2009-12-31-世界卫生组织-Guidelines_on_evaluation_of_similar_Biotherapeutic_Products_SBPs_,_Annex_2,_TRS_No_977_40页_489kb

Summary of Guidelines on Evaluation of Similar Biotherapeutic Products (SBPs)

These guidelines provide principles for evaluating and licensing similar biotherapeutic products (SBPs), which are products designed to be highly similar to licensed originator biotherapeutics. The aim is to ensure that SBPs meet acceptable standards of quality, safety, and efficacy for public health purposes, while reducing data requirements through a stepwise approach based on similarity.

Key principles include a comparability exercise involving head-to-head comparisons with a reference biotherapeutic product (RBP), demonstrating similarity in quality, nonclinical, and clinical aspects. RBPs must be originator products with full licensing data, and SBPs must match in dosage form, route, and other attributes.

Quality evaluation requires thorough characterization, including physicochemical, biological, and immunogenic properties, using state-of-the-art analytical methods. Nonclinical and clinical studies must be conducted to assess safety, efficacy, and immunogenicity, with a focus on equivalence/non-inferiority designs for clinical trials. Pharmacovigilance is essential for post-marketing surveillance to detect rare adverse events.

National regulatory authorities (NRAs) play a crucial role in regulating SBPs, and harmonization is encouraged through international guidelines. The guidelines are a "living" document, intended to be updated with scientific advancements.

Main Sections Overview:

• Introduction (Aim and Scope): Addresses global biotherapeutic evaluation amid cost concerns, defining SBPs and their distinctions.
• Scientific Considerations: Outlines challenges in licensing SBPs due to complexity and the necessity of comparability exercises.
• Key Principles for Licensing: Emphasizes stepwise reduction in data requirements via similarity demonstration.
• Reference Biotherapeutic Products (RBPs): Guidelines on RBP selection and justification.
• Quality, Nonclinical, and Clinical Evaluation: Detailed criteria for analytical, pharmacokinetic, pharmacodynamic, and efficacy studies.
• Regulatory Roles: Functions of NRAs and WHO in overseeing SBP licensing and monitoring.

Recommendations:

• Similarity must be proven through integrated data, with equivalence/non-inferiority margins pre-defined.
• SBPs are not interchangeable with generics and require tailored regulatory frameworks.
• Post-licensing pharmacovigilance ensures ongoing safety assessment.