Non-clinical and clinical development of biopharmaceuticals including biosimilars and the Module 4 and 5 requirements

• Principles of non-clinical development
• Principles of clinical development
• Immunogenicity
• Case studies on monoclonal antibodies, erythropoietin and a biosimilar
• Case presentation on the tri-functional monoclonal antibody Catumaxomab
• Development and registration of biosimilars



Learning Objectives

• Understand the specific challenges in the pre-clinical development of biopharmaceuticals
• Understand the essential clinical development approach
• Learn to assess the benefit-risk ratio during the whole life cycle of a product
• Understand the data requirements specific to biosimilars
• Learn why immunogenicity is a typical issue for biopharmaceuticals and how it is investigated and handled appropriately
• Allows to conduct and steer the relevant regulatory strategy for CTA and MAA submissions for biopharmaceuticals due to knowing the rules and required data for Modules 4 and 5 of biopharmaceuticals

Content

Biopharmaceuticals play an important role in the pharmaceutical industry since they deliver an increasing number of candidates for new product developments. They are however special and require certain considerations in their pre-clinical and clinical development. They are pleiotrop and immunogenic and their non-clinical pharmacodynamic and safety characterisation is often hampered by insufficiently relevant animal models. Biosimilars are developed following a special regulation and expanded programme as compared to conventional generics. This all has implications on the way biopharmaceuticals are developed and regulated. The particulars of the non-clinical and clinical requirements of biopharmaceuticals are covered and the specific considerations on the development of biosimilar medicinal products are part of this seminar as well. In this seminar case studies will also be presented on successfully authorized products, such as on a tri-functional monoclonal antibody and on a biosimilar. The seminar also introduces how to perform a benefit-risk assessment at the authorization stage and how to update it during life-cycle of the product.

Agenda

Day 1: Tuesday, 06 March 2012 (10.30 - 18.00)

Day 2: Wednesday, 07 March 2012 (09.00-18.00)

Immunogenicity
• What is immunogenicity?
• How is immunogenicity caused?
• How is immunogenicity tested?
• Risk based approach?

Erythropoeitin

The principle of clinical development of biopharmaceuticals
• Target rationale definition
• Dose-finding justification
• Proof of Concept and confirmatory studies
• Relevant efficacy and safety studies - pivotal trials
• Principles of the clinical review and decision on the benefit-risk conclusion
• Module 2 sections 2.5 and 2.7
• Presentation of clinical data in Module 5 of the CTD