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EMA Documents and Guidelines

Overview of scientific gene therapy guidelines by the EMA. Adopted as well as draft document can be found here. Below is a selection of guidelines.

ICH Considerations - General Principles to Address Virus and Vector Shedding (EMEA/CHMP/ICH/449035/2009). Assessment of shedding can be utilized to understand the potential risk associated with transmission to third parties and the potential risk to the environment. The scope of this document excludes shedding as it relates to environmental concerns because it is regulated differently in various regions. The focus of this document is to provide recommendations for designing non-clinical and clinical shedding studies when appropriate. In particular, emphasis will be on the analytical assays used for detection, and considerations for the sampling profiles and schedules in both non-clinical and clinical studies. The interpretation of non-clinical data and its use in designing clinical studies is also within the scope of this paper, as well as the interpretation of clinical data in assessing the need for virus / vector transmission studies. Date for transmission to CHMP: July 2009.

Guideline on Scientific Requirements for the Environmental Risk Assessment of Gene Therapy Medicinal Products (EMEA/CHMP/GTWP/125491/2006). This guideline deals with the scientific principles and methodology to be used for the environmental risk assessment (ERA) of gene therapy GMO-containing medicinal products for human use, as required for marketing authorisation (MA) under the centralised procedure. Guidance is given on application of the methodology laid down in the Directive 2001/18/EC on the deliberate release of genetically modified organisms into the environment. Date for coming into effect: November 2008.

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.