On the creation of a national orphan program

“To improve the quality and accessibility of medical care for patients with rare diseases, it is necessary to form a national orphan program, Sergey Kutsev, Director of Medical Genetics Scientific Center named after the Academician N.P. Bochkov, believes.

At the meeting of the Expert Council on Public Health in the Federation Council, Sergey Kutsev, Director of Medical Genetics Scientific Center, chief freelance specialist of the Ministry of Healthcare for medical genetics, said that formation of the national orphan program would help to increase the quality and availability of medical care for patients with rare diseases.

"It seems to me that the time has come to create a national orphan program in state program 'Development of Health Care,' where all the efforts that are associated with both diagnosis and treatment of these patients are combined," S. Kutsev said.

According to him, it is necessary to develop criteria and procedures for selecting nosologies for centralized drug provision, to solve the issue of drug provision to patients over 18 years old, to create budget planning mechanisms, taking into account the market launch of new medicinal products, to expand neonatal screening and gradual "orphanization" of uncommon nosologies, and to organize the register of orphan diseases with the help of professional non-profit organizations. All these measures are proposed to be combined in the form of a national orphan program, which will help identify current problems. According to S. Kutsev, its creation will not require additional financial injections.

The initiative was supported by Vitaly Omelyanovsky, General Director of the Center for Expertise and Quality Control of Medical Care of the Ministry of Healthcare. According to him, so far there is no unified system that specifies the stages of inclusion of new medicinal products in the state registry, including the Circle of Goodness list.