Experts spoke about the problem of accessibility of medicines in the regions

On April 23, a round table on "Rare adults: a direct public conversation" was held at the Izvestia press center. During a round table organized by the House of Rare, experts, doctors and patients discussed key issues of drug provision for adults with orphan diseases in Russia. The participants shared their personal experiences and talked about the difficulties faced by "rare" patients in the regions.

A patient diagnosed with Paroxysmal nocturnal hemoglobinuria (APH), Alexey Nesterov, said that he had been transferred to another, less effective drug.: "The availability of therapy for APG is not just a matter of quality of life, but a choice between life and death. Even small interruptions in treatment can lead to an exacerbation of the disease. I managed to participate in a program on a new drug. This made it possible to improve the condition and reduce hospital visits. We wish affordable and stable provision of medicines and thank the doctors and everyone involved for their efforts."

Cardiologist, Associate Professor of MONICA's Department of General Medical Practice, PhD Olga Dvorina emphasized the importance of proper routing for "rare" patients: "For patients with orphan diseases, correct routing is critically important, in addition to medical care. Often, the appointment of therapy for such patients takes place in federal centers, and further management is carried out by regional services. Therefore, local procedures for providing medical care are necessary. Using the example of pulmonary arterial hypertension, continuity and interaction of cardiology, pulmonology and rheumatology services are important. Given the high risks of disability and mortality in patients with orphan diseases, it is necessary to regularly evaluate the effectiveness of the therapy used and escalate it in accordance with current clinical recommendations."

Kirill Kulyaev, an expert in medical law, drew attention to the need to reform the mechanism of financial assistance to the regions:
"Effective, affordable and stable drug provision is a key element of quality medical care. However, regions often face difficulties in providing medical care for rare patients.
The ruling of the Constitutional Court obliged the authorities to develop an additional mechanism for financial assistance to the regions. In order for this solution to be implemented as efficiently as possible, it is necessary:

• To develop criteria for allocating subventions from the federal budget, taking into account the opinions of regions and patient organizations, in order to solve real problems.;
• Expand the categories of diseases that will be covered by subventions, including them in the list approved by Government Decree No. 403. ;
• to organize centralized purchases, which will reduce drug prices and enable regions to redistribute them."

Anastasia Tatarnikova, Chairman of the House of Rare, summed up the meeting: "It is necessary to build a system for evaluating the effectiveness of therapy throughout life. This requires continuity of treatment between the pediatric and adult parts of the medical care system. And we need a single register. The result, in addition to the quality of medical care, will be a more rational planning of the need for expensive drugs and medical technologies."