兒童藥品臨床綜合評價方法研究

劉璐 肖月 劉暢 曲素欣 李蓉 于寶東 莫曉媚 劉克軍 閆美興

中圖分類號 R95 文獻標志碼 A 文章編號 1001-0408（2022）02-0142-04

DOI 10.6039/j.issn.1001-0408.2022.02.03

摘 要 目的 為我國兒童藥品臨床綜合評價工作的全面開展提供借鑒。方法 以兒童抗過敏藥物為例，從主題遴選、評價內容與維度、評價指標、評價方法及評價結果報告等環節探索醫療機構開展兒童藥品臨床綜合評價的方法。結果與結論 在開展兒童藥品臨床綜合評價時，可在國家臨床綜合評價相關指南指導下，按照重要性、相關性以及可評估性三大原則遴選評價主題，然后圍繞安全性、有效性、經濟性、適宜性、可及性、創新性6個維度，開發建立適宜的評價指標體系，對待評藥品進行定性及定量數據整合分析。在評價中，應圍繞兒童臨床基本用藥實踐及決策需求，規范、科學、合理地定義不同維度證據所需的核心指標集和標準數據集，規范收集和使用真實世界數據，并結合其他類別證據，真正發揮其在我國兒童藥品臨床綜合評價中的優勢作用。

關鍵詞 兒童藥品;抗過敏藥物;臨床綜合評價

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

隨著經濟發展和生活環境的改變，兒童過敏性疾病[包括變應性鼻炎（allergic rhinitis，AR）、哮喘、過敏性結膜炎、濕疹、食物過敏等]的發病率在全球范圍內呈現增長趨勢[1-2]。過敏性疾病已極大地影響了患兒及其家庭成員的生活質量，嚴重全身過敏反應甚至可能危及患兒生命。受限于兒童臨床藥理學發展的不充分與真實世界研究證據的缺失，部分已上市的抗過敏藥物尚無兒童適宜劑型和規格，或缺乏應用于患兒的科學證據，或存在藥品說明書不規范、無法指導患兒用藥，或存在濫用、超適應證用藥等諸多問題。