BLOG

Japan Advances Regulatory Reforms on RWD Utilization, Conditional Approval, and Pediatric Drug Development

2026.01.21

• Conditinal Approval
• Drug Lag
• MHLW
• Pediatric Drug Development
• Pharmaceutical Industry
• Real-World Data

Introduction to Regulatory Revisions

According to the notification issued by Japan’s Ministry of Health, Labour and Welfare (MHLW) on December 26, 2025 (001627808.pdf), Japan has introduced key regulatory provisions under the amended Pharmaceuticals and Medical Devices Act to modernize drug development and approval pathways.

The revisions focus on three strategic areas:

• expanded use of real-world data (RWD) in regulatory submissions,
• reform of the conditional approval system for drugs targeting rare and serious diseases, and
• measures to promote pediatric drug development to address Japan’s ongoing “drug-loss” challenge.

Utilization of Real-World Data in Regulatory Applications

The revised Act explicitly establishes a legal basis for the use of real-world data (RWD) in marketing authorization applications and post-approval evaluations.
Until now, RWD had been used on a case-by-case basis, but the amendment clarifies that RWD can formally support regulatory decision-making, including:

• Supplementing clinical trial data where conventional trials are difficult to conduct
• Supporting efficacy and safety evaluation for rare diseases or limited patient populations
• Enhancing post-marketing safety surveillance and lifecycle management

By clarifying RWD’s regulatory role, Japan aims to increase predictability for sponsors and encourage innovative development approaches aligned with global regulatory trends.

Reform of the Conditional Approval System for Rare and Serious Diseases

Japan has revised its conditional approval framework for drugs targeting rare and life-threatening diseases, where conventional confirmatory trials may be difficult or time-consuming.

Key changes include:

• A shift from requiring “clear efficacy demonstrated in exploratory trials” to “reasonable predictability of clinical usefulness”
• Greater flexibility in the type and amount of data accepted at the time of approval
• Reinforced expectations for post-approval data collection and follow-up

These revisions are intended to lower regulatory hurdles at the approval stage, while maintaining safety through post-marketing obligations, thereby accelerating patient access to innovative therapies.

Promoting Pediatric Drug Development to Address Drug Loss

To address persistent drug loss in pediatric medicines, the amendment introduces measures to encourage early, structured pediatric development planning.

Key elements include:

• Stronger regulatory encouragement for companies to prepare pediatric development plans when seeking approval for adult indications
• Incentives linked to pediatric development, including extended re-examination periods
  • Standard drugs: extension from 8 to 10 years
  • Orphan drugs: extension from 10 to 12 years

These measures aim to reduce delays in pediatric access to medicines already approved overseas and to align Japan more closely with global pediatric development strategies.

Conclusion

The revised Pharmaceuticals and Medical Devices Act reflects Japan’s strategic shift toward greater regulatory flexibility and innovation. By formalizing RWD use, reforming conditional approval for rare and serious diseases, and strengthening incentives for pediatric drug development, Japan seeks to reduce drug-loss, accelerate patient access, and enhance its attractiveness as part of global development programs.