美国生物药立法指南实施进展与绩效评价研究

The performance of BsUFA along with developing laws and regulations of biosimilar biologic products in the United States

美国政府对医疗保健的支出总费用逐年攀升且居高不下。美国市场畅销药中生物药占比越来越高,大部分畅销的生物药价格每年均在上涨,最高年增长率为36%。这些生物药的美国专利多在2016—2020年到期,且均已超过数据独占期。《生物药价格竞争和创新法案》开启了美国生物类似药研发政策大门,BsUFA I绩效达成,完成了生物类似药批准从0到1的跨越。截止2017年FDA已批准生物类似药9个,无重大修订或重新提交的情况下,审核时限均未超过10个月,完全符合已制定BsUFA I绩效目标。本文对美国生物类似药研发的背景、法规指南的制定以及生物类似药用户付费法案(Biosimilar User Fee Act,BsUFA)的执行等进行了综述,以期对我国生物类似药发展提供一定借鉴和参考。

The health care spending paid by the United States has been rising and staying in the high level for several years.The top selling drugs in the US include more biological products than before,and most top selling biologicals created higher and higher prices,with the highest annual growth rate of 36% for some biological products.Most of the biologicals mentioned above will lose patent protection by 2016-2020,and be not appropriate for data exclusivity.Biologics Price Competition and Innovation Act is a reform act creating biosimilar pathway and followed by a series of regulations and guidelines.BsUFA I has met its performance goal,and the first biosimilar approved by FDA achieved zero breakthrough.By 2017,FDA has approved nine biosimilars,and the review time completely meets BsUFA I performance goal,except the products required for major revision or resubmission,not more than 10 months.Trough sorting out and thinking of the background,regulations and guidelines for biosimilars, as well as BsUFA implementation,this review is anticipated to provide valuable experience and reference for our biosimilar development.