A gene therapy study involving humans will likely require assessment by a number of different bodies.
The bodies use different procedures, based on various legislation and regulations, which are almost always based on EU legislation and regulations. Any changes, if at all feasible, would therefore take several years. For this reason, in order to achieve a result as quickly as possible, one of the main prerequisites in the establishment of the Gene Therapy Office was to remain within existing legislation and regulations. The guidelines are therefore based on the various procedures in existence, although agreements have also been made between the bodies involved as to how more cooperation and coordination can be achieved. The intention is to make things simpler and clearer for the investigator.
The following pages describe the details of this cooperation and coordination and then provides details on the individual procedures, using diagrams and short clarifications of the different steps.
The decisions of the CCMO (Central Committee on Research involving Human Subjects) and of the Ministers of VWS (Health, Welfare and Sport) and IenW (Infrastructure and Watermanagement) are formally unrelated and the three bodies each retain their own responsibility. However, synchronising the procedures does enable the bodies to be informed of one another’s point of view and prevents the investigator receiving contradictory decisions or statements. For this reason, and with a view to making the whole process more transparent and simpler for the investigator, a number of improvements have been made:
- A single application form, which reduces the overlap in the data requested.
- A single ‘office’ for the submission of the application, located at the GMOGenetically Modified Organism office.
- Automatic distribution of the application form by the Gene Therapy Office to all the bodies involved.
- All bodies appoint a permanent case manager per application. These case managers are jointly responsible for mutual coordination during the different procedures, including the possibility of attending commission meetings as non-participants.
- Feedback to the investigator through the Gene Therapy Office with regard to the final assessments of the different bodies.