世界卫生组织发布结核病长效注射剂目标方案概况
正文 · 中文译文
世界卫生组织（WHO）发布了用于结核病预防和治疗的长效注射剂目标方案特征概述。结核病（TB）的预防和治疗目前仍依赖于需持续数月每日口服的方案，而对许多人来说，在如此长的时期内坚持服药十分困难。长效注射剂（LAIs）可显著减轻服药者及提供相关服务的卫生系统的负担。长效注射剂已经改变了其他疾病（最为显著的是HIV）的管理方式，目前已有几种用于结核病的长效注射剂候选药物正在进入或即将进入临床开发阶段。 “长效注射剂能让结核病的预防和治疗对需要的人来说变得简单得多。这些特征概述在早期就向研发者阐明，一个新方案必须达到哪些要求才能在实践中有意义，同时不牺牲疗效、安全性或患者的选择权。”世界卫生组织HIV、结核病、肝炎和性传播感染部门主任Tereza Kasaeva博士表示。 目标方案特征概述（TRPs）在开发早期就明确了用于结核病预防治疗或结核病治疗的新型长效注射剂方案必须达到哪些要求才能在实践中有用。这些概述面向制药行业、学术界、研究机构、产品开发合作伙伴、受结核病影响的社区、民间社会组织及捐助方。针对每一特征，概述都定义了最低要求——产品必须满足该要求才值得继续推进，以及最优要求——最理想且现实可及的目标。 结核病预防治疗的特性目标是，在单次就诊时通过单次注射完成全疗程。结核病治疗的特性目标是，疗程不超过四个月，给药次数不超过三次。两者均要求由训练有素的卫生工作者在卫生系统最低可行层级进行注射，注射量小，无需冷链，且疗效和安全性至少不低于现有口服方案。本文件还阐述了各特性之间的权衡，并涉及试验设计、总体治疗负担、可扩展性、公平可及性、价格透明和成本问题。 世界卫生组织召集了一个科学目标产品特征开发小组来制定这些特性。该小组包括试验研究者、药理学家、监管人员、国家结核病规划官员、有结核病经历者以及民间社会代表。他们的工作基于景观分析、2026年4月至5月的公开咨询，以及社区和提供者调查、焦点小组讨论和一项多国离散选择实验。 针对偏好不接受注射的人群，应并行推进更优口服方案的工作，实施过程中应保留患者的选择权。
正文 · 英文原文
The World Health Organization (WHO) has published Target regimen profiles for long-acting injectables for tuberculosis prevention and treatment. Tuberculosis (TB) prevention and treatment still depend on daily oral regimens taken over months, and sustaining adherence over such periods is difficult for many people. Long-acting injectables (LAIs) could significantly lighten the burden for people taking TB medicines and the health system that delivers them. They have already changed the management of other conditions, most visibly HIV, and several LAI candidates for TB are now entering or approaching clinical development. “Long-acting injectables could make TB prevention and treatment much simpler for the people who need them. These profiles tell developers, early, what a new regimen must achieve to make that difference in practice, without trading away efficacy, safety or patient choice,” said Dr Tereza Kasaeva, Director of the WHO Department for HIV, Tuberculosis, Hepatitis and Sexually Transmitted Infections. The target regimen profiles (TRPs) set out, early in development, what a new LAI regimen for TB preventive treatment or for the treatment of TB disease would have to achieve to be useful in practice. They are intended for the pharmaceutical industry, academia, research institutions, product development partners, TB-affected communities, civil society organizations and donors. For each characteristic they define a minimal requirement, which a product must meet to be worth pursuing, and an optimal requirement, the most desirable yet realistically achievable target. The profile for TB preventive treatment aims at a full course given as a single injection at one encounter. The profile for TB disease aims at a regimen of no more than four months, given in three or fewer dosing encounters. Both call for injections given by a trained health worker at the lowest feasible level of the health system, small injection volumes, no cold chain, and efficacy and safety at least as good as those of current oral regimens. The document also sets out the trade-offs between characteristics, and addresses trial design, overall treatment burden, scalability, equitable access, transparent pricing and cost. A Scientific TRP Development Group was convened by WHO to develop these profiles. The Group included trialists, pharmacologists, regulators, national TB programme officers, people with lived experience of TB and civil society representatives. They were informed by a landscape analysis, a public consultation in April and May 2026, and community and provider surveys, focus group discussions and a multicountry discrete choice experiment. Work on better oral regimens should continue in parallel, for people who would prefer not to receive injections, and implementation should preserve patient choice.