Cell & gene therapy (ATMP)
Advanced Therapy Medicinal Products (ATMPs) represent a fast-growing field of human therapies based on genes, tissues or cells. ATMPs can be classified into three main types:
- gene therapy medicines (GTMP): these contain genes that lead to a therapeutic, prophylactic or diagnostic effect. They work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources;
- somatic-cell therapy medicines (SCMP): these contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor. They can be used to cure, diagnose or prevent diseases;
- tissue-engineered medicines (TEP): these contain cells or tissues that have been modified so they can be used to repair, regenerate or replace human tissue;
ATMPs revolutionize the treatment approach of many previously incurable diseases. It is expected that the number of newly developed and authorised ATMPs is going to increase significantly over the next few years as a result of the blossoming science in this area. In the EU ATMPs can obtain Marketing Authorization (MA) only in Centralised Procedure (CP). Due to the complex nature of these products, it is extremely important to understand the relevant regulations and key procedural aspects. In the European Union (EU) the regulatory framework for ATMPs is primarily established in Directive 2001/83/EC but also further in many other Directives and Regulations applicable in EU member states, especially Regulation (EC) No 1394/2007.
All the regulations in force ensure that new medicinal products classified as ATMPs are subject to appropriate assessment by the European Medicines Agency (EMA). It should guarantee their appropriate quality, safety and efficacy in clinical trials, and further in patient population, when finally granted a MA. Failure to meet the requirements may significantly delay or even hold a product development and/or increase its costs. Therefore, understanding the requirements that ATMPs must meet as well as familiarity with the relevant registration procedures are important factors to successfully lead the product to MA. In the case of ATMP products, the issues of Market Access (Pricing and Reimbursement) are also extremely important. These activities should be planned at the beginning of product development.
A special case of an ATMP product is ATMP-HE, an advanced therapy medicinal product – a hospital exception, not liable to the EMA, but regulated by European directives and regulations (including Directive 2001/83/EC and Regulation EC 1394/2007). Manufacturing of these products is approved by the competent authority of the Member State. The ATMP-HE product is prepared on a non-routine basis under the exclusive responsibility of a physician in order to comply with an individual physician’s prescription following applicable quality standards and is manufactured per GMP principles.
ATMP-HE products may be administered to patients for whom there are no other effective treatments already available, and the information obtained in the course of administration of ATMP-HE products may be used as data in the initial stages of development of a medicinal product and may help register the ATMPs under investigation.
SciencePharma has a team of experts and reverent experience in ATMPs to support you in the development and assessment ATPMs.
Research and development
We offer consulting services in the field of assessing the potential of various projects development of medicinal products, devices or dietary supplements.
Preclinical and clinical development
SciencePharma is a leading, highly experienced consultancy company providing state-of-the-art services in a field of nonclinical and clinical studies.
Audits
Having broad experience in auditing of API manufacturers as well as of contract medicinal products facilities we offer independent GMP auditing services.
Regulatory Affairs
We offer multidisciplinary services in the field of Regulatory Affairs including support for pre-registration, registration and post-registration.
Pharmacovigilance
Pharmacovigilance is the process of monitoring the safety of medicines. Marketing Authorization Holders have a legal obligation to set up and maintain a Pharmacovigilance system.