中美突破性治疗药物制度实施效果的比较研究

A comparative study of the implementation effects of breakthrough therapeutic drug systems in China and the United States

摘要

目的: 借鉴美国突破性治疗药物(breakthrough therapy designation,BTD)制度的有益经验,为完善我国BTD制度提供建议和参考。方法: 通过对中美BTD制度的实施效果进行比较分析,揭示两国BTD制度的实施效果与差距。结果与结论: 中美BTD认定申请数量总体均呈增长趋势,但我国BTD认定率和认定获批率均显著低于美国,这可能与申请质量参差不齐和政策细化程度不足有关。我国BTD药物上市前临床开发时间较短,但审评时间较长,反映了两国药品监管机构在临床试验指导力度上的差异。在抗肿瘤药物临床试验设计方面,我国更倾向于单臂、非随机设计,以客观缓解率为主要终点;而美国试验设计则更为丰富与多元。此外,尚未有BTD中药获批上市。建议我国药品监管机构借鉴美国经验,制定BTD审评管理规范,加强审评人员的主导作用,设立跨学科团队,加强临床试验研制指导,提高研发及审评效率;同时,加强BTD认定药物叠加附条件批准上市后的监管,并推动BTD中药的申报与审批。医药企业应加强研发团队BTD法规和技能培训,提高BTD药物申请的质量,促进产品加快上市以满足患者临床需求。

Abstract

Objective: To draw on the useful experience of the breakthrough therapy designation (BTD) system in the United States (US) to provide suggestions and references for improving the BTD system in China. Methods: Through a comparative analysis of the implementation effects of the BTD systems in China and the US, the implementation effects and gaps of the BTD systems in the two countries were revealed. Results & Conclusion: The numbers of applications for BTD recognition in China and the US were generally on the rise, but the rates of BTD recognition and approval in China were significantly lower than those in the US, which may be related to the uneven quality of the applications and the lack of policy refinement. China's BTD drugs had a shorter pre-market clinical development time, but a longer review time, reflecting the difference in the strength of clinical trial guidance between the two countries' drug regulatory agencies. In terms of clinical trial design for antitumour drugs, China preferred single-arm, non-randomised design with objective remission rate as the primary endpoint; whereas the trial designs in the US were richer and more diverse. In addition, no BTD Chinese medicine has been approved for marketing. It is recommended that China's drug regulatory agencies learn from the US experience, formulate BTD review and management standards, strengthen the leading role of reviewers, set up interdisciplinary teams, strengthen the guidance of clinical trial development and improve the efficiency of research and development and review; at the same time, strengthen the supervision of BTD-recognised medicines after superimposed conditional approval for listing, and promote the declaration and approval of BTD traditional Chinese medicines. Pharmaceutical companies should strengthen the training of R&D teams on BTD regulations and skills, improve the quality of BTD drug applications, and promote the accelerated marketing of products to meet patients' clinical needs.

关键词

突破性治疗药物 / 药品注册 / 实施效果

Key words

breakthrough therapy designation / drug registration / implementation effect