What is the process for approval and access to medicines?
Regulators recommend the authorisation of a medicine where the benefits are judged to be greater than the risks. Following regulatory approval, there are different national pricing and reimbursement processes, which in most cases, must conclude before patients have the opportunity to access treatment.
However, for some patients who have an exceptional need for rapid access, waiting may not be an option. Currently, there are pathways to enable such patients to access therapies in a minority of countries. Where they do exist, early access schemes were designed with chronic therapies (which have to be taken continuously) in mind. These schemes are often less suited to single-administration therapies such as gene therapies. As a result, patients who need early access to these therapies could miss out.
