Registration of blood products, vaccines and ATMPs

• Registration of blood products and the use of the Plasma Master File
• Development and regulation of vaccines
• Case Study: Approval of HPV vaccines in the EU
• ATMPs


Learning Objectives

• Get a detailed introduction into the product classes of blood products, vaccines and ATMPs
• Understand why blood products, vaccines and ATMPS require a particular development programme
• Understand the underlying principle of the Plasma Master File and the Antigen Master File
• Learn the specific requirements applicable for pandemic vaccines
• Get an insight into a special case of a vaccine presented by a regulatory and a company representative
• Learn the specific regulation of ATMPs and the practical requirements for development

Content

Three unique product classes are introduced with respect to their regulation, the essential requirements for their development, their unique characteristics. Blood products stand for classical biologicals as they are manufactured from a biological source, i.e. human plasma. Their use is still required for many life-threatening and chronic diseases despite the progress recombinant technology made. Vaccines are more and more recombinant proteins and an increasing number of newly developed products is available which are all of special importance for the health systems. Both blood products and vaccines require special considerations in their manufacture, selection of starting material, testing and characterization and clinical development. Advanced therapy medicinal products are also unique as they are based on gene therapy, somatic cell or tissue engineering approaches. For these products the conventional requirements for biopharmaceuticals often do not apply as they are developed and manufactured for individual patients. The development process of a biopharmaceutical has principle features in common with conventional drugs but is special in various aspects since their protein nature requires specific considerations. The three featured product classes illustrate how the current regulatory system ensures that a holistic approach of interaction between the manufacturing/testing, preclinical and clinical team is followed during development.

Agenda

Day 1: Wednesday, 14 December 2011 (10.30 - 18.00)

Future regulation of seasonal and pandemic influenza vaccines
• Pre- and post-authorisation procedures and documentation requirements
• Stakeholders and their roles
• Crisis management plan
• Dynamic Benefit / Risk assessment

Day 3: Friday, 16 December 2011 (09.00 - 16.00)

The industry perspective and experience with the recent pandemic influenza
• Practice of registration of pandemic influenza vaccines
• Governmental contracts, storage and supply
• Communication
• Lessons learned for future practice

Cervarix as an example for successful authorisation of a HPV vaccine
• Introduction
• Regulatory history
• Clinical development - primary authorization and life cycle of the clinical indication
• Success factors