Masterstudiengang "Drug Regulatory Affairs"


Non-Clinical and Clinical Development of Advanced Therapy Medicinal Products (ATMPs)

Dr. Agnieszka Pietas (Abschlußjahr: 2009)

Language: English

This work presents the current European Union regulatory framework with regard to non-clinical and clinical development of advanced therapy medicinal products (ATMPs) and raises regulatory points for consideration for pharmaceutical companies developing these products. To round off the information selected examples of development programmes for ATMPs are presented.

The development of these medicinal products is at a relatively early stage, and both pharmaceutical companies developing the products and the regulatory agencies involved in giving advice and assessing marketing authorization applications, have much to learn. For this reason regulatory procedures pertaining to ATMPs were aimed by the legislators to provide for sufficient flexibility, so as to easily accommodate the rapid evolution of science and technology in this field. Frequent revisions and amendments of the regulatory requirements are therefore foreseen.

In general, the same regulatory principles apply to ATMPs as for other types of biotechnology medicinal products. However, technical requirements, in particular the type and amount of preclinical and clinical data necessary to demonstrate the safety and efficacy of an ATMP, may be highly specific making strategy for regulatory approval just unique as the product itself.

There are a number of specific issues that should be thoroughly investigated in non-clinical studies with ATMPs, such as viability, longevity, distribution, growth, differentiation and migration of somatic cellular products; potential for germline transmission and/or reactivation of vector for gene therapy products; the duration of observations etc. Other important factors to consider are determining the most useful animal model and extrapolation from animals to the human situation.

A clinical development programme for an investigational ATMP should proceed through an orderly series of exploratory and confirmatory clinical studies. The number of clinical studies as well as the specific design requirements for each of these studies depends upon multiple factors, including the characteristics of the investigational product, the route of product administration, the characteristics of the target patient population and the proposed product indication. Due to unique and diverse structural and biological properties of ATMPs, their development should be subject to case-by-case risk analysis and may require product-dependent designs for clinical studies.

Consequently, this work can provide only a broad outline of the major points to consider in designing non-clinical and clinical studies with ATMPs.

Pages: 67