2011-05-31-世界卫生组织-Paediatric_clinical_trials_guidance_for_assessors_41页_525kb

Paediatric clinical trials guidance for assessors outlines a rigorous framework for evaluating clinical trials involving children. The key elements include:

1. Ethical and Scientific Scrutiny: Assessors must evaluate if trials are necessary, ethically sound (aligned with Declaration of Helsinki principles), and scientifically valid, ensuring maximal benefit and minimal risk to children.

2. Specific Areas of Assessment:

   • Medicine Description: Covers regulatory status, formulation (palatability, dosing accuracy for small children), and bioavailability considerations.
   • Non-clinical Safety: Requires extensive toxicology data, including juvenile studies, to ensure safety for developing organs.
   • Pharmacology: Emphasizes age-specific pharmacokinetics/pharmacodynamics due to maturation effects, requiring specialized modeling.
   • Trial Design: Includes feasibility assessments, pediatric-specific endpoints, and robust safety monitoring (e.g., DSMB use).

3. Regulatory and Pediatric-Specific Considerations: Requires waiver considerations under ICH E11, PIPs/WRs, and evaluation of pediatric evidence gaps. Trials must account for differences between pediatric and adult populations.

4. Risk Management: Utilizes pediatric risk classification systems (no/minimal risk, minor increase, greater increase) to inform ethical approval.

5. Bioequivalence: Generally applies to adults; pediatric trials focus on proper formulation and safety, not bioequivalence assessment.

6. Regulatory Alignment: References ICH guidelines (E3, E6, E9, E10, E11) and WHO/EUDRACT standards to ensure trial protocols meet international regulatory expectations.

This guidance ensures consistent, child-centric evaluation of pediatric clinical trials by regulatory assessors.