The TOPRA European Medicines Agency Update on Advanced Therapies "our aim is to get new products to patients as soon as we can"
Patrick Celis, Quality of Medicines, Pre-authorisation Unit, EMEA
To serve the need for getting advanced therapies to patients across Europe, the first EU legislation on gene therapy, somatic cell therapy and tissue engineering products will come into place at the end of this year.
At the third annual EMEA Legislation Update, hosted jointly by The Organisation for Professionals in Regulatory Affairs (TOPRA) and the European Medicines Agency (EMEA), the EMEA announced for the first time updated plans on the regulation of Advanced Therapy Medicinal Products (ATMP) where it became apparent that the regulation on advanced therapies is progressing significantly.
The Annex I update of Directive 2001/83/EC, which comes into effect on 30 December 2008, will address:
- Updated definitions for gene therapy medicinal products and stem cell therapy medicinal products to align with scientific development and definition of a tissue engineered product
- Risk-based approach for product development
- Requirements specific for ATMP eg interactions between cells and structural components
- Additional flexibility where needed eg non-clinical / clinical development
The EMEA’s new Committee on Advanced Therapies (CAT) will meet in January to discuss key areas in the certification process of emerging gene therapy, somatic cell therapy and tissue engineering products, including:
- How to find the correct regulatory routes for guidance documents (eg cell-based tumour vaccines)
- How to deal with products that have already been used without evidence?
- Regulation of long-term follow-up of efficacy
- How to perform first-in-human trials?
- How to deal eg with the risk of inserational mutagenesis?
This specialised expertise will guarantee the best scientific evaluation of benefit/risk balance, legal certainty and suitable rules in a predictable, reliable, accountable and integrated EU system and transparency, collaboration, education and communication for the benefit of patients.
Patient organisation representatives can keep one step ahead with TOPRA, with the most up to date training and conference programme for those who have an interest in Regulatory Affairs in the healthcare sector. A full list of events can be found at www.topra.org/events.
