Advanced therapy medicinal products (ang. Advanced Therapy Medicinal Product) is a new category of drugs in which cells, tissues and genes or their combination may be the active substance. ATMP products have great potential for the treatment of various diseases such as cancer, infections, genetic and neuro degradation (neuro-degradaing) diseases as well as disorders of the cardiovascular system. They also play an important role in regenerative medicine as a new therapeutic approach in the treatment of lesions resulting from trauma, pathological conditions, and the natural aging of the body.
There are three categories of advanced therapy medicinal products:
- Cell Therapy Medicinal Products
- Gene Therapy Medicinal Products
- Tissue Engineered Products
Medicinal products used in advanced therapies are the most innovative and complex technologies, which must be regulated by law providing protection of life and health of patients. In order to ensure a common legal standards Regulation (EC) No 1394/2007 of the European Parliament and of the Council on advanced therapy medicinal products (ie. ATMP Regulation) were accepted. It establishes standards for the classification, production, licensing and supervision by the European Medicines Agency according to the highest existing standards, which are performed in order to control the quality, safety and efficacy. The aim of the ATMP Regulation is also to take care of free movement of products ATMP in the European Union.
Despite the necessity to register ATMP in the European Medicines Agency European law does not interfere with national legislation. New products based on cells used in therapy must meet the requirements of existing laws and regulations and national regulations. In Poland, the most important provision for tissues and cells is the Act of July 1, 2005 r.o collection, storage and transplantation of cells, tissues and organs, so-called. Transplant Act of 1 July 2005. In addition, each manufacturer producing advanced therapy medicinal products needs to get a permisssion for the manufacture of the product from Main Pharmaceutical Inspector.
In the highly specialized procedure which is the cell culture in order to prepare for patient transplant the most important requirement is a working conditions. Work can be carried out only in rooms like “clean room”, in the cleanliness class A – this in turn can only be surrounded by rooms in the cleanliness class B, where the air quality and the quantity of particles is monitored. The premises should be carried out to control the number of particles and microbiological monitoring.
The work carried out in “clean room” must be carried out under the supervision and strictly controlled. Appropriate training and maintaining work discipline allows you to maintain conditions ensuring effective work but inappropriate behavior leads to a reduction in the level of cleanliness. All equipment, appliances, and items must be in a particular room and must be used in a appropriate way, sometimes also thay should be designe in a certain way. Personnel directly involved in the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells should be appropriately qualified and provided with timely and relevant training. During processing, they must be provided in a dust-free overalls, masks, gloves, shoes.
In addition to the microbiological control of the rooms, the entire process should be checked and under control. Sterility testing and other checks can not cover only the final stage of production and final product inspection. A significant disadvantage and danger in the case of cell cultures is the inability to get the results of microbiological tests before the transplant. This is an important issue, which applies to all procedures related to the transplantation of cells.
Preparation of this new group of complex medicinal products face many difficulties. They are associated with the production requirements, requirements of the pharmaceutical industry, large financial outlay and a difficult legislative process of registration and the accreditations requirements. Despite the complexity of the procedures and relatively not well-established way of development, the process of translation ATMP is carried out mainly by small and medium-sized enterprises in conjunction with universities. Establishment of cooperation between research centers and clinics enables the implementation of new technologies through the implementation of research projects and clinical trials.
If you are interested in this subject we would like to invite you for a training/ to joinf a workshop, where we will discuss in detail:
- legislation on advanced therapy medicinal products ATMP
- marketing authorization of ATMP
- production conditions of ATMP