Health Intervention and Technology Assessment Program, Ministry of Public Health, Nonthaburi, Thailand.
Division of Neurology, Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.
Int J Technol Assess Health Care. 2023 Nov 17;40(1):e4. doi: 10.1017/S0266462323002714.
The purpose of this study is to evaluate the validity of the standard approach in expert judgment for evaluating precision medicines, in which experts are required to estimate outcomes as if they did not have access to diagnostic information, whereas in fact, they do.
Fourteen clinicians participated in an expert judgment task to estimate the cost and medical outcomes of the use of exome sequencing in pediatric patients with intractable epilepsy in Thailand. Experts were randomly assigned to either an "unblind" or "blind" group; the former was provided with the exome sequencing results for each patient case prior to the judgment task, whereas the latter was not provided with the exome sequencing results. Both groups were asked to estimate the outcomes for the counterfactual scenario, in which patients had not been tested by exome sequencing.
Our study did not show significant results, possibly due to the small sample size of both participants and case studies.
A comparison of the unblind and blind approach did not show conclusive evidence that there is a difference in outcomes. However, until further evidence suggests otherwise, we recommend the blind approach as preferable when using expert judgment to evaluate precision medicines because this approach is more representative of the counterfactual scenario than the unblind approach.
本研究旨在评估专家判断中评估精准医学的标准方法的有效性，即要求专家在没有获得诊断信息的情况下估计结果，而实际上他们已经获得了这些信息。
14 名临床医生参与了一项专家判断任务，以评估在泰国患有难治性癫痫的儿科患者中使用外显子组测序的成本和医疗结果。专家被随机分配到“不盲”或“盲”组；前者在判断任务之前提供了每位患者病例的外显子组测序结果，而后者则没有提供外显子组测序结果。两组都被要求估计反事实情景下的结果，即患者没有接受外显子组测序。
我们的研究没有显示出显著的结果，可能是由于参与者和病例研究的样本量都很小。
不盲法和盲法的比较没有确凿的证据表明结果存在差异。然而，在进一步的证据表明相反之前，我们建议在使用专家判断评估精准医学时采用盲法，因为这种方法比不盲法更能代表反事实情景。
