Machine learning-based models for prediction of innovative medicine reimbursement decisions in Scotland.

Yitong Wang, Keith Tolley, Clément Francois, Mondher Toumi
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Abstract

Objective: This study aimed to investigate the critical factors for reimbursement decisions of innovative medicines in Scotland and to explore the feasibility of machine learning models for predicting decisions.

Method: All appraisals for innovative medicines issued by the Scottish Medicines Consortium (SMC) from 2016 to 2020 were screened to extract decision outcomes and 24 explanatory factors. SelectKBest with chi-square test was used for factor selection. The factors with P-value <0.05 were considered to have statistically significant associations with decision outcomes and were selected. Six machine learning models including decision tree, random forest, support-vector machine, Xgboost and K-nearest neighbours and logistic regression were used to build models with selected factors. Indicators comprising accuracy, precision, recall, F1-score were used to evaluate the performance of models.

Result: A total of 111 appraisals were identified, among which, 47 medicines were recommended, 48 recommended with restricted use and 16 not recommended. Seven were identified to be significant and selected for the prediction models. The factors of request for restriction on indication by manufacture, uncertainty of economic evidence, validation of primary outcomes and acceptance of comparator were identified as the most important predictors for SMC decisions. Four models had good prediction performance with both accuracy and F1-score over 0.9 in the internal validation, and random forest had the best prediction performance.

Conclusion: Low uncertainty of economic evidence, validated primary outcomes and accepted comparators were significantly associated with positive SMC decisions. Machine learning models may be feasible for predicting reimbursement decisions in the future.

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基于机器学习的苏格兰创新药品报销决策预测模型。
目的:本研究旨在调查苏格兰创新药物报销决策的关键因素,并探讨机器学习模型预测决策的可行性。方法:筛选2016 - 2020年苏格兰医药协会(SMC)发布的所有创新药评价,提取决策结果和24个解释因素。采用SelectKBest卡方检验进行因子选择。结果:共鉴定出111项评价,其中推荐用药47种,限制用药48种,不推荐用药16种。其中7个被认为是显著的,并被选择用于预测模型。要求限制生产适应症、经济证据的不确定性、主要结果的验证和比较物的接受度等因素被确定为SMC决策的最重要预测因素。在内部验证中,4个模型的预测精度和f1得分均在0.9以上,其中随机森林的预测效果最好。结论:经济证据的低不确定性,验证的主要结局和接受的比较物与积极的SMC决策显着相关。机器学习模型在预测未来的报销决策方面是可行的。
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