New Alliance Against Rare Diseases: CIS Creates Orphan Consortium

On June 21, in Moscow, as part of the First CIS Forum on Orphan Diseases, leading experts from Armenia, Belarus, Kazakhstan, Uzbekistan and the Kyrgyz Republic gathered to discuss current issues in the treatment of rare diseases. The forum was organized by the National Association of Experts on Rare Diseases (NAERD) with the support of the Russian Medical Branch of the Russian Academy of Sciences, the CIS Executive Committee, the National Medical Chamber and the Academician N.P. Bochkov Federal State Budgetary Scientific Institution "Medical Genetics Research Center".

One of the key achievements of the event was the decision to create the CIS Orphan Consortium. The consortium is necessary to consolidate the efforts of the CIS countries, exchange experience and knowledge, organize joint clinical trials, use real clinical practice data (RWE and RWD), and improve approaches to drug provision for patients with rare diseases.

The participants came to the common opinion that one of the key problems in our countries is the availability of innovative drugs, so it is necessary to pay special attention to the development of common approaches to optimizing the procurement of orphan drugs. Consolidation of procurement volumes between the CIS countries will reduce the cost of expensive drugs and increase their availability to patients. Such a step will help both save resources and strengthen the drug supply systems of the participating countries.

The next important area of ​​​​the consortium's activities should be cooperation in the field of collecting and analyzing real-world clinical practice data (RWE/RWD). These data are critically important for understanding the true clinical efficacy, safety and cost-effectiveness of orphan drugs, since these drugs are often registered with limited clinical data, which leads to high uncertainty when making decisions about their financing. Conducting cooperative studies to collect and analyze real-world clinical practice data reduces this uncertainty, allows you to adjust approaches to diagnosis and treatment, clinical guidelines and decisions on inclusion / non-inclusion in restrictive lists.

Thanks to the efforts of the forum and the creation of the consortium, the CIS countries will be able to strengthen their cooperation between the clinical community, healthcare organizers, non-profit organizations, including the patient community, and charitable foundations, which will contribute to improving the quality and accessibility of treatment, as well as the development of new technologies for the diagnosis and treatment of rare diseases.