REIMBURSEMENT OF THERAPY
Despite the fact that there is no treatment for Usher Syndrome (yet), a great deal of the health care budget is taken up.
These high costs are the result of life-long care, support and aids. The adjustments in the living and working environments, the economic loss as a consequence of full disability for work and the decrease of the quality of life is even added to this. There is no therapeutic cure. The current treatments consist of genetic advice to parents, prenatal diagnostic testing and intervention of hearing rehabilitation.
There is a solution for Usher Syndrome: correction of the genetic defect by, for example, gene therapy, an unfulfilled medical need.
The current remuneration system for medicines is based on medicines that are to be taken for a long time and this does not fit in well with these one-off treatments in the form of gene therapy. It is clear, however, that if these treatments work well or even better, one-off treatments are preferred above life-long therapies.
Expectations are that the advantages of this one-off therapies will in the long term justify the high costs. Some arrangements have been made already for certain gene therapies based on the result of the treatment. If a treatment does not lead to the required result, the manufacturer will reimburse an amount. Another proposal for paying this kind of expensive therapies is an annual payment for a number of years instead of the entire amount at once.