Results of the session "Legal and regulatory space for rare diseases: challenges, approaches, prospects" II CIS Orphan Forum | Moscow, June 27, 2025
The session is devoted to one of the key issues in the development of care for patients with rare diseases — the formation of a coordinated legal and regulatory framework in the CIS and EAEU countries.
Moderators:- Sergey Ivanovich Savashinsky, Deputy Chairman of the Working Group on the Development of Principles of Bioethics and Evidence-Based Medicine of the Expert Council on Healthcare under the CIS IPA, Director of the International Non-Commercial Organization “Eurasian Cooperation on Clinical Guidelines and National Health Development”
- Nuriya Zagitovna Musina, Executive Director of the CIS Orphan Consortium, Director of International Cooperation at NAERES
The focus is on differences in definitions of orphan status, the lack of coordinated approaches to registration and evaluation of drugs — this leads to unequal access of patients to innovative therapy and hinders interstate cooperation at the post-registration stages.
The main topic of discussion was the need to harmonize approaches within the EAEU and the CIS.
Today, registration of medicinal products in the EAEU countries is carried out according to uniform rules, however, orphan status is determined at the national level, which:
- limits the possibility of submitting a dossier for registration in individual countries;
- impedes the implementation of such initiatives as joint clinical evaluation and joint price negotiations.
The session participants, including representatives of regulators, expert and international organizations, discussed the prospects for developing a model law on drug provision in the CIS, which could enshrine:
- uniform approaches to determining orphan status;
- registration procedures;
- post-registration monitoring;
- state support measures.
Among the key proposals is to initiate a common procedure for assigning orphan status to medicinal products at the level of The EAEU through the Expert Committee, similar to the mechanism for recognizing drugs of “special importance.”
This would eliminate current barriers and speed up patient access to therapy.
The session showed high interest in issues of legal coordination and emphasized the importance of interstate dialogue to improve the sustainability and fairness of healthcare systems in relation to orphan diseases.
