Department of Pediatrics, Dalhousie University, 5850/5980 University Ave, Halifax, Nova Scotia B3K 6R8, Canada; Canadian Center for Vaccinology, Dalhousie University, 5850/5980 University Ave, IWK Health Centre, Nova Scotia Health Authority, Halifax, NS, Canada.
Canadian Center for Vaccinology, Dalhousie University, 5850/5980 University Ave, IWK Health Centre, Nova Scotia Health Authority, Halifax, NS, Canada; World Health Organization, Avenue Appia 20, Geneva, Switzerland.
Vaccine. 2020 Jan 29;38(5):1089-1095. doi: 10.1016/j.vaccine.2019.11.033. Epub 2019 Nov 27.
National Regulatory Authorities approve the indications for vaccine use in the product information. Occasionally, National Immunization Technical Advisory Groups (NITAGs) make off-label recommendations for use in different age groups, populations, and dosing schedules from the product information. We sought to determine the rationale, policies and procedures for NITAG off-label recommendations.
We conducted an environmental scan of Global NITAG Network members, immunization program managers and regulators in 38 high-, middle- and low-income countries. Participants completed an online survey regarding policies, procedures, and legislation governing development of off-label recommendations. A sub-sample of respondents met for a focus group and interviews which were analyzed qualitatively.
Thirty-four people responded from 26/38 (68%) countries surveyed; 76% of respondents were NITAG members or immunization program managers. Recommendations for off-label vaccine use were made in 14/26 (54%) countries; the NITAG made those recommendations in 8/14 (57%) countries. Reasons for off-label vaccine recommendations included response to disease outbreaks or vaccine shortages. Only one country had standard operating procedures for developing off-label recommendations while 6/14 (43%) countries had policies for implementing off-label recommendations. Nine respondents from 8 countries agreed to participate in a focus group (n = 6) or individual interviews (n = 3). Barriers to off-label recommendations included legal concerns, lack of standard definition for off-label use, and manufacturer reluctance to update product information. Facilitators included confidence in the decision-making process, and transparency of open communication among stakeholders.
Best practice guidelines are needed that define off-label use and outline a transparent, evidence-based approach to develop off-label recommendations.
国家监管机构根据产品信息批准疫苗的使用适应症。偶尔，国家免疫技术咨询小组（NITAGs）会根据产品信息之外的不同年龄组、人群和剂量方案提出非标签使用建议。我们旨在确定 NITAG 非标签建议的基本原理、政策和程序。
我们对全球 NITAG 网络成员、免疫规划管理人员和 38 个高、中、低收入国家的监管机构进行了环境扫描。参与者完成了一份关于制定非标签建议的政策、程序和法规的在线调查。部分受访者参加了焦点小组和访谈，对其进行了定性分析。
从调查的 38 个国家中的 26 个国家（68%）有 34 人做出回应；76%的受访者是 NITAG 成员或免疫规划管理人员。14/26（54%）个国家提出了非标签疫苗使用建议；NITAG 在 8/14（57%）个国家提出了这些建议。提出非标签疫苗建议的原因包括应对疾病爆发或疫苗短缺。只有一个国家有制定非标签建议的标准操作程序，而 6/14（43%）个国家有执行非标签建议的政策。来自 8 个国家的 9 名受访者同意参加焦点小组（n=6）或个人访谈（n=3）。非标签建议的障碍包括法律问题、缺乏非标签使用的标准定义以及制造商不愿更新产品信息。促进因素包括对决策过程的信心以及利益相关者之间透明的公开沟通。
需要制定最佳实践指南，定义非标签使用并概述一种透明的、基于证据的方法来制定非标签建议。
