Parents of Children with Achondroplasia Demand Definitive Date for Drug Treatment
Parents of children suffering from achondroplasia, a rare genetic condition that may lead to dwarfism, are urging the Georgian Ministry of Health to provide a definitive date when the treatment with the novel medicine may commence. They have set a deadline of July 28 for the Ministry and threatened to resume their 24-hour protest otherwise.
Ministry’s position
On July 23, the Ministry of Health reported that experts developed a protocol for diagnosing and treating achondroplasia using the product ‘Vosoritide.’ The draft guideline was submitted to the National Council of Guidelines for examination and agreement on June 27.
According to the Ministry, during the council meeting on July 3, a tentative approval deadline for the updated protocol was announced for July 15-20. On July 19, the protocol group submitted the revised version to the Ministry after seeking advice from an international expert.
On the same day, written updates were provided to all members of the Coordination Council for Rare Diseases, including the parents. The Ministry emphasized that the parents were in constant phone contact with the responsible employee of the Ministry to stay informed about the progress, and they consistently attended the Coordinating Council for Rare Diseases meetings.
The Ministry clarified that a reasonable review period is necessary for the Guidelines Council to make an objective decision on the treatment protocol and that the Council members had until July 24 to establish their position. The Ministry explained that adherence to deadlines was intended to reduce risks associated with introducing a new medicine. They highlighted, that negotiations with “Biomarin”, the manufacturer of the medicine “Vosoritide” to finalize the contract terms for supplying the medicine were going on without impediment.
Parents worry
Despite the deadline set for the Council, the protocol hasn’t been approved yet, and a specific date for the start of treatment for the children has not been determined.
One of the parents, Makuna Gochiashvili, said: “In our country, doctors created a protocol for the diagnosis of achondroplasia from scratch, which is a very big step forward. In other countries, the drug was introduced without updating the protocol because it already existed. A month ago, the draft was almost finished, it was almost ready, and a month was quite enough for them to officially approve it. However, deadlines are constantly being pushed back, and we also feel that the process may drag out so much that the treatment becomes useless for some children [since it needs to be administered at a certain age to be effective]. Time is crucial for these children”.
The parents of children suffering from achondroplasia are urging the state to be more specific and transparent concerning the timing. They want an official confirmation that the children will receive the medicine no later than September 30. They emphasize that this deadline is crucial to alleviate constant worry about potential delays in responding to the pharmaceutical company. They also urge the Prime Minister, Irakli Garibashvili, “to engage, monitor and accelerate the process.”
At the end of April and beginning of May, parents of children with achondroplasia protested for 19 days in front of the government administration, demanding that appropriate drugs be imported into the country. The parents decided to end the protests after being promised that they would be allowed to attend weekly meetings at the Ministry of Health and be involved in the work to secure the drug.
Initially, government officials said they had no plans to import the drug, citing its experimental nature and side effects. The Prime Minister even urged parents not to allow politicians to use them. However, after continued protests, the sides reached an agreement, and the parents were promised that guidelines and protocols for treating achondroplasia would be developed in an accelerated manner.
Also Read:
- 24/05/2023 – Achondroplasia Added to State Rare Disease Treatment Program
- 06/05/2023 – Parents of Children Diagnosed with Achondroplasia End Round-the-Clock Protests
- 24/04/2023 – PM to Parents of Children with Achondroplasia: Do not Let Politicians Use You
- 21/04/2023 – Parents of Children with Achondroplasia Demand Meeting with Prime Minister
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