A project for cooperative research and collection of real-world clinical practice data in the treatment of rare diseases.

The CIS Orphan Consortium project on conducting cooperative studies and collecting real-life clinical practice data aims to create a scientific basis for improving the diagnosis, treatment and management of rare diseases in the countries of the region. This project unites the efforts of experts, medical institutions and patient organizations to obtain reliable information on real-life clinical practice, the use of drugs and treatment outcomes.

The collection and analysis of real-life clinical practice data, as well as the cooperative studies within which they are conducted, are of key importance, since they provide valuable additional information not only on the clinical efficacy and safety, but also on the cost-effectiveness of orphan drug therapy. These drugs are often registered in conditions of limited clinical data, which is due to objective reasons such as the rarity of the disease and the small number of patients for traditional clinical trials. In such conditions, real-life clinical practice data become an indispensable tool for reducing uncertainty and improving the validity of decisions in the field of financing the treatment of rare diseases.

Clinical uncertainty combined with the high cost of orphan drugs poses a significant risk of ineffective spending when making decisions about their financing. Analysis of real clinical practice data allows not only to reduce this uncertainty, but also to adjust decisions already made, increasing their effectiveness. In addition, these data play an important role in revising clinical approaches and recommendations, which contributes to the continuous improvement of treatment standards.

Cooperation within the CIS provides a unique opportunity to significantly expand the scope of knowledge about orphan drugs. Combining efforts and exchanging data between countries in the region allows us to take into account the diversity of clinical experience and generate a more complete picture of the therapeutic and economic aspects of treating rare diseases. This contributes not only to improving the quality of medical care, but also to the development of more sustainable strategies for managing orphan diseases.

We invite medical institutions, research centers, pharmaceutical companies and patient organizations to cooperate. By working together, we can create conditions for sustainable development of the system of care for patients with rare diseases, improving their quality of life and improving the healthcare system in the CIS countries.