Table 2.
Key EU (EMA) regulatory guidance documents and reflection papers for ATMPs. The EMA and its specialist group the Committee for Advanced Therapies publishes guidance documents and reflection papers to assist developers of cell and gene therapies. The table provides their titles and document identifiers.
| guidance |
|---|
| — guideline on human cell-based medicinal products (EMEA/CHMP/410869/2006) |
| — guideline on the non-clinical studies required before first clinical use of gene therapy medicinal products (EMEA/CHMP/GTWP/125459/2006) |
| — guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (EMA/CAT/GTWP/671639/2008) |
| — guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to advanced therapy medicinal products (EMA/CAT/CPWP/686637/2011) |
| — guideline on safety and efficacy follow-up—risk management of advanced therapy medicinal products [EMEA/149995/2008] |
| — guideline on scientific requirements for the environmental risk assessment of gene therapy medicinal products [EMEA/CHMP/GTWP/125491/2006] |
| — detailed guidelines on good clinical practice specific to advanced therapy medicinal products [ENTR/F/2/SF/dn D(2009) 35810] |
| — reflection paper on stem cell-based medicinal products [EMA/CAT/571134/2009] |
| — reflection paper on classification of advanced therapy medicinal products [EMA/CAT/600280/2010] |
| — draft reflection paper on clinical aspects related to tissue engineered products [EMA/CAT/CPWP/573420/2009] |
| — reflection paper on management of clinical risks deriving from insertional mutagenesis [EMA/CAT/190186/2012] |
| — European Directorate for the Quality of Medicines—guide to the quality and safety of tissues and cells for human application 1st edition |
| — Ph. Eur. Monograph 5.2.12 on raw materials for the production of cell-based and gene therapy products [Pharmeuropa—Issue 26.4, 2014] |
| — annex 2 of Directive 2003/94/EC: manufacture of biological medicinal products for human use |
| — guideline on potency testing of cell-based immunotherapy medicinal products for the treatment of cancer (CHMP/BWP/271475/06) |
| — guideline on development and manufacture of lentiviral vectors (CPMP/BWP/2458/03) |
| — EMA Scientific Guideline: quality, pre-clinical and clinical aspects of gene transfer medicinal products (CHMP/GTWP/234523/09) |
| — EMA Scientific Guideline: gene therapy product quality aspects in the production of vectors and genetically modified somatic cells (3AB6A) |