Based on the results of the state health technology assessment (HTA), the State Expert Center of the Ministry of Health of Ukraine recommends including the medicinal product velmanase alfa in the lists of medicinal products procured with state funds using the managed access agreements (MAA) procedure.
Velmanase alfa is used as enzyme replacement therapy for patients with non-neurological manifestations of mild to moderate alpha-mannosidosis.
Alpha-mannosidosis is a rare orphan genetic disease. It occurs due to a deficiency of a certain enzyme (alpha-D-mannosidase), which leads to the accumulation of oligosaccharides in the blood. Subsequently, complications from the musculoskeletal system and respiratory organs may occur, and development may be delayed.
According to global statistics, the disease occurs in approximately 1 people per 500 thousand population.
Clinical trials have shown that treatment with velmanase alfa helps significantly reduce the level of oligosaccharides in the blood (on average by 70,5%) and improve functional indicators – endurance and lung function over a year of treatment.
Long-term observations (up to 12 years) have shown that in children treatment contributes to improvement or stabilization of the condition, while in adults it helps maintain the stability of the course of the disease. The most pronounced effect is observed in patients who started therapy as early as possible, which emphasizes the importance of early diagnosis of the disease and timely initiation of enzyme replacement therapy.
The recommendation to include the medicinal product in state procurement may contribute to expanding patients' access to the necessary treatment.
HTA conclusion at the link https://surl.li/dtqtyy
Background information
Managed access agreements are a tool that the state uses to ensure patients' access to new, innovative and expensive medicinal products. Its essence lies in conducting direct negotiations with manufacturers of original medicinal products and concluding special agreements with them. Thanks to the confidentiality of the terms, in particular regarding the cost and volumes of supply, the state can obtain such medicinal products at prices significantly lower than market prices. After the agreements are concluded, the medicinal products are procured with state funds, which allows patients to receive the necessary treatment free of charge.
