Questions? 10 seconds to sign up
Join the platform
Need Regulatory Help? Try Our Platform
Post your regulatory questions or request quotations from verified pharmaceutical consultants worldwide. Get matched with experts who specialize in your market.
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)
Ask Anything
We'll follow up with you personally.
Related Articles
Approximately 5 minutes
Consideration in the development of drugs for pulmonary arterial hypertension in Japan
PMDA’s Early Consideration document provides key strategic points for planning clinical development of PAH drugs in Japan, emphasizing multi-regional trials, choice of endpoints, pediatric considerations, and appropriate study design to address unmet needs.
Approximately 5 minutes
Points to Consider in the Clinical Development of Drugs for Transthyretin Amyloid Cardiomyopathy (Early Consideration) in Japan
This PMDA Early Consideration document outlines strategic clinical development points for transthyretin amyloid cardiomyopathy (ATTR-CM) drugs in Japan, including rare disease considerations, endpoint selection, trial design, and multi-regional development.
Approximately 5 minutes
Points to Consider for the Design of Clinical Trials to Assess the Effects of Psychotropic Drugs on Driving Performance in Japan
This PMDA Early Consideration outlines key considerations for designing clinical trials to assess impacts of psychotropic drugs on driving performance, focusing on clinically meaningful driving impairment and appropriate evaluation methods.
Approximately 5 minutes
Japan Specialty Fulfillment for Medical Devices: Low Volume, High Value Sales
Specialty Fulfillment is a crucial service for foreign medical device manufacturers targeting low-volume, high-value sales in Japan, often utilizing an Independent License Holder to manage customs, storage, and distribution to maintain high profit margins.
Approximately 5 minutes
Points to Consider for the Design of Clinical Trials to Assess the Effects of Psychotropic Drugs on Driving Performance in Japan
This PMDA Early Consideration outlines key considerations for designing clinical trials to assess impacts of psychotropic drugs on driving performance, focusing on clinically meaningful driving impairment and appropriate evaluation methods.
Approximately 5 minutes
Guideline for Clinical Evaluation of Antibacterial Drugs in Japan
The PMDA’s *Guideline for Clinical Evaluation of Antibacterial Drugs* provides comprehensive principles for designing and conducting clinical studies of antibacterial drugs for regulatory approval, reflecting current scientific knowledge and global harmonization.
Approximately 5 minutes
Considerations for Non-clinical Studies of Combination Prescription Drugs with Similar Formulations in Japan
PMDA’s Early Consideration document clarifies how non-clinical pharmacology and toxicology studies should be approached for combination prescription drugs whose formulations are similar to approved products.
Approximately 5 minutes
Points to Consider for Clinical Development of Drugs Intended for Treatment of Psoriatic Arthritis in Japan
This PMDA Early Consideration outlines current regulatory thinking for clinical development of psoriatic arthritis (PsA) drugs in Japan, including disease characterization, endpoints, study design considerations, and use of global data.
Approximately 5 minutes
On Release of the Guideline for Clinical Evaluation of Oral Hypoglycemic Agents in Japan
The *Guideline for Clinical Evaluation of Oral Hypoglycemic Agents* provides comprehensive procedures for planning, conducting, and evaluating clinical studies of oral hypoglycemic agents in Japan, focusing on glycemic control markers, safety evaluation, and inclusion of relevant patient populations.
Approximately 5 minutes
Considerations for Non-Clinical Studies in the Development of Diagnostic Radiopharmaceuticals in Japan
PMDA’s Early Consideration (Mar 26, 2025) explains when non-clinical packages for diagnostic radiopharmaceuticals can be streamlined (e.g., microdose), and how to address safety pharmacology, DDI/ADME, toxicity, and radiolytic impurities.
Approximately 5 minutes
Guideline for Clinical Evaluation of Diagnostic Radiopharmaceuticals in Japan
PMDA/MHLW’s guideline (PFSB/ELD Notification No. 0611-1) explains how to design non-clinical and clinical evidence for diagnostic radiopharmaceutical approval in Japan, emphasizing imaging accuracy, clinical significance, and radiation dosimetry.
Approximately 5 minutes
Checklist for Japan’s 30-day Clinical Trial Notification Review (Initial CTN, Oncology Drugs)
PMDA’s early-consideration checklist summarizes what reviewers expect to see in an initial CTN for oncology drugs—covering safety reporting workflows, Phase 1 population ethics, starting dose rationale, DLT/tolerability evaluation, informed consent essentials, and required attached documents.
Approximately 5 minutes
Statistical Considerations for Phase I Oncology Trials in Japan (Safety-focused Early Consideration)
PMDA’s early-consideration paper explains what statistical evidence and simulation outputs should support oncology Phase I dose-escalation designs in Japan’s 30-day CTN review, with emphasis on safety, termination behavior under excessive toxicity, and managing design modifications like backfilling or schedule changes.