What is the Sakigake designation?

The Pharmaceuticals and Medical Devices Agency (PMDA) of Japan introduced the “Sakigake” system in 2015 to help accelerate the development and approval of innovative and novel drugs & medical devices. Under the pilot system, applicants looking to seek the Sakigake designation for their product were allowed a timeframe of 1 month per year to do so. However, the permanent system went into effect in 2020 after which sponsors could seek the designation any time of the year. Furthermore, it enabled sponsors to submit a Sakigake-designated new drug application (NDA) within 30 calendar days of submitting a Biologics License Application (BLA) or New Drug Application (NDA) for the same medicinal product with any other Agency. Interestingly, the term “Sakigake” means “pioneer” in the Japanese language.

The Sakigake Designation System

The Sakigake system provides sponsors with an accelerated approval pathway that not only includes an expedited review process but also priority consultation and pre-application scientific advice. The first step is for the medicinal product to satisfy the following conditions to consider the Sakigake designation:

1. The product should be developed in Japan and be anticipating an application approval (ideally having had PMDA consultation from the start of R&D stage)
2. Data that demonstrates prominent improvement in effectiveness compared to existing therapy (data on mechanism of action, non-clinical and early phase clinical studies).

The advantages associated with this designation include prioritized consultation here the sponsors only have to wait 1 month compared to 2 months which is the usual case. They also receive substantial pre-application consultation before application, and a prioritized review i.e., reduced timeline of 6 months compared to 12 months. Moreover, a PMDA manager is assigned who is responsible for taking overall management for the entire approval process including conformity assurance, quality management, safety measures, and review. Post-marketing safety measures are also strengthened. Sponsors must submit applications to the Evaluation and Licensing Division (ELD). Once the PMDA has done their review, they shall inform the sponsor within 60 days. The ELD is then to approach the sponsor who has submitted the application wherein the result is to be notified within 30 days after the submission.

What are the regulatory challenges associated with Sakigake designation?

The Sakigake system is associated with some regulatory challenges despite offering numerous benefits to sponsors. For example, the eligibility criteria for the designation are relatively stringent and focus on medical needs, innovative treatments, and a “Japan-first” application approach. Therefore, many sponsors may not be able to qualify their product and would have to look for alternative pathways to obtain accelerated approval.

Secondly, the more complex the treatment, the more difficult it is to meet the PMDA’s safety and efficacy standards within shortened timelines. Hence, sponsors may find that they face challenges in balancing the development speed with comprehensive data requirements which could affect the quality of evidence and may even increase the likelihood of post-market obligations.

Lastly, considering the unique regulatory framework of Japan, foreign companies are required to navigate localized requirements which often necessitate substantial adaptation of clinical data and compliance processes.

DDReg’s Capabilities

DDReg offers comprehensive regulatory support for companies seeking Sakigake designation and other accelerated pathways with Japan’s PMDA. With expertise in navigating Japan’s unique regulatory landscape, DDReg provides clients with support to move through the entire approval process.