Based on the results of the state HTA, the Center recommends considering the possibility of providing access to risdiplam for the treatment of patients with the orphan disease spinal muscular atrophy type II and non-ambulatory type III.
The full text of the conclusion of the state HTA is published on the Center’s website http://surl.li/tidcv
Disease priority: for which patients is it important?
Spinal muscular atrophy (SMA) belongs to the list of rare (orphan) diseases that lead to reduced life expectancy and disability of patients. It is characterized by damage to the motor neurons of the spinal cord and impairment of the functions of various organs and systems.
Providing patients with SMA with the necessary treatment is included in the Action Plan for the implementation of the Concept for the Development of the System of Medical Care for Patients Suffering from Rare (Orphan) Diseases for the years 2021-2026, approved by the Government.
About the medicinal product under assessment
The health technology assessment (HTA) that was conducted established the clinical benefits of risdiplam compared with best supportive care: after just one year of treatment it makes it possible to stabilize and improve the motor function of patients.
Risdiplam has already proven its efficacy. In 2022, based on the results of the HTA conducted and the managed entry agreements concluded, the state was able to purchase and provide maintenance therapy with risdiplam to young patients with SMA type I.
Recommendations of the Authorized Body
Based on the results of the state HTA conducted, the Center recommends considering the possibility of providing patients with risdiplam for the treatment of treatment-naive patients with type II and non-ambulatory type III spinal muscular atrophy, using the procedure of negotiating managed access agreements, taking into account the conditions specified in the conclusion, with subsequent monitoring of data on the number of patients and data on the efficacy and safety of risdiplam in real clinical practice in Ukraine.
