A Qualitative Interview Study on Expanded Access Clinical Trials for Compassionate Use in Japan

Area of Science:

• Drug development and regulatory affairs
• Clinical trial management
• Patient access to novel therapeutics

Background:

• Expanded Access Clinical Trials (EACTs) provide crucial access to investigational drugs for life-threatening diseases.
• The EACT system in Japan was launched in 2016.
• Understanding pharmaceutical companies' perspectives on EACTs is vital for system optimization.

Purpose of the Study:

• To investigate the current state and perceptions of Expanded Access Clinical Trials (EACTs) in Japan.
• To gather insights from pharmaceutical companies on the practicalities and challenges of EACT implementation.
• To identify areas for improvement in the EACT system based on industry feedback.

Main Methods:

• Conducted semi-structured interviews with 10 pharmaceutical companies developing innovative drugs in Japan.
• Selected companies developing drugs for which EACTs might be relevant, regardless of prior EACT experience.
• Focused on understanding company views and experiences with the EACT system.

Main Results:

• All interviewed companies were aware of EACTs; 12 trials were conducted with a 2.5% implementation rate for pivotal trials.
• "Physician and medical institution requests" were the primary driver for EACT implementation (90.0%).
• Key barriers included "system applicability" (5 companies), with 80.0% suggesting improvements like financial aid (60.0%) and procedural simplification (60.0%).

Conclusions:

• Japanese pharmaceutical companies identify significant issues with the current EACT system.
• There is a strong consensus on the need for system revisions, including a single patient access system, enhanced support resources, and streamlined procedures.
• Eight years post-establishment, the EACT legislation in Japan appears due for revision to better facilitate patient access to critical treatments.