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(hào) R95 文獻(xiàn)標(biāo)志碼 A 文章編號(hào) 1001-0408（2022）02-0142-04

DOI 10.6039/j.issn.1001-0408.2022.02.03

摘 要 目的 為我國(guó)兒童藥品臨床綜合評(píng)價(jià)工作的全面開(kāi)展提供借鑒。方法 以兒童抗過(guò)敏藥物為例，從主題遴選、評(píng)價(jià)內(nèi)容與維度、評(píng)價(jià)指標(biāo)、評(píng)價(jià)方法及評(píng)價(jià)結(jié)果報(bào)告等環(huán)節(jié)探索醫(yī)療機(jī)構(gòu)開(kāi)展兒童藥品臨床綜合評(píng)價(jià)的方法。結(jié)果與結(jié)論 在開(kāi)展兒童藥品臨床綜合評(píng)價(jià)時(shí)，可在國(guó)家臨床綜合評(píng)價(jià)相關(guān)指南指導(dǎo)下，按照重要性、相關(guān)性以及可評(píng)估性三大原則遴選評(píng)價(jià)主題，然后圍繞安全性、有效性、經(jīng)濟(jì)性、適宜性、可及性、創(chuàng)新性6個(gè)維度，開(kāi)發(fā)建立適宜的評(píng)價(jià)指標(biāo)體系，對(duì)待評(píng)藥品進(jìn)行定性及定量數(shù)據(jù)整合分析。在評(píng)價(jià)中，應(yīng)圍繞兒童臨床基本用藥實(shí)踐及決策需求，規(guī)范、科學(xué)、合理地定義不同維度證據(jù)所需的核心指標(biāo)集和標(biāo)準(zhǔn)數(shù)據(jù)集，規(guī)范收集和使用真實(shí)世界數(shù)據(jù)，并結(jié)合其他類別證據(jù)，真正發(fā)揮其在我國(guó)兒童藥品臨床綜合評(píng)價(jià)中的優(yōu)勢(shì)作用。

關(guān)鍵詞 兒童藥品;抗過(guò)敏藥物;臨床綜合評(píng)價(jià)

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(jīng)濟(jì)發(fā)展和生活環(huán)境的改變，兒童過(guò)敏性疾病[包括變應(yīng)性鼻炎（allergic rhinitis，AR）、哮喘、過(guò)敏性結(jié)膜炎、濕疹、食物過(guò)敏等]的發(fā)病率在全球范圍內(nèi)呈現(xiàn)增長(zhǎng)趨勢(shì)[1-2]。過(guò)敏性疾病已極大地影響了患兒及其家庭成員的生活質(zhì)量，嚴(yán)重全身過(guò)敏反應(yīng)甚至可能危及患兒生命。受限于兒童臨床藥理學(xué)發(fā)展的不充分與真實(shí)世界研究證據(jù)的缺失，部分已上市的抗過(guò)敏藥物尚無(wú)兒童適宜劑型和規(guī)格，或缺乏應(yīng)用于患兒的科學(xué)證據(jù)，或存在藥品說(shuō)明書(shū)不規(guī)范、無(wú)法指導(dǎo)患兒用藥，或存在濫用、超適應(yīng)證用藥等諸多問(wèn)題。