Regulatory information

The regulations governing research in Europe is complex and changing. In an attempt to make keeping up with current regulations as easy as possible you will find in these pages information on EU-wide and national organisations with a remit for regulation as well as relevant documents and updates.

Disclaimer!
ESGCT has compiled this page for its members as helpful information. The European union legislation published in the paper version of the 'official Journal of the European Communities' is the only one to be deemed as official.

EUROPEAN-WIDE AGENCIES

European Medicines Agency (EMA)

The EMEA was established by Council Regulation (EEC) No 2309/93 of 22 July 1993 and London was chosen as its seat. The agency is in charge of coordinating scientific resources existing in Member States with a view to evaluating and supervising medicinal products for both human and veterinary use. On the basis of the Agency’s opinion, the European Commission authorizes the marketing of new medicinal products and arbitrates between Member States for other medicinal products in case of disagreement. Information about regulation of gene and cell therapy can be searched on the EMEA Web Site. Currently, you can find on the EMEA webpage under "Human Medicines" the section "Advanced Therapies" with information relevant for gene and cell therapy.

The common Website for the medicines authorities in the European Union (Heads of Medicines Agencies) is http://www.hma.eu/.

NATIONAL MEDICAL AGENCIES

Under http://www.hma.eu/ you will find a list with links to the EU Member State Medicines Agencies. You can find those specific to your country by following the link "Choose your country".

Useful documents from the European Parliament, the Council and the EMEA

The authorisation and supervision of medicinal products for human use including GMP (Good Manufacturing Practice) guidelines.

On 22 November 2002, the European Commission,DG Enterprises released updated versions of Chapter 1 "Marketing Authorisation", Chapter 2 "Mutual Recognition" and Chapter 3 "Community Referral" of the Notice to Applicants on Medicinal Products on the procedures for marketing authorisation following the adoption of the Community Code relating to medicinal products for human use (2001/83/EC of the European Parliament and of the Council) in force since 18 December 2001.

Modified proposal for a Regulation of the European Parliament and of the Council laying down Community procedures for the authorisation and supervision and pharmacovigilance of medicinal products for human and veterinary use and establishing a European Agency for the Evaluation of Medicinal Products (COM (2002) 735 Final).

Proposal for a REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Agency for the Evaluation of Medicinal Products.

Proposal for a DIRECTIVE OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL amending Directive 2001/83/EC on the Community code relating to medicinal products for human use.

Proposal for a DIRECTIVE OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL amending Directive 2001/82/EC on the Community code relating to veterinary medicinal products.

DIRECTIVE 2001/83/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 6 November 2001 on the Community code relating to medicinal products for human use.

Commission Directive 91/356/EEC of 13 June 1991 laying down the principles and guidelines of good manufacturing practice for medicinal products for human use.

Council Directive 89/381/EEC of 14 June 1989 extending the scope of Directives 65/65/EEC and 75/319/EEC on the approximation of provisions laid down by law, regulation or administrative action relating to proprietary medicinal products and laying down special provisions for medicinal products derived from human blood or human plasma.

Council Directive 75/319/EEC of 20 May 1975 on the approximation of provisions laid down by law, regulation or administrative action relating to medicinal products.

Council Directive 65/65/EEC of 26 January 1965 on the approximation of provisions laid down by law, regulation or administrative action relating to medicinal products.

Directives about use of genetically modified micro-organisms.

COUNCIL DECISION 2002/813/EC of 3 October 2002 establishing, pursuant to Directive 2001/18/EC of the European Parliament and of the Council, the summary notification information format for notifications concerning the deliberate release into the environment of genetically modified organisms for purposes other than for placing on the market.

COUNCIL DECISION 2002/812/EC of 3 October 2002 establishing pursuant to Directive 2001/18/EC of the European Parliament and of the Council the summary information format relating to the placing on the market of genetically modified organisms as or in products.

COUNCIL DECISION 2002/811/EC of 3 October 2002 establishing guidance notes supplementing Annex VII to Directive 2001/18/EC of the European Parliament and of the Council on the deliberate release into the environment of genetically modified organisms and repealing Council Directive 90/220/EEC.

COMMISSION DECISION 2002/623/EC of 24 July 2002 establishing guidance notes supplementing Annex II to Directive 2001/18/EC of the European Parliament and of the Council on the deliberate release into the environment of genetically modified organisms and repealing Council Directive 90/220/EEC.

DIRECTIVE 2001/18/EC OF THE EUROPEAN PARLIAMENT ANDOF THE COUNCIL of 12 March 2001 on the deliberate release into the environment of genetically modified organisms and repealing Council Directive 90/220/EEC.

Council Directive 98/81/EC of 26 October 1998 amending Directive 90/219/EEC on the contained use of genetically modified micro-organisms.

Commission Directive 97/35/EC of 18 June 1997 adapting to technical progress for the second time Council Directive 90/220/EEC on the deliberate release into the environment of genetically modified organisms.

Commission Directive 94/15/EC of 15 April 1994 adapting to technical progress for the first time Council Directive 90/220/EEC on the deliberate release into the environment of genetically modified organisms.

Council Directive 90/219/EEC of 23 April 1990 on the contained use of genetically modified micro-organisms.

Council Directive 90/220/EEC of 23 April 1990 on the deliberate release into the environment of genetically modified organisms.

Question and Answers on the regulation of GMOs in the EU.

Good Clinical Practice (GCP).

DIRECTIVE 2001/20/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use.

Advanced Therapies - Scientific Guidelines

Gene Therapy Working Party (GTWP)

• Work Plan
• Mandate
• Reports

EMEA/CHMP guidance documents

• CHMP/GTWP/587488/08 Reflection Paper Quality, non-clinical and clinical issues relating specifically to recombinat adeno-associated viral vectors (Released for consultation Mar 2009)
• CHMP/GTWP/60436/07 Guideline on Follow-up of patients administered with gene therapy medicinal products (Released for consultation May 2008)
• CHMP/GTWP/125491/06 Guideline on Scientific Requirements for the Environmental Risk Assessment of Gene Therapy Medicinal Products (Adopted by CHMP May 2008)
• Overview of comments received on the above draft
• EMEA/273974/05 Guideline on Non-Clinical testing for Inadvertent Germline transmission of Gene transmission of Gene Transfer Vectors (Adopted by CHMP November 2006)
• Overview of comments received on the above draft
• EMEA/CHMP/GTWP/125459/06 Non-clinical studies required before first clinical use of gene therapy medicinal products (Adopted by CHMP May 2008)
• Overview of comments received on the above draft
• CHMP/BWP/2458/03 CHMP Guideline on Development and Manufacture of Lentiviral Vectors
• CPMP/BWP/3088/99 Note for Guidance on the Quality, Preclinical and Clinical Aspects of Gene Transfer Medicinal Products

ICH activities

• ICH website
• EMEA/25059/02 ICH Communication Paper on First Workshop on Gene Therapy (9 September 2002)
• Programme, report and speakers presentations from the EMEA/ICH Workshop on Viral/Vector Shedding (30 October 2007, Rotterdam)

Cell therapy and tissue engineering

• Guideline on Human cell-based medicinal products
• Overview of comments received on the above draft

Cell-based Products Working Party (CPWP)

• Work Plan 2008

EMEA/CHMP guidance documents

• CPMP/1199/02 Points to Consider on Xenogeneic Cell Therapy Medicinal Products

Pharmacovigilance

Draft guideline on Safety and Efficacy Follow-up - Risk Management of Advanced Therapy Medicinal Products.

The guideline is released for public consultation. Please, send comments to Dr Jan Petracek at jan.petracek@emea.europa.eu by 15th August 2008.

GMP Advice

While EMEA welcomes questions on its activities and the regulatory framework within which it operates, specific questions on the interpretation of GMP requirements should be addressed, ideally by the Qualified Person, directly to the relevant supervisory authority of the Member State in which the manufacturing authorisation holder is located. Manufacturers based in third countries should contact the authority supervising the authorised importer.

Documents of interest:

• Eudralex Volume 4: GMP Guidelines
• Directive 2003/94/EC
• Directive 91/412/EEC
• Good Distribution Practice
• Information for Qualified Persons on compliance with the requirements of the Marketing Authorisation Revision 1
• Update on the status of revision of Chapter 5 of the GMP Guide concerning "Dedicated Facilities"
• Community project on the practical implementation of the new obligations for manufacturing authorisation holders (Art. 46f/50f Directive 2001/83(2)/EC)
• Update on GMP for Advanced Therapy Medicinal Products

Good Clinical Practice (GCP)

http://www.emea.europa.eu/Inspections/GCPgeneral.html

NORTH AMERICA REGULATORY AGENCY

The Center for Biologics Evaluation and Research (CBER) of the US Food and Drug Administration (FDA)

National Institutes of Health

FDA - CDER - Guidance Documents

Useful links

Our partner journal Human Gene Therapy

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