In August 2023, the Ministry of Health removed Voxzogo, a drug used under the Emergency Access to Drug Technologies (RDTL) procedure, from the list of reimbursed drugs. This preparation was the only agent used in treating a rare genetic disease in children – achondroplasia. Without reimbursement, the cost of therapy for one sick child amounts to about 1.5 million zlotys annually. In response to this decision, a strike will take place in Warsaw on October 6, aimed at restoring hope for children’s access to treatment.
At the end of summer, the Ministry of Health deprived children suffering from achondroplasia of funding for the breakthrough Voxzogo therapy. The drug itself was added to the list of high-innovation technologies last year, indicating promising effects in the treatment of achondroplasia, which affects several dozen children in Poland each year.
People with achondroplasia not only struggle with short stature and disproportionate body structure, but also a host of neurological, cardiac, orthopedic, and laryngological complications. Additionally, every infection carries the risk of hearing loss, while every case of apnea carries the risk of death.
“Rehabilitation and brain MRIs are a daily reality for my 13-year-old child. As a parent, knowing that there is a drug available, I cannot calmly watch that in our country there is no possibility of reimbursement for treatment, which costs about 1.5 million zlotys annually and can only last until the patient’s growth plates close, so around 12-16 years old. Every day without the drug is a lost chance for a better life. I’m not just fighting for greater height, but for the health and life of my child. Widening of the cranial opening, improvement of cardiovascular, neurological, orthopedic conditions, these are just part of the effects of the Voxzogo therapy,” says protest organizer Renata Ostrowska.
Voxzogo reduces or completely eliminates complications associated with achondroplasia. Its effectiveness has been confirmed by the European Medicines Agency and thus approved for standard trade within the European Union. Voxzogo is reimbursed in many European countries including Germany, Czech Republic, Russia, Switzerland, France, Portugal, Austria, and Spain. It was also available in Poland under the RDTL procedure, a different form of public funding, but only until August this year.
“Voxzogo therapy allows our children to avoid surgical bone lengthening with Ilizarov apparatus, which involves immense pain and is fraught with the risk of complications leading to severe disability. I am fighting for the quickest possible way to fund Voxzogo treatment in Poland and ask the Ministry of Health to engage in discussions with the pharmaceutical company BioMarin so that the Voxzogo drug can be included in the reimbursement as quickly as possible,” says Olga Prokopek, mother of a 6-year-old Amelka suffering from achondroplasia.
During the demonstration, the families of the patients expect a discussion with a representative from the Ministry of Health. Without an alternative treatment option and with no response from the Ministry, they have been left with no choice.
The protest will take place on October 6, 2023, at 1:00 pm in Warsaw. The march will start at the Chancellery of the Prime Minister (Ujazdowskie Avenue 1/3) and end at the Ministry of Health building (Miodowa Street 15). All information is available at: https://fb.me/e/3dzWI4hCr
