Good clinical practice (GCP) is essential for clinical trials conducted for all medicinal products, however this guidance outlines specific requirements for GCP which are applicable to ATMPs.
Advanced therapy medicinal products (ATMPs) are innovative products, such as gene and cell therapies, which can pose a wide variety of challenges to the preparation of clinical trials. Difficulties could include manufacturing constraints and the short shelf-life of the product. Additionally, it may be challenging to use placebo controls due to the complex mode of application and training requirements to administer ATMPs.
The guidelines discuss various GCP criteria, including clinical trial design, non-clinical studies, quality of the investigational ATMP, trial safety, upstream interventions and administration procedures. The guideline also states that the use of each product should be traceable from delivery to the clinical trial site through to administration to the subject, and for ATMPs that contain cells or human tissues, from the subject to the donor of the cells or tissues. Furthermore, it is recommended that the retention period of ATMP samples can be adjusted to accommodate the varying stability and shelf-life of ATMPs. Considerations regarding protection of clinical trial subjects are outlined, such as obtaining informed consent, long-term follow-up and safety reporting of adverse events. The sponsor should provide information and training to the investigator on any additional protocol necessary for the reporting of adverse events in the clinical trial. The sponsor should also profusely monitor the conduct of the clinical trial.
Compliance with GCP is mandatory for clinical trials in the European Union (EU). To access the complete guidance, click here. These guidelines are to be read alongside the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines. They also coincide with the recent consultation on a manufacturing guide for ATMPs.