儿童药品临床综合评价方法研究

刘璐 肖月 刘畅 曲素欣 李蓉 于宝东 莫晓媚 刘克军 闫美兴

中图分类号 R95 文献标志码 A 文章编号 1001-0408（2022）02-0142-04

DOI 10.6039/j.issn.1001-0408.2022.02.03

摘 要 目的 为我国儿童药品临床综合评价工作的全面开展提供借鉴。方法 以儿童抗过敏药物为例，从主题遴选、评价内容与维度、评价指标、评价方法及评价结果报告等环节探索医疗机构开展儿童药品临床综合评价的方法。结果与结论 在开展儿童药品临床综合评价时，可在国家临床综合评价相关指南指导下，按照重要性、相关性以及可评估性三大原则遴选评价主题，然后围绕安全性、有效性、经济性、适宜性、可及性、创新性6个维度，开发建立适宜的评价指标体系，对待评药品进行定性及定量数据整合分析。在评价中，应围绕儿童临床基本用药实践及决策需求，规范、科学、合理地定义不同维度证据所需的核心指标集和标准数据集，规范收集和使用真实世界数据，并结合其他类别证据，真正发挥其在我国儿童药品临床综合评价中的优势作用。

关键词 儿童药品;抗过敏药物;临床综合评价

Research about clinical comprehensive evaluation methods of pediatric drugs：taking pediatric anti-allergic drugs as an example

LIU Lu1，XIAO Yue2，LIU Chang1，QU Suxin1，LI Rong1，YU Baodong1，MO Xiaomei1，LIU Kejun2，YAN Meixing1（1. Dept. of Pharmacy， Qingdao Women and Childrens Hospital， Shandong Qingdao 266034， China; 2. China National Health Development Research Center， Beijing 100044， China）

ABSTRACT   OBJECTIVE To provide reference for clinical comprehensive evaluation of pediatric drugs in China. METHODS Taking pediatric anti-allergic drugs as an example， the clinical comprehensive evaluation methods of pediatric drugs in medical institutions were explored from the aspects of theme selection， evaluation content and dimension， evaluation index， evaluation method and evaluation result report. RESULTS & CONCLUSIONS During the clinical comprehensive evaluation of pediatric drugs， under the guidance of relevant national guidelines for clinical comprehensive evaluation， the evaluation topics could be selected according to the three principles of importance， relevance and evaluability， and then an appropriate evaluation index system could be developed around the six dimensions of safety， effectiveness， economy， suitability，accessibility and innovativeness; qualitative and quantitative data integration analysis of the drugs to be evaluated were performed. In the evaluation， it is necessary to focus on childrens clinical basic drug use practice and decision-making needs， normatively， scientifically and reasonably define the core index set and standard data set required by different dimensions of evidence， standardize the collection and use of real-world data， and effectively combine other types of evidence to truly play its advantageous role in the clinical comprehensive evaluation of pediatric drugs in China.

KEYWORDS   pediatric drug; anti-allergic drug; clinical comprehensive evaluation

隨着经济发展和生活环境的改变，儿童过敏性疾病[包括变应性鼻炎（allergic rhinitis，AR）、哮喘、过敏性结膜炎、湿疹、食物过敏等]的发病率在全球范围内呈现增长趋势[1-2]。过敏性疾病已极大地影响了患儿及其家庭成员的生活质量，严重全身过敏反应甚至可能危及患儿生命。受限于儿童临床药理学发展的不充分与真实世界研究证据的缺失，部分已上市的抗过敏药物尚无儿童适宜剂型和规格，或缺乏应用于患儿的科学证据，或存在药品说明书不规范、无法指导患儿用药，或存在滥用、超适应证用药等诸多问题。