WHO Georgia representative says introduction of achondroplasia drug “long process”

Silviu Domente, the representative of the World Health Organisation in Georgia, on Wednesday said the introduction of Voxzogo (vosoritide), an experimental drug for treating achondroplasia, in many countries would be a “long process”, in comments on the backdrop of requests by parents in the country for the Government to introduce and finance the medication. 

In comments after a meeting of the Coordinating Council Working on Issues of Rare Diseases, hosted at the Georgian National Centre for Disease Control and Public Health, the WHO official said its participants discussed achondroplasia, a rare genetic condition related to bone growth complications, and its treatment.

The meeting once again demonstrated how complex the issue is, and how many questions need to be clarified. At the same time, clinical evidence for this remedy is very scarce”, Domente said in reference to the medicine that was approved in 2021 by the European Medicines Agency and the United States Food and Drug Administration. 

Pointing to procedures, he said the approval of the drug for use in Georgia required a consent of a selected group of health professionals based on clinical evidence and trials, noting that in some countries, the process involved pharmacoeconomic analysis and cost-effectiveness analysis and was a long process. 

He noted the subsequent stage involved approval of funding of the expensive drug by the Government, with the duration of the process also dependent on the country’s healthcare and budget systems. 

Parents of children with achondroplasia launched their protests last month. Photo: RFE/RL 

Even if approved for use and [if] the authorities also accept its funding, there is no guarantee that the drug will be immediately available locally and will become reimbursable”, the WHO representative said. 

Domente stressed the Georgian Government had taken “important steps” to ensure the “timely provision” of possible treatment, by launching the Council to study the drug and make a decision on its approval for use and funding, and starting talks with the producer, the details of which he said was being kept confidential at this stage. 

He also highlighted the Georgian authorities had offered “comprehensive medical checks” and “all available treatment” to the children suffering from the disease until the completion of mandatory procedures and the potential introduction of the drug.

Since it is a long process, the authorities and health professionals have expressed their opinion not to wait for the final decision. Today we have heard the Council has already started work to develop a protocol for such treatment and before the protocol is made available, the children will be offered all available services”, he noted, and added the offer covered surgical and hormonal treatments, as well as management of all possible complications.

Lali Margvelashvili, a member of the Council, said Georgia’s Health Ministry had requested the group to provide a guideline on treatment with the drug “as soon as possible”, with the deadline of late August. 

Georgian Prime Minister Irakli Garibashvili on Monday said the complication with ensuring access to the drug for children in the country was its potential side effects and not funding from his Government.

• Possible side effects, not funding, issue with innovative achondroplasia drug for children - PM

He also accused domestic opposition groups of inciting the parents’ protests and “using the topic for their narrow political interests”, calling the circumstances “immoral”. 

Based on various sources, achondroplasia occurs in one in 20,000-30,000 children worldwide. While no exact statistics have been released for Georgia, the parents have estimated the number of children with the illness to be between 20-30 in the country.

They also put the cost of an annual dose for each patient at ₾200,000 ($79,000), however Health Minister Zurab Azarahashvili cited the number of $200,000 last week.

Along with slowing bone growth, the disease can cause a number of health issues like curvature of the spine, shortness of breath, ear infections, narrowing of spinal canal or hydrocephalus in rare occasions.