Quality, preclinical and clinical aspects of gene therapy medicinal products - Scientific guideline
This guideline is a revision of the Note for Guidance on the Quality, Preclinical and Clinical aspects of gene transfer medicinal products (CPMP/BWP/3088/99), which was published in 2001. It defines scientific principles and provides guidance for the development and evaluation of Gene Therapy Medicinal Products (GTMPs) intended for use in humans and presented for Marketing Authorisation Application (MAA). Its focus is on the quality, safety and efficacy requirements of GTMPs.
Keywords: Gene therapy medicinal products, advanced therapy medicinal products (ATMPs), quality, non-clinical, clinical
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Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products (PDF/427.63 KB)
Adopted
First published: 13/07/2018
Last updated: 13/07/2018
EMA/CAT/80183/2014 -
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Overview of comments on 'Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products' (EMA/CAT/80183/2014)' (PDF/1.72 MB)
First published: 13/07/2018
Last updated: 13/07/2018
EMA/801611/2016 -
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Draft guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products (PDF/548.89 KB)
Draft: consultation closed
First published: 20/05/2015
Last updated: 20/05/2015
Consultation dates: 20/05/2015 to 31/08/2015
EMA/CAT/80183/2014 -
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Concept paper on the revision of the note for guidance on the quality, pre-clinical and clinical aspects of gene transfer medicinal products (PDF/51.52 KB)
Draft: consultation closed
First published: 13/01/2010
Last updated: 13/01/2010
Consultation dates: 17/12/2009 to 31/03/2010
EMA/CHMP/GTWP/BWP/234523/2009
- Biologicals: active substance
- Multidisciplinary: gene therapy
- Regulation (EC) No 2309/93
- Regulation (EC) No 1394/2007
- Directive 2001/83/EC
- Directive 2009/120/EC
- Directive 2003/94/EC
- Directive 2009/120/EC
Safety and efficacy follow-up - risk management of advanced therapy medicinal products- Directive 2009/41/EC
- Directive 2001/18/EC
- Clinical trials in small populations
- Development and manufacture of lentiviral vectors
- Environmental risk assessments for medicinal products containing, or consisting of, genetically modified organisms (GMOs)
- Excipients in the dossier for application for marketing authorisation of a medicinal product
- Follow-up of patients administered with gene therapy medicinal products
- Good pharmacovigilance practices
- ICH Considerations: general principles to address virus and vector shedding
- ICH Considerations: oncolytic viruses
- ICH E4 Dose response information to support drug registration
- ICH E6 (R1) Good clinical practice
- ICH M3 (R2) Non-clinical safety studies for the conduct of human clinical trials for pharmaceuticals
- ICH Q5B Analysis of the expression construct in cell lines used for production of rDNA-derived protein products
- ICH Q5D Derivation and characterisation of cell substrates used for production of biotechnological/biological products
- ICH S6 (R1) Preclinical safety evaluation of biotechnology-derived pharmaceuticals
- ICH S8 Immunotoxicity studies for human pharmaceuticals
- Management of clinical risks deriving from insertional mutagenesis
- Minimising the risk of transmitting animal spongiform encephalopathy agents via human and veterinary medicinal products
- Non-clinical studies required before first clinical use of gene therapy medicinal products
- Non-clinical testing for inadvertent germline transmission of gene transfer vectors
- Plasma-derived medicinal products
- Preclinical pharmacological and toxicological testing of vaccines
- Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells
- Quality, non-clinical and clinical issues relating specifically to recombinant adeno-associated viral vectors
- Risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to Advanced Therapy Medicinal Products
- Scientific requirements for the environmental risk assessment of gene-therapy medicinal products