2016-01-13-世界卫生组织-Regulatory_assessment_of_approved_rDNA-derived_biotherapeutics,_Annex_3,_TRS_No_999_16页_144kb

Annex 3 provides a framework for the regulatory assessment of rDNA-derived biotherapeutics previously licensed with data that do not meet current international standards. This guidance emphasizes the need for a stepwise regulatory review process to ensure the products' quality, safety, and efficacy align with WHO guidelines.

Key sections include:

1. Introduction: Addresses biotherapeutic products licensed with incomplete data (e.g., generic pathway or limited analytical, nonclinical, and clinical data).

2. Regulatory Expectations: Requires head-to-head comparability studies for similar biotherapeutic products (SBPs) to ensure similarity to reference products (RBPs). SBPs are not considered generic medicines.

3. Review of Products on the Market: NRAs must identify products licensed without adequate data and conduct a stepwise review, involving manufacturers in a plan to address gaps.

4. Stepwise Regulatory Assessment: Outlines a five-step process for NRAs, including identifying products with gaps, determining appropriate actions, and reassessing data.

5. Regulatory Actions: Decisions based on risk-benefit assessments may include requiring additional data or re-evaluation. Capacity-building support is recommended.

6. Other Considerations: The importance of pharmacovigilance, terminology, and international collaboration, along with transparency in the review process, is highlighted.

Overall, the document provides a flexible approach to enhancing access to safe and effective biotherapeutic products through a structured, evidence-based regulatory oversight.