Clinical trials with GMOs: Tools for risk assessment and risk management

For the preparation of the notification dossiers by the applicants and their assessment by the advisory bodies and experts, a (non-exhaustive) list of useful guidelines developed by official bodies is listed below.

Because an increasing number of clinical trials with GMOs involve the use of advanced therapy medicinal products (ATMP), the European Commission created a FAQ, a repository of national regulatory requirements, and good practice documents which can be retrieved from the webpages dedicated to ATMP. These documents are endorsed by a large number of countries and aim at streamlining the information that needs to be provided in the context of (multi-centric) clinical trials. In particular, good practice documents have been developed for adeno-associated viral vectors (AAV) and human cells genetically modified by means of viral vectors.

The European Medicines Agency (EMA) has published several scientific guidelines to help applicants prepare marketing-authorisation applications for human medicines and veterinary medicines. Though less detailed or advanced experimental data may suffice depending on the stage of development of the investigational medicinal product and the scale of its release, these guidelines also serve as supporting tools for the clinical trials with GMOs.
Particularly in relation to the environmental risk assessment of GMO medicinal products the following guidelines (non-exhaustive list) can be of interest (links to the EMA website):

The following guidelines, related to the quality, non-clinical and clinical aspects, may also be of interest (links to the EMA website):

The Biosafety and Biotechnology Unit (SBB) also issued scientific publications that are relevant to the biosafety assessment of GMO (investigational) medicinal products, in particular: