Local Regulatory Experts
Connect with regulatory affairs consultancies specializing in this region.
Qualtech Consulting Corporation
Taiwan, China, Japan, Singapore, Hong Kong, Malaysia, Philippines, Vietnam, Australia, Germany, Korea, Thailand, USA
A specialized medical device consulting firm offering a one-stop solution for complex global regulatory challenges. We offer real-time regulatory and clinical support, local representation, and QMS services across 13 markets, ensuring efficient market entry and compliance.
Cobridge Co., Ltd.
Tokyo, Japan
We assist medical device companies with the medical device registration and approval in Japan. Regulatory consulting services and DMAH services for foreign manufacturers to enter Japanese market.
MDREX, Medical Device, Digital Health Consulting Group
Seoul, Republic of Korea (HQ), Japan Office
We offer total solutions for market entry in South Korea and global expansion (e.g., Japan, USA, Europe). Key areas include product approval, reimbursement listings (HIRA), and Quality System certification (KGMP). They are particularly strong in innovative products like SaMD, medical wearables, and 3D printing for medical use, and provide in-depth expertise in cybersecurity and clinical trial planning.
CMIC Holdings Co., Ltd.
Tokyo, Japan (HQ), Osaka, Japan, Beijing, China, Seoul, South Korea, Taipei, Taiwan, Singapore, New York, USA, London, UK, Frankfurt, Germany, Sydney, Australia
We operate globally, specializing in accelerating the development, manufacturing, and commercialization of drugs and medical devices. Their expertise spans Phase I to IV clinical trials, regulatory affairs, quality assurance, and manufacturing, with a strong focus on the Japanese and Asian markets. Key services include clinical operations (CRO), manufacturing (CDMO/CMO), site management (SMO), and comprehensive health analysis and solutions.
January 6, 2026
Approximately 5 minutes
Points to Consider in Developing Drugs for Pediatric Inflammatory Bowel Disease in Japan
Points to Consider in Developing Drugs for Pediatric Inflammatory Bowel Disease in Japan
1. Background
In March 2025, the Pharmaceuticals and Medical Devices Agency (PMDA) published an Administrative Notice outlining Points to Consider in Developing Drugs for Pediatric Inflammatory Bowel Disease (IBD), including ulcerative colitis (UC) and Crohn’s disease (CD). This guidance was issued to support early planning of clinical studies tailored to pediatric populations, as most approved IBD agents in Japan have been studied primarily in adults. (pmda.go.jp)
Children with IBD have unique challenges compared to adults; for example, diagnostic and activity assessments such as endoscopy pose a greater burden, and standard adult symptom indexes may not fully capture pediatric disease activity. Given these differences, the guidance helps sponsors anticipate pediatric-specific planning needs.
2. Pediatric Unmet Needs and Limited Options
Although a variety of therapies (including biologics and targeted agents) have been approved for moderate to severe IBD in adults, pediatric dosage and administration information are often lacking, leading to limited approved options for children. As a result, product labels frequently state that clinical studies in pediatric populations have not been conducted. This gap underscores the need for pediatric-focused development.
3. Key Considerations for Pediatric Development
3.1 Identify Pediatric Endpoints
Pediatric trials should define appropriate efficacy and safety endpoints, which may differ from adult measures. Surrogate or adapted measures that reflect pediatric disease severity and progression should be justified scientifically.
3.2 Assess Burden and Feasibility
Invasive procedures such as endoscopy require careful consideration regarding burden and feasibility in children. Protocols should minimize unnecessary procedures and incorporate pediatric-appropriate assessments.
3.3 Dosing Strategy
Designing age- and weight-appropriate dosing regimens is crucial, incorporating pharmacokinetic and pharmacodynamic data to ensure safety and efficacy across pediatric subgroups.
3.4 Extrapolation from Adult Data
Where feasible, extrapolation of efficacy from adult data may be considered, especially in older pediatric subpopulations, but the rationale must be scientifically justified with supportive data.
3.5 Safety Monitoring
Enhanced safety monitoring strategies tailored to pediatric physiology and developmental status should be included in study designs, with provisions for long-term follow-up where appropriate.
4. Early Engagement with PMDA
The notice emphasizes early consultation with PMDA during planning stages. Sponsors are encouraged to discuss pediatric trial designs, endpoints, and regulatory strategies to align study plans with regulatory expectations and avoid delays during review.
5. Summary
The Points to Consider guidance provides a framework to address pediatric-specific challenges in IBD drug development, encouraging thoughtful planning of clinical studies that balance scientific rigor with ethical and practical considerations for children. As pediatric drug development is still evolving, these considerations may be updated with emerging evidence and scientific advancement. (pmda.go.jp)
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