In an investigation to control the work of competent institutions initiated following a protest by the parents of children suffering from spinal muscular atrophy over an alleged delay with the only medicine’s application, the Protector of Citizens ascertained that the medicine was not registered in Serbia, but that its clinical trial was approved and studies were initiated including only patients who met the conditions in the subjects recruitment phase.
After learning from the media that children with spinal muscular atrophy have been waiting for the medicine "Risdiplam" for six months, which is also the only therapy that these children can receive, and that their condition has been worsening in the meantime, the Protector of Citizens initiated an own-initiative investigation to control work regularity and legality of the Child and Adolescent Neurology and Psychiatry Clinic, Republic Health Insurance Fund as well as the Medicines and Medical Devices Agency of Serbia.
The Protector of Citizens has ascertained that a request for the issuance of a license for the registration of a medicinal product of the mentioned brand name has not been submitted to the Medicines and Medical Devices Agency of Serbia until 02 November 2020, nor has a request been filed for the import of a medicine for which a permit in Serbia was not issued.
The Agency’s statement reads that they will be able to decide on the medicine in the event when an authorized legal entity or a representative office of a foreign manufacturer submits an application to the Agency for issuing licenses for the medicine and when the authorized importer, pursuant to the Health Institution’s recommendation, files a request for the import of an unregistered medicine through public acquisitions or donations, i.e. humanitarian aid.
The Agency adds that clinical trials for the mentioned medicine have been approved and studies have been launched, involving only those patients who met the conditions in the phase of recruiting the examinees, as well as that are no obstacles for the studies to continue.
According to the documentation submitted to the Protector of Citizens, only those subjects who meet the conditions for inclusion can be included in the clinical trial, which means that this type is not an optional availability for all the patients who would be interested in starting medicine therapy.