Opinion: 'Funding treatment for Duchenne muscular dystrophy in Georgia is a matter of priorities'
Duchenne muscular dystrophy in Georgia
Former Georgian health minister Andria Urushadze commented on the treatment of children with Duchenne muscular dystrophy.
According to him, there are approximately 60–80 people in Georgia with this diagnosis, and the estimated state budget required for their treatment amounts to around 30–35 million lari ($11–13 million) per year. He added that if the government manages to implement regional procurement, these costs could be reduced further.
For several months, parents of children with Duchenne muscular dystrophy in Georgia have been holding protests outside the government administration, demanding access to the necessary medications.
Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterised by muscle wasting and weakness. According to the organization “Together Against Duchenne Muscular Dystrophy,” around 100 children in Georgia have been diagnosed with the condition. Due to the lack of adequate medication and treatment in the country, 1–3 children die from the disease each year.
Andria Urushadze: “It is difficult to remain indifferent to families demanding funding for medicines to treat Duchenne muscular dystrophy.
What response should the Ministry of Health give? In my view, treating these children in Georgia is not a question of finances, but of priorities. By allocating just over 1% of the budget, the state can save the lives of dozens of children and protect them from severe disability. This claim is based on information available to me, which I will share with you.
Duchenne muscular dystrophy (DMD) is a severe genetic disorder that causes progressive muscle wasting. The disease primarily affects boys. Due to a lack of the protein dystrophin, muscles gradually lose their function, ultimately leading to loss of mobility as well as respiratory or cardiac failure. In Georgia, approximately 60–80 people are living with this diagnosis.
Estimates that around 60–80 patients require the medication are based on three main sources:”
- Duchenne muscular dystrophy is a rare condition, but its global prevalence is relatively stable. Statistically, it affects one in every 3,500–5,000 live-born boys. In Georgia, the average number of male births in recent years has been around 20,000 per year. Applying this prevalence rate to the potential patient age group (0–20 years), it can be estimated that there are likely between 60 and 80 patients in the country.
- A patient organisation in Georgia — the Duchenne Foundation — maintains an internal registry. It brings together parents of children with confirmed diagnoses, and the number of patients currently registered in its database falls within this same range.
- The Georgian Ministry of Health and Social Protection also holds data on patients enrolled in the state programme for the management of rare diseases. Some patients are included in government sub-programmes for rehabilitation or supportive care, and these figures likewise point to a similar number of patients.
Why are these figures important? Knowing the exact number of patients allows the state to plan its budget. Eighty patients is the “upper threshold” used to calculate the maximum possible cost, so that no child is left without treatment. For a more precise answer, the ministry should maintain a single electronic register of such patients.
As for treatment, US (FDA) and European (EMA) regulators have recently made several groundbreaking decisions:
- Givinostat / Duvyzat: A new drug approved by the US Food and Drug Administration (FDA) in March 2024, while the European Medicines Agency (EMA) granted marketing authorisation in June 2025. It is the first non-steroidal medicine intended for patients aged six and above with all mutation types.
- Vamorolone / Agamree: Approved in the US in October 2023 and in Europe later the same year. It is the latest alternative to steroids, designed to minimise such serious side effects as growth delay and bone weakening.
- Elevidys / gene therapy: Approved in the US in 2023–2024. It is a one-off injection that delivers a gene to help maintain muscle function. It is funded only in the United States.
Among European countries, Italy, Spain and Scotland have already decided to fund treatment with Givinostat, while the process is under consideration in the UK, Ireland and Germany. Poland, for example, is part of a Givinostat distribution agreement in Central and Eastern Europe, alongside Romania, the Czech Republic, Hungary, Bulgaria, Slovakia, the Baltic states, Croatia and Slovenia. This agreement lays the groundwork for future availability and procurement at a price below the listed one, although it does not in itself mean automatic state funding.
As for Vamorolone, in most countries it is either already funded — including Germany, Austria and the UK — or is at the final stage of review. In many countries, before universal funding is introduced, an individual funding model is used for specific patients. Notably, European countries use different strategies to ensure access to medicines. Italy, for instance, is globally known for its “payment by results” model, under which the state pays the manufacturer only if the medicine proves effective for a particular patient.
Budget calculations for Georgia: Treating 80 patients in Georgia would cost around 30–35 million lari ($11–13 million) per year. This figure is not merely a theoretical assumption; an accurate calculation must take into account the average weight of patients and the dosage required, which is approximately 75–80 vials per patient per year.
However, it is important to understand that the state usually does not pay the officially listed price for a medicine, and it is realistic to factor in a discount of around 35–40% that manufacturers offer to countries such as Georgia. On that basis, the annual cost per patient falls on average to 420,000 lari [$12,4 million].
Impact on the budget:
- Within the medical programmes budget (2.22 billion lari / $820 million) this amounts to just 1.58%;
- Relative to total government spending: approximately 0.11%.
Georgia’s healthcare budget in 2026 increased by 200 million lari ($74 million). Only 17.5% of this increase would be enough to fully cover treatment for all patients with Duchenne muscular dystrophy. These costs could be reduced further if regional procurement is achieved. Pooling demand with neighbouring countries and purchasing in bulk would likely force manufacturers to lower prices even more. In addition, introducing a “payment by results” model would protect the state from ineffective spending.
All of this requires cooperation, public awareness, and, most importantly, respect for and support of the families for whom resolving this issue is a matter of survival.
Duchenne muscular dystrophy in Georgia
