SUMMARY

Hundreds of clinical trials are currently investigating cell and gene therapies. Also referred to as: Advanced Therapy Medicinal Products. This is a new generation of medical therapies based on cells, genes, tissues or a combination thereof. For example, new cancer therapies have recently become available that use the patient’s own immune cells as a weapon against tumours.

The patient is often both starting point and destination in these therapies: their own cells are transported to a special facility and processed in a number of ways. The resulting medical product – for example immune cells, stem cells or a cornea – is then returned to the hospital. It is given to the patient there. This is a medical and logistical operation that demands a great deal from all involved.

Cell and gene therapies are often designed as a cure or aim to achieve lasting improvements in symptoms or quality of life in a wide range of conditions, including cancer, eye injury, Blood clotting diseases or immune deficiencies. The term ATMP encompasses a number of different technologies, such as gene therapy products, somatic cell therapy products and tissue therapy products.

Since 2009, fourteen cell and gene therapies have received marketing authorisation in Europe. These market launches have been a mixed success. They illustrate the new possibilities offered by these therapies, but also highlight a number of the bottlenecks. For example slow uptake in medical practice, and long debates about costs and reimbursement.

Cell and gene therapies are so innovative and different from traditional medicines that they run into the limitations of existing routines and regulations. This brochure hopes to disseminate knowledge about these therapies and the specific requirements for their production and application.

Raising awareness about cell and gene therapies among all involved parties (researchers, policy makers, companies, doctors and insurers) is a requirement for ensuring these new applications success-fully reach the patient in the coming years.

Photo: Peter Boer