At the end of 2022, several important changes were made to create a system for the treatment of orphan patients. In particular, the Law of Ukraine on Managed Access Contracts was adopted, authorizing the procurement of drugs at a reduced price. At the end of the year, Medical Procurement of Ukraine for the first time purchased expensive drugs for patients suffering from a rare disease – spinal muscular atrophy (SMA).
Children from Khmelnytskyi and Chernivtsi oblasts who were diagnosed with CMA type 1 have already received treatment with the innovative drug purchased by Medical Procurement of Ukraine at the expense of the state budget under the mechanism of managed access contracts. The course of treatment is ongoing and it offers the prospect of alleviating or leveling the symptoms of the disease.
SMA was detected thanks to the introduction of expanded neonatal screening, which was launched in Ukraine. Thus, since the fall, newborn babies have been tested for 21 orphan diseases free of charge. Early diagnosis of rare diseases allows to start treatment of children as early as possible – even before symptoms appear – which ensures effective treatment.
During this time, more than 51 thousand tests were conducted on the basis of regional neonatal screening centers, of which more than 28 thousand by the Lviv Regional Center and about 23 thousand by the Kyiv Regional Center. These are modern high-tech laboratories equipped with the latest equipment and laboratory information systems. There were 73 confirmed positive results.
Also among the improvements the Ministry of Health is currently working on is the formation of a network of reference centers for rare (orphan) diseases to ensure coordination and improve the quality of medical care for patients suffering from such diseases, as well as attracting the most qualified specialists to provide them with medical care.
