The creation of the BRICS Orphan Consortium was announced at the III CIS Orphan Forum
Health ministries of six CIS countries presented national care systems; regulators, industry, experts and patients discussed ways to accelerate access to orphan therapies
On May 25, 2026, the 3rd CIS Orphan Forum opened online — an annual international expert platform bringing together over 100 speakers from 21 countries of the Commonwealth, BRICS, the Middle East, Africa, Asia, Europe and Latin America. On the first day of the forum, two plenary sessions and a thematic session dedicated to the production, regulation and supply of orphan drugs were held.
The main news of the forum was the initiative to establish the BRICS Orphan Consortium — an international platform for expert interaction, experience exchange and joint practical initiatives in the field of rare diseases — presented publicly for the first time.
The forum took place exactly one year after the World Health Assembly adopted resolution WHA78.11, which established rare diseases as a global health priority. This date provided a symbolic context for the entire first day’s agenda.
Plenary Session 1. BRICS Cooperation
The forum was opened by Alexander Grigorievich Rumyantsev, President of the CIS Orphan Consortium, Academician of the Russian Academy of Sciences, President of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of the Russian Ministry of Health:
Alexander Grigorievich Rumyantsev noted the CIS Orphan Consortium’s accession to Rare Diseases International and emphasized the fundamental importance of genetic diagnostics, professional assessment of orphan technologies, and cooperation in scientific research among countries.
Greeting from the CIS Council
Elena Vladimirovna Shamal, Advisor to the Department for Cooperation in Political, Humanitarian and Social Spheres of the CIS Executive Committee, Secretary of the CIS Healthcare Cooperation Council, delivered a welcome address on behalf of the CIS Executive Committee and the CIS Healthcare Cooperation Council.
Elena Shamal noted that uniting the efforts of CIS and BRICS countries in discussing care for patients with orphan diseases is a dialogue where every country’s experience is valuable: Russia’s financial and regulatory approaches, the production of affordable generics and biosimilars, China’s experience in clinical trials, Brazil’s model of patient rights protection, the expansion of newborn screening in Belarus, the consolidation of orphan care in Kazakhstan, and the development of diagnostics from primary care in Uzbekistan.
Elena Shamal also emphasized that international cooperation will eventually allow consolidating efforts for joint procurement of medicines and the joint formation of pricing approaches, since price remains one of the key factors in the accessibility of orphan therapies.
Greeting from the Russian Ministry of Health
Anna Viktorovna Frolova, Deputy Director of the Department for Regulation of Medicines and Medical Devices, delivered a welcome address on behalf of the Russian Ministry of Health. The symbolic timing underscored the meeting’s special significance: exactly one year ago, on May 25, 2025, the World Health Assembly adopted the historic resolution WHA78.11.
Key Russian achievements in recent years:
• The orphan list includes more than 300 rare diseases.
• Over 5 years, more than 90 orphan drugs have been registered, 28 of which are domestically developed.
• 44 authorizations for clinical trials of orphan drugs have been issued, 29 for Russian developments.
• The registry for the 14 high-cost nosologies program includes more than 170,000 patients with orphan diseases.
• The “Circle of Good” foundation supports more than 30,000 children with over 100 rare diseases.
• Since 2026, a new co-financing mechanism for regions has been launched to provide for orphan patients.
WHO Global Perspective
Tarun Dua, Specialist, Neurological Disorders and Neurosciences Program, World Health Organization, presented the WHO position and the roadmap for implementing resolution WHA78.11: preparation of a technical report — discussion with member states in July 2027 — presentation of an action plan at the World Health Assembly in May 2028. The key principles for future work are a patient lifecycle approach, interdisciplinary interaction, and placing the person at the center of healthcare.
Voice of the Patient Community: Rare Diseases International
Sara Brambilla, Global Program Manager of Rare Diseases International (RDI), presented the work of the global alliance of patient organizations — the voice of the patient community in shaping the global agenda for rare diseases. RDI works with more than 30 partners, pooling resources, knowledge and expertise to implement joint programs.
For 2026, RDI focuses on two key strategic objectives: establishing rare diseases as a full-fledged priority of healthcare systems, and ensuring that the needs of patients and their families are considered in all global discussions. A separate area is supporting the implementation of resolution WHA78.11: data collection, formation of working groups, and a structured approach to taking regional priorities into account.
Global Rare Disease Network. The experience in treating rare diseases is distributed extremely unevenly across the world: some countries have accumulated significant specialized knowledge, while in others it is difficult for specialists to access the necessary information. RDI’s program maps existing global resources and expertise and develops mechanisms for cross-border clinical cooperation and common patient routing.
Registries and a Minimum Data Set. RDI is working to enable all countries, including those with limited resources, to participate in creating registries. One tool is defining a minimum data set that can serve as a basis for a registry in any national system.
BRICS Orphan Consortium Initiative
Nuriya Zagitovna Musina, Executive Director of the CIS Orphan Consortium, presented three new strategic initiatives:
• BRICS Orphan Consortium — an expert network to harmonize approaches, conduct joint research, exchange evidence, and reduce the cost of orphan therapies in BRICS countries.
• Industrial Expert Council under the CIS Orphan Consortium — a permanent channel for interaction with manufacturers, developers, regulators, and payers.
• Orphan Innovation Platform — to advance academic developments for rare and ultra-rare diseases toward clinical application.
The CIS Orphan Consortium unites 31 organizations from 8 Commonwealth countries; in 2026, the Consortium joined Rare Diseases International — a global alliance of organizations in the field of rare diseases.
Experience of BRICS Countries
Professor Zhang Shuyang, Director of the Rare Disease Center of Peking Union Medical College Hospital (PUMCH), presented the “PUMCH model” — an integrated interdisciplinary care system. The average time to diagnosis in China has been reduced from four years to less than four weeks. In 2024, China’s first independent specialized inpatient rare disease unit opened: among patients admitted without a diagnosis, 60% received one during hospitalization. The national registration platform unites more than 100 institutions and contains data on more than 100,000 cases across 218 rare diseases; the biobank includes more than 310,000 biological samples. Since February 2025, an AI model for rare diseases has been applied in collaboration with the Chinese Academy of Sciences.
Ayman El-Khattab (UAE), Ali Akbarisari (Iran), and Kelly du Plessis (South Africa) also presented their countries’ experiences and supported the BRICS Orphan Consortium initiative.
Plenary Session 2. National Care Systems in CIS Countries
The session was moderated by Alexander Grigorievich Rumyantsev and Tamara Uktamovna Aripova, Academician of the Academy of Sciences of the Republic of Uzbekistan, Director of the Institute of Immunology and Human Genomics of the Academy of Sciences of the Republic of Uzbekistan.
Six CIS countries presented their national approaches to organizing care.
Russia
Rafael Firnyalovich Shavaliev, Director of the Department of Medical Care for Children, Obstetric Services and Public Health of the Russian Ministry of Health, reported that the federal registry of rare diseases includes 26,326 patients (of whom 11,222 are minors). Prenatal screening covers 96.6% of pregnant women; in 2025, under the expanded newborn screening program, more than 1.16 million newborns were examined, and 1,128 children with congenital and hereditary diseases were identified. 191 clinical guidelines for rare diseases have been approved.
Uzbekistan
Dilorom Ilkhamovna Akhmedova, Chief Pediatrician of the Republic of Uzbekistan, reported that 35.4% of the country’s population are children. Prenatal and neonatal screening for phenylketonuria and congenital hypothyroidism has been conducted since 1996; neonatal screening for cystic fibrosis was launched in 2025. The new state program for 2026–2030 increases the number of orphan diseases covered by the budget from 5 to 10 nosologies (adding primary immunodeficiencies, Duchenne muscular dystrophy, Gaucher and Pompe diseases). The age limit has been extended from 18 to 21 years. In September 2026, Samarkand will host a regional forum on orphan diseases.
Kazakhstan
Guldey Turekhanovna Begalieva, Department of Maternal and Child Health of the Ministry of Health of the Republic of Kazakhstan, spoke about a model unique to the region: the country has appointed a Commissioner for Rare Diseases and established a central orphan center; regional orphan centers are in the process of being formed. Treatment decisions are made by multidisciplinary teams and submitted to the Ministry of Health’s expert council. Since 2026, the procurement of orphan drugs has been centralized through a single distributor.
Belarus
Irina Vladimirovna Zhevneronok, Chief Freelance Specialist for Hereditary Neuromuscular Diseases in Children of the Ministry of Health of the Republic of Belarus, reported that newborn screening in the country has been conducted since 1978; expansion to 36 nosologies is planned by 2030. The registry of patients with neuromuscular diseases has grown from 280 to 568 people in five years. In 2025, 34 private sector organizations transferred more than 7.5 million Belarusian rubles (about 2.5 million US dollars) for the treatment of children with SMA.
Kyrgyzstan
Nurgul Saparkulovna Ibraeva, Head of Department of the Ministry of Health of the Kyrgyz Republic, reported that the criterion for an orphan disease is no more than 5 cases per 100,000 population. As part of digitalization, a national registry of orphan diseases is being created; the treatment of SMA alone costs more than 1 billion soms from the high-tech fund.
Armenia
Natella Klimentovna Kostandyan, Geneticist at the Center for Medical Genetics and Primary Health Care, reported that thanks to early detection and treatment of periodic disease (familial Mediterranean fever), there have been no new cases of amyloidosis in the country in the last 20 years. Since June 2025, Armenia has been conducting non-invasive prenatal testing independently.
Summing up the session, Alexander Grigorievich Rumyantsev noted:
The gold standard example, he said, is hemophilia: over 20 years of systematic government work (since 2006), the life expectancy of patients with hemophilia A and B in Russia has reached that of the general population. Alexander Grigorievich presented the “Orphascope” information resource for physicians, which can be adapted to the language of any Commonwealth country.
Session 1. Production, Regulation and Drug Supply
The final session of the first day brought together representatives of government authorities, industry, the expert community, international organizations and patient associations. The central theme was how to shorten the path from scientific development to actual patient access to therapy.
The session was opened by Alexey Leonidovich Kedrin, Chairman of the Board of the Association of Pharmaceutical Manufacturers of the Eurasian Economic Union (APM EEU):
Anna Igorevna Lakhtanova, Chair of the Medicines Regulation Committee of the Association of International Pharmaceutical Manufacturers (AIPM), presented proposals for developing common approaches to defining orphan drug status. The key point is separating the regulatory recognition of orphan status from decisions on drug supply financing.
Marina Ivanovna Durmanova, President of the Association for Support and Development of Pharmaceutical Activities of the Republic of Kazakhstan, presented changes in the system of access to orphan therapy: lowering the orphan disease criterion, use of unregistered drugs through clinical protocols, accelerated registration via a “single window” mechanism, and the development of centralized procurement.
Kamila Zarubina, Managing Director for Science and Technology in Medicine and Biotechnologies at the Skolkovo Foundation, presented international experience in supporting biomedical development — specialized investment funds, venture philanthropy, and the participation of patient organizations in advancing research.
Emilia Ruan, International Science and Technology Cooperation Center NORXIN, spoke about the experience of international scientific and technological cooperation — developing research networks, accelerated mechanisms for bringing innovative drugs to market, and collaboration between countries in the field of rare diseases.
Daniil Sergeevich Talyansky, CEO of Generium JSC, presented the experience of full-cycle orphan drug development — from scientific research to clinical implementation — with a special focus on developing gene therapy and creating domestic technology platforms.
Alexey Alexandrovich Fedorov, Expert at the All-Russian Union of Patients, emphasized the importance of patient community participation in discussions regarding therapy accessibility.
Concluding the discussion, Nuriya Zagitovna Musina noted:
Day 1 Summary
Day 1 of the 3rd CIS Orphan Forum brought together three levels of the agenda: global (WHO, RDI, the BRICS Orphan Consortium initiative), regional (national systems of six CIS countries), and operational (production, regulation and drug supply). The main cross-cutting themes were the need for international cooperation among Commonwealth and BRICS countries, accelerating the patient journey from diagnosis to therapy, and transitioning from experience exchange to practical joint initiatives.
