Highlights
- •Usage of real-world data (RWD) for clinical implementation is gaining popularity.
- •Use of SCRUM-Japan Registry for the phase II TRIUMPH study was investigated
- •We've paved way for RWD approval in Japan
- •Future steps for building regulatory-grade registries and using RWD must be defined
Abstract
Utilizing real-world data (RWD) for effective clinical implementation is becoming
more and more appealing as the cost of drug development rises, especially for patients
with rare diseases and rare molecular subtypes for whom conducting randomized controlled
trials is challenging. If a regulatory approval methodology based on RWD as an external
control group can be established, drug development for rarer fractions can be accelerated
by lowering costs and time, as well as reducing physical and emotional burdens on
both patients and healthcare professionals. Since 2017, we have been prospectively
collecting the clinical data of standard therapies in patients with rare molecular
fractions under the SCRUM-Japan Registry platform, which is a qualified registry utilized
as external control data for regulatory submission. Based on the results of the phase
II TRIUMPH study (UMIN000027887) and the extracted data from the SCRUM-Japan Registry,
the pharmaceutical company submitted an application for pertuzumab and trastuzumab
in patients with HER2-positive metastatic colorectal cancer in April 2021. Pertuzumab
and trastuzumab were approved as expanded indications on March 28, 2022, as 6 cases
out of 14 extracted from the SCRUM-Japan Registry were classified and utilized as
“evaluation material” under the review process of the Pharmaceuticals and Medical
Devices Agency (PMDA). Through the TRIUMPH study and the SCRUM-Japan Registry, we
have paved the way for regulatory approval of RWD in Japan. In future, we must define
the steps for constructing regulatory-grade registries and the method/process for
utilizing RWD by accumulating case experiences.
Keywords
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Article Info
Publication History
Published online: October 21, 2022
Accepted:
October 19,
2022
Received in revised form:
October 18,
2022
Received:
September 3,
2022
Publication stage
In Press Journal Pre-ProofIdentification
Copyright
© 2022 Elsevier Inc. All rights reserved.
