Kazakhstan parents urge ministry to keep rare disease drug in list
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Parents of children with neurofibromatosis type I in Kazakhstan are urging the Health Ministry to keep the drug Selumetinib (Koselugo) in the state list of orphan disease medications. The ministry has proposed excluding the drug due to low evidence levels, but parents say it is the only effective therapy for dozens of children. The draft order is open for public discussion until July 22.
The Proposed Exclusion
The Kazakh Health Ministry published a draft order revising the list of orphan diseases and their medications, citing a prevalence criterion of no more than 10 per 100,000 population. Selumetinib and other drugs are proposed for exclusion due to low evidence levels, according to the ministry. The ministry claims that the volume of medical care for patients will not be limited and will be preserved in full.
Parental Concerns
Saltanat Makhambetova, co-founder of the Zhan Zharygy foundation, said that over 100 people in Kazakhstan have confirmed NF1 diagnosis, but only 66 children currently receive therapy. She warned that if the order takes effect, local health departments will lack funding grounds to provide the drug. In the Abai region, three children have not received a single package of the drug since 2024 despite the current list still including it.
Funding and Appeals
Previously, the Republican Charitable Foundation partially subsidized drug purchases, but in 2025 it announced budget optimization and stopped funding orphan patients, shifting responsibility to local executive bodies. Parents have sent official appeals to the Health Ministry and the UNICEF office in Kazakhstan demanding the preservation of current treatment guarantees. They note that most families cannot afford the treatment independently.
What's Next
The public discussion period ends on July 22, after which the ministry may finalize the order. It remains unclear whether the ministry will revise its decision in response to parental appeals and whether alternative funding sources will be secured.
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Kazakhstan parents urge ministry to keep rare disease drug in list
