CONCLUSION

Different from traditional medicines:

• Medical potential. Cell and gene therapies treat the underlying genetic cause of medical conditions. This means they can have a long-term, positive effect on patient health, and in some cases even provide a cure.

• Single treatment. Cell and gene therapies are often only administered once or a few times within a short period of time. this is in contrast with many medicines for chronic conditions which often need to be taken daily, for the rest of the person’s life.

• High costs in advance. Because the therapy is administered once, all treatment costs occur at the beginning, at a point in time when the long-term effects of treatment are still unclear.

• Complex manufacturing. Cell and gene therapies are manufactured via complex processes. Some cell therapies use the patient’s own cells. This means the treatment is unique for each patient, requiring its own manufacturing process. Specialised centres and highly trained staff are required to ensure this manufacturing process is performed properly. Because live cells are often used, manufacturing is susceptible to disruptions and errors.

• Storage. Some cell and gene therapies contain live cells that have specific storage and transportation requirements. The limited time that cells can survive outside the body must also be taken into account.

• Specific rules for marketing authorisation and pharmacovigilance. The EMA has defined a specific set of regulations and legislation for the marketing authorisation of cell and gene therapies, and for post-marketing pharmacovigilance. Among other reasons, this is because many are composed of biological materials. In some cases, this material is modified genetically. This means the therapy is classified as ‘genetically modified’. Pharmacovigilance rules taken into account the specific risks of cell and gene therapies.