The streets of Tbilisi were covered with umbrellas on a rainy Monday as parents of children with a rare genetic disorder and their supporters marched to demand that the state fund an expensive new drug to treat the condition.
About a dozen children in Georgia are known to be in need of the medication to treat achondroplasia, a disorder interfering with bone growth that leads to dwarfism, and also causing various health complications. Their desperate parents look with hope at Voxzogo, a recently developed drug approved by several major drug agencies, including in the U.S. and EU, and are asking the government to fund its import.
The authorities say they will only launch the process once they know more about the observed risks and effects of the drug in European countries. But parents say they are running out of time, patience, and trust that the government is truly committed to helping them.
"For 16 months, we have taken every step calmly and have been waiting for the Ministry of Health to start treating our children," one of the parents told RFE/RL's Georgian service on April 23. "Unfortunately, 16 months was not enough for them and they want us to keep waiting and waiting for them. There is no more time to lose, our children don't have this much time."
For about a week, the parents have been spending days and nights near the government chancellery asking for Prime Minister Irakli Garibashvili to personally meet them. Heart-rending images of mothers begging, breaking down in tears, and even fainting radiated from the scene. They say they have been asking for the drugs for over a year, and were let down over and over again by the authorities despite initial hopes of getting help.
Voxzogo -- generic name vosoritide -- was developed by the U.S.-based BioMarin Pharmaceutical to increase linear growth in children with achondroplasia. The medication secured accelerated approval from the U.S. Food and Drug Administration (FDA) and the EU Medicines Agency (EMA) in 2021. The annual cost of treatment with the drug per person is said to be as high as $200,000, an expense far out of reach for nearly all patients.
The drug is meant to be used from an early age and is effective only in children and teenagers with open bone growth plates, meaning it is of no use once the bones stop growing.
The parents therefore feel a sense of urgency to address their children's pain and discomfort.
But the government says they have to wait, arguing that despite international approval, the medication is still under observation and other countries have yet to start funding treatment with Voxzogo from their state budgets.
"We guarantee that if by September several EU member countries take responsibility and fund the newly developed medication to treat achondroplasia from their state budgets, Georgia will be among the frontrunner countries to share this responsibility and carry out necessary procedures to purchase the drug," Health Minister Zurab Azarashvili said during a meeting with parents on April 20.
Other ministry officials have reiterated these arguments and pledges, claiming they have to wait for more efficacy proofs and expert opinions before making a decision to fund the treatment.
The ministry says it is consulting the World Health Organization (WHO) and other relevant international bodies. Silviu Domente, WHO representative in Georgia, said that the body "for the time being didn't develop any recommendations regarding this medicine for the member states to include it in those funding schemes." He also said some European countries are still in the process of assessing the drug.
But the parents claim they have been hearing inconsistent arguments from the ministry, and believe that the authorities' reluctance is based on financial, rather than medical caution. The parents and their supporters also argue there are enough success stories on the effects of the medication, compared to the risks of side effects, to make them want immediate access for their children -- before it's too late.
On April 24, Prime Minister Garibashvili broke his silence to repeat the ministry's arguments and say that the money -- about $4 million annually -- is not the issue.
"This sum is not critical for us, but God forbid, if after giving this medication, the health condition of these children deteriorates and things take a turn for the worse," he said during a government meeting. "Of course, we would bear responsibility in such a case."
Garibashvili, while expressing sympathy with the parents, added fuel to the fire by repeatedly warning against the opposition "exploiting" their concerns for their own "political campaign" (some opposition politicians have attended the parents' rallies).
The government head, however, still refused to meet personally with the parents, leaving for Brussels immediately after the announcement.
In the meantime, the issue attracted wide attention in Georgia, and more groups went to express solidarity with the parents and children. Those convinced that the delay is caused by government hesitation over funding have suggested authorities cut non-essential spending such as the millions of dollars the state reportedly spent to organize big-name pop concerts this summer.
The controversy has also led to more public awareness about the challenges facing those with the disorder, as families report physical pain, at times life-threatening complications, and psychological trauma due to social stigmas. Parents have complained that the authorities are unfairly reducing their plight to concerns about their children's shorter stature.
"If finances are not the issue … we implore Mr. Prime Minister to meet us for half an hour. We know very well what we are asking for our children's health," one of the mothers said addressing Garibashvili after his statement.
Nini Gabritchidze is a Tbilisi-based journalist.