The State Expert Center, the authorized body for state health technology assessment (HTA), recommends considering the possibility of negotiating managed access agreements for the medicinal product nintedanib.
The relevant state HTA conclusion is published on the Center's website at: http://surl.li/tnxqo
Nintedanib is used for the treatment of mild to moderate idiopathic pulmonary fibrosis (IPF) in adults with a forced vital capacity (FVC) of ≥50% of the predicted value.
Disease priority: for which patients is it important?
Idiopathic pulmonary fibrosis is a chronic, rapidly progressive rare (orphan) disease characterized by gradual fibrosis of lung tissue. The International Society for the Treatment of IPF has characterized the disease as a specific form of chronic, progressive, fibrosing interstitial pneumonia of unknown cause, occurring predominantly in elderly people. By order of the Ministry of Health of Ukraine (of 27.10.2014 No. 778), idiopathic pulmonary fibrosis was included in the list of orphan diseases that lead to a shortened life expectancy of patients or their disability and for which recognized treatment methods exist.
About the medicinal product under assessment
The clinical efficacy and safety of nintedanib in IPF were studied in the randomized, double-blind, placebo-controlled INPULSIS trials. Nintedanib was superior to placebo in the annual rate of decline in forced vital capacity of the lungs in patients and in the time to the first exacerbation. The number of patients with at least one exacerbation was significantly lower in the nintedanib group – 4,9% compared with 7,6%. At the same time, no statistically significant effects of nintedanib were found on:
- on the adjusted mean change in SGRQ score at week 52 of treatment compared with baseline values (difference of -1,43 points)
- on mortality (5,4% compared with 7,80%).
UO recommendations
Taking into account a certain uncertainty regarding the expected clinical benefit of nintedanib, as well as the cost-ineffectiveness according to the recommended ICER threshold scale in Ukraine, it is recommended:
- consider the possibility of negotiating managed access agreements for the medicinal product nintedanib,
- further monitoring of real-world clinical practice data on the number of patients, clinical efficacy indicators (dynamics of forced vital capacity, time to exacerbation and number of exacerbations, mortality) and their analysis.
It is also necessary to develop and approve sectoral standards in the field of healthcare of Ukraine (clinical protocols and/or standards of medical care) that would regulate the provision of medical care in the treatment of IPF and take into account modern algorithms for the diagnosis, treatment, and monitoring of the condition of such patients.
