Case Study
How to Catch Up Global Clinical
Development:
Strategy Example
Strategy: After PoC completed in global, Japan development program starts.
The aim is to catch up and join global pivotal study to include Japanese patients
and to file JNDA at the same time with global NDA
Case Study A: Completed
Japan
Ph I in 1 Year
Background
- Company A was starting enrollment of their pivotal ph II oncology study
- Wanted to include Japan in the pivotal study
- However, the pivotal Recommend Dose (RD) hadn’t been confirmed safe in Japanese population which is a requirement before Japan can join.
Solution
- JCRO and clinical site had a first meeting with Company A and agreed to conduct the following study in Japan:
- Design: Ph I, open label, dose-escalation (low, RD and high dose) to evaluate safety and PK
- Patients: 9 (3x3) Japanese Subjects with solid tumors
- Sites: 1
- Services: JCRO managed everything including PMDA consultation with PV and DM/BIOS sub-contracted.
Outcome
- Clinical study completed in 9 months from first meeting
- CTN submitted 2 months after contract
- FPI was achieved 2 months after CTN
- Clinical outcome favorable:
- Compound well tolerated at all dose levels and PK results from RD consistent with global data
- JCRO managed Ph II part in collaboration with local CRO.
Caught up with global recruitment becoming 3rd highest recruiting country out of 12
Case Study B: PMDA
Consultation
in 3 Months
Background
- Company B was starting enrollment of their pivotal Ph II study with their compound targeting a rare gene cancer mutation
- Wanted to include Japan in the pivotal study
- However, the pivotal Recommend Dose (RD) hadn’t been confirmed safe in Japanese population which is a requirement before Japan can join.
Solution
- JCRO had a meeting with Company B and agreed to conduct the following study in Japan
- Design: Combined Ph I/II protocol, open label, RD (1 dose only) to evaluate safety, efficacy and PK
- Services: JCRO managed everything including PMDA consultation. DM, BIOS, MW and global PV managed by Sponsor.
Outcome
- JCRO conducted PMDA Formal Consultation 3 months after contract
- 1 dose cohort in Ph I was agreed with PMDA
- PMDA agreed to Ph I/II design and J-NDA strategy
- 1st J-CTN was submitted 2 months after PMDA Formal Consultation. It was approved after discussions and negotiations around DLT definitions together with PMDA