Children's Ombudsman and Patient Ombudsman appeal to drug manufacturer for reimbursement

Achondroplasia is a rare genetic disease manifested, among other things, by short stature. About 560 children in Poland struggle with it. The Children's Ombudsman and the Patient Ombudsman have made a joint request to the manufacturer of the drug, which is intended for children affected by achondroplasia, to apply for reimbursement.

- We aim to ensure that every child, regardless of age, gender, nationality or stage of illness, has real access to medical services and feels safe during the therapeutic process, Monika Horna-Cieślak and Bartłomiej Chmielowiec argue.

The RPD and the MPC have recently received several hundred submissions from patients, their parents and relatives, in which they point to a problem related to the lack of access in Poland to an innovative therapy with a substance called vosoritide.

All unanimously emphasize that no drug technology is reimbursed in the country for the treatment of achondroplasia. It is only possible to perform a reimbursable surgical procedure to stretch the radius/elbow, femur and tibia/shaft bones. However, it is associated with possible complications, pain and periodic exclusion from social life, and only slightly alleviates some of the effects of achondroplasia.

The advocates jointly point out that children with achondroplasia are particularly vulnerable to mental health disorders, experiencing stigma and rejection from their peer group.

Meanwhile, in 2021. The European Medicines Agency has approved a modern and effective therapy for the treatment of achondroplasia. Also according to Polish experts involved in the treatment of low-grade achondroplasia in children, the use of vosoritide is the only effective method of treating this disease, and due to its serious nature, the implementation of the therapy cannot be delayed.

In Poland, the initiation of proceedings for systemic reimbursement of a given drug can only take place as a result of an appropriate application by the manufacturer to the Minister of Health. Hence the joint appeal of the RPD and the MPC to the company producing the drug. - We are committed to ensuring that Polish children suffering from achondroplasia feel safe and have a chance to function at the same level as their peers in other European countries," stress the signatories of the letter.

Source: MPC