Global drug innovation in the East has significantly increased in recent years. China is one of the major players and has been committed to regulatory reform since 2015, with the establishment of a supportive legal framework, opening the market for innovative products. As a result, more global and local companies are launching novel drugs in China.
To date, one of the most noticeable regulatory policy changes by the National Medical Products Administration (NMPA) is the introduction of accelerated approval pathways. In 2020, the new version of the Provisions for Drug Registration has outlined four expedited procedures including Breakthrough Therapy, Conditional Approval, Priority Review, and Special Approval.
How should you use such a roadmap to plan the development of your product?
What are the criteria and benefits of each option?
In the first of this series, we will introduce the Breakthrough Therapy Designation (BTD) in China. The BTD program by NMPA is very similar to an accelerated pathway such as Breakthrough Therapy Designation by the FDA.
The BTD in China is designed for innovative drugs targeting the prevention and treatment of serious life-threatening diseases or diseases that seriously affect the quality of life, for which no effective prevention and treatment option is available.
When applying for BTD, the investigational drugs must demonstrate clinical superiority. Once granted, the sponsor will be eligible for the following benefits:
- Receive guidance from CDE (CENTER FOR DRUG EVALUATION, NMPA) via a dedicated interaction channel during the clinical trial. This would reduce the development risk and achieve better understanding of expectations from the regulatory agency.
- The investigational drug will also be eligible for priority review during the marketing authorization application, which could significantly shorten the project timeline.
Since 2020, more than 133 BTDs have been granted by NMPA, though the overall acceptance rate was revealed to be low (16.3% in 2020 and 20.3% in 2021) . By a pooled analysis , the BTD program is shown to significantly shorten the clinical development time and marketing authorization approval time, and further justify the value of BTD for the innovative products.
When a sponsor is considering BTD application in China, we recommend starting the planning early, it is advised to apply during the Phase I or Phase II studies, no later than the commencement of Phase III studies in general. For investigational products targeting oncology, it is critical to take into consideration the local medical consensus/treatment guidelines and the market landscape when determining the indication for BTD application.
 CDE evaluation report 2020 and 2021.
Published on: Jan 20, 2023