Regulation of innovative medicinal products including advanced therapies (ATMP)

Medicinal products for advanced therapies are regulated according to the ATMP Regulation 2007/1394/EC

This page informs about the ATMP regulation in the EU and provides an overview over relevant contact points for additional information regarding the development of innovative medicinal products, including ATMP.

Definitions

Definitions from REGULATION (EC) No 1394/2007 on advanced therapy medicinal products

ATMP (Advanced Therapy Medicinal Products) are biological medicinal products based on cells, tissue and genes. They are divided in gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineered products and combined advanced therapy medicinal products.

Gene therapy medicinal products

as defined in Part IV of Annex I to Directive 2001/83/EC, chapter. 2.1

Gene therapy medicinal product means a biological medicinal product which has the following characteristics:

(a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence;

(b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence.

Gene therapy medicinal products shall not include vaccines against infectious diseases

Somatic cell therapy medicinal product

as defined in Part IV of Annex I to Directive 2001/83/EC, chapter 2.2

Somatic cell therapy medicinal product means a biological medicinal product which has the following characteristics:

(a) contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physio[1]logical functions or structural properties relevant for the intended clinical use have been altered, or of cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor; (b) is presented as having properties for, or is used in or administered to human beings with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues. For the purposes of point (a), the manipulations listed in Annex I to Regulation (EC) No 1394/2007, in particular, shall not be considered as substantial manipulations.

Tissue engineered product

as defined in the ATMP REGULATION (EC) No 1394/2007, point (b):

Tissue engineered product means a product that:

— contains or consists of engineered cells or tissues, and

— is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue.

A tissue engineered product may contain cells or tissues of human or animal origin, or both. The cells or tissues may be viable or non-viable. It may also contain additional substances, such as cellular products, bio-molecules, biomaterials, chemical substances, scaffolds or matrices.

Is it a medicinal product? Ask DKMA

Definition of medicines and other products

Is it an ATMP? Ask EMA

Advanced therapy classification | European Medicines Agency (europa.eu)

Regulatory advice

Regulatory advice from DKMA: National scientific advice:

National Scientific Advice (laegemiddelstyrelsen.dk)

Regulatory advice from EMA:

Innovation in medicines | European Medicines Agency (europa.eu)

Support to SMEs | European Medicines Agency

Scientific advice and protocol assistance | European Medicines Agency

Good Manufacturing Practice (GMP)

Legislation on GMP and GDP:

Legislation on GMP and GDP (laegemiddelstyrelsen.dk)

GMP for ATMP:

EudraLex - Volume 4 (europa.eu)

Guidelines GMP for ATMP

Clinical trials

Clinical trials of medicines in Denmark

Clinical trials of medicines (laegemiddelstyrelsen.dk)

Particular demands for first-in-human clinical trials:

Strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products - Scientific guideline | European Medicines Agency (europa.eu)

EMA guideline on documentation requirements regarding ATMP in clinical trials:

Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials - Scientific guideline | European Medicines Agency (europa.eu)

EMA guidelines regarding ATMP:

Guidelines relevant for advanced therapy medicinal products | European Medicines Agency

EDQM guide to the quality and safety of tissues and cells for human application:

Guide to the quality and safety of tissues and cells for human application - European Directorate for the Quality of Medicines & HealthCare

Contact point for the clinical trial department:

Telephone: 4488 9123 / mail: clinical trials

Recommendation on the format of the submitted documentation for clinical trial applications:

As described in EU Regulation No 536/2014, the applicant is recommended to provide documentation prepared in accordance with international guidance such as that of the ICH Common Technical Document (CTD) format as also followed in the EMA scientific guidelines.

In addition to the guidance found for clinical trials under Eudralex – Volume 10 as listed below, reference is also given to the Notice to Applicants and the marketing authorization application templates.

Here detailed guidance for the description of the CTD for medicinal products for human use can be found.

Even though it is for marketing authorization applications, it provides a helpful description of the format and content of the regulatory documentation, which can be accommodated to the stage of development of the investigational medicinal product.

Notice to Applicants on the Common Technical Document

Template for marketing authorization with detailed guidance for non-clinic

Template for marketing authorization with detailed guidance for quality/CMC

Clinical Trial Guidelines

EudraLex - Volume 10 - European Commission (europa.eu)

Chapter III - Quality

And chapter V - Additional Documents