先 elicitation、先验分布建模和模拟，以通过保证计算评估成功概率（PoS）。根据现有证据，专家们对葵花籽油保湿疗法是否会对新生儿死亡率产生任何可衡量的益处的可能性进行了评估。使用统计分析系统对效应大小的分布进行建模和模拟，以获得总体保证，这代表了计划研究的 PoS。然后应用决策框架来描述研究满足预先选择的决策终点的能力。
<1500 克的婴儿有 47%的可能性出现阳性结果（定义为死亡率显著相对降低≥15%），45%的可能性出现阴性结果（定义为死亡率显著相对降低<10%），8%的可能性出现考虑区（即死亡率降低 10%至<15%）。
QDM 是一种来自工业界的新颖工具，可用于优先投资全球卫生，补充现有的工具[例如，儿童健康与营养研究倡议]。将 QDM 应用于新生儿研究的结果表明，它有很高的可能性产生明确的结果。这些发现鼓励未来形成公私合作伙伴关系以促进健康。
