EMA Documents and Guidelines
Guideline on Follow-up of patients administered with gene therapy medicinal products (CHMP/GTWP/60436/07). The guideline is describing recommendations for clinical monitoring and follow-up after treatment with Gene Therapy (GT) medicinal products in order to detect early signals of delayed adverse reactions, to prevent clinical consequences of such reactions and to ensure timely treatment and to gain information on the long-term safety and efficacy of the intervention. The principles laid down in this guideline are applicable for patients enrolled in clinical trials using GT medicinal products and for patients administered with authorised GT medicinal products. The follow-up recommendations take into consideration the risk profile of the gene therapy, the disease, co-morbidity and the patient target population and characteristics. Released for Consultation: May 2008.
Guideline on the non-clinical studies required before first clinical use of gene therapy medicinal products (EMEA/CHMP/GTWP/125459/06). This guideline defines scientific principles and provides guidance to applicants developing gene therapy medicinal products (GTMPs). Its focus is on the non-clinical studies required before the first use of a GTMP in human subjects. Date for coming into effect: November 2008.
General Principles to Address the Risk of Inadvertent Germline Integration of Gene Therapy Vectors (CHMP/ICH/469991/2006). This document identifies general principles for investigating and addressing risks for inadvertent germline integration and provides considerations to minimise this potential risk in humans enrolled in clinical trials. This document applies to gene therapy vectors and could also apply to oncolytic viruses. Released for information: November 2006.
Environmental Risk Assessments for Medicinal Products Containing, or Consisting of, Genetically Modified Organisms (GMOs) (Module 1.6.2) (EMEA/CHMP/BWP/135148/2004). The application of the Centralised procedure to Marketing Authorisation (MA) applications for medicinal products consisting of or containing GMO(s) (GMO(s) as or in medicinal products) constitutes the scope of this document. Proposals for using GMOs in clinical trials fall outside the scope. Specifically, the guidance presented outlines both the procedural issues affecting applications for MA for these products and the information related to the Environmental Risk Assessment (ERA) which should be included in the applications. Date for coming into operation: End of 2005.
Guideline on Development and Manufacture of Lentiviral Vectors (CHMP/BWP/2458/03). This guideline describes quality aspects and non-clinical testing that are in general relevant for lentiviral vectors that are intended for ex vivo or in vivo application. Date for coming into operation: November 2005.