The III CIS Orphan Forum has come to an end: the Commonwealth countries are entering into cooperation with BRICS

Published 26 May 2026

Last update 28 May 2026

May 26, 2026. Results of Day 2 of the 3rd CIS Orphan Forum: Key Sessions in Three Parallel Tracks

On May 26, 2026, the 3rd CIS Orphan Forum concluded. The second day’s program ran in three parallel tracks — 11 thematic sessions were held at the intersection of health policy, innovative therapy, and clinical practice. Detailed analyses of individual sessions, including nosological ones, will be published separately. This article provides an overview of the key sessions that set the thematic framework for the day.

Gene and Cell Technologies

The session showed that CIS countries already have developments in gene and cell technologies for rare diseases, and their implementation depends primarily on funding and regulation. In Russia, a major step forward has been taken — a high-tech medical care tariff has been established, and scaling of the technology is now expected. For CIS countries, the academic production of CAR-T remains the most acceptable model due to greater cost affordability.

Mikhail Aleksandrovich Maschan, Deputy Director for Science at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of the Russian Ministry of Health, reported that Russia has developed a network of more than 10 academic CAR-T programs. At the NMRC, about 200 patients have received therapy since 2018. The first global indication for CAR-T therapy was an orphan disease — in 2017, the first product was registered in the USA for children with refractory acute lymphoblastic leukemia.

“The distributed academic model, which is dominant and working, allows us to deliver therapy to patients quickly and with an acceptable financial and economic framework. The key limitation, in my view, is not scientific but regulatory. Without a regulatory framework for early clinical trials, it will be very difficult for us to move forward.”

The experience of Belarus was shared by Natalya Evgenievna Konoplya (N.N. Alexandrov Republican Scientific and Practical Center for Pediatric Oncology and Medical Radiology): since June 2021, 94 patients with various types of lymphoma have received CAR-T therapy. Overall complete response at day 30 was 61.7%; for follicular lymphoma — 85.7%.

The infrastructure of Uzbekistan was presented by Gleb Olegovich Bronin (Scientific and Practical Medical Center for Pediatric Oncology, Hematology and Immunology, Tashkent): the center receives 2,312 primary patients per year, a 10-year national plan for childhood cancer has been adopted, and a laboratory for CAR-T cell production is planned to open by the beginning of next year.

A model of individualized gene therapy drugs was presented by Denis Vladimirovich Rebrikov (Pirogov Russian National Research Medical University): for loss-of-function hereditary diseases in Russia, there are potentially about 6,000 patients per year across 300 nosologies; under-construction GMP facilities will be able to produce up to 100 individualized gene therapy drugs per year each.

The real scale of the gap between development and implementation was highlighted by Alexander Sergeevich Malogolovkin (Sechenov First Moscow State Medical University):

“Despite progress in individualized, targeted and replacement gene therapy, about 70–75% of developments are still in the preclinical stage. That is, not all developments reach clinical application.”

“When we actually have ultra-orphan technologies, individualized, and no single country can handle this alone, we need to bring this to the BRICS level, to a larger patient population, to make it acceptable for our countries,” — Nuriya Musina.

Health Technology Assessment (HTA)

The session brought together experts from 7 BRICS countries — Brazil, China, UAE, South Africa, India, Iran and Russia — and served as a direct illustration of the forum’s main theme. The key practical outcome: participants agreed to continue joint work within BRICS to improve HTA methodologies, horizon scanning, prepare joint publications and conduct joint training seminars.

Opening the session, Nuriya Zagitovna Musina identified the central methodological problem:

“Traditional HTA methods for orphan drugs do not always work well. Most countries often require broader coverage, larger patient populations that do not exist.”

Everton Nunes da Silva (National Institute of Cardiology, Brazil) presented the experience of the national CONITEC commission: about 50 drugs are analyzed annually in the country, with a decision time of 6–9 months. For orphan and ultra-rare diseases, a more flexible review of evidence is allowed.

Yinyao Chen (School of Public Health, Fudan University, China) spoke about the multi-level medical insurance system: in 2018, 121 rare diseases were included in the national catalog; by 2023, that number had reached 207; more than 100 drugs for rare diseases are included in the reimbursement system.

Kefa Al Kawasme (Department of Health Abu Dhabi, UAE) reported that a separate health technology assessment unit was officially established in 2025. For high-cost technologies, a threshold has been set — more than 100,000 dirhams per patient or more than 10 million dirhams per population. In the next 10 years, about 60 new gene therapy drugs for rare diseases are expected.

“Rare diseases really deserve their own, dedicated pathway for HTA assessment,” — Kefa Al Kawasme.

The system of financing orphan drugs in Russia was presented by Dmitry Georgievich Shchurov, Head of the Horizon Scanning Center at Sechenov First Moscow State Medical University. Experts from South Africa, India and Iran also presented the experience of their countries.

“In the case of rare diseases, no country can work in isolation from others,” — Dmitry Shchurov.

Orphan Centers and Patient Routing

The session showed that CIS countries are moving from fragmented management of patients with rare diseases to the creation of specialized centers, registries, regional coordinators and clear pathways from suspicion of diagnosis to treatment and follow-up.

An important conclusion of the session: many national republican clinics are not formally “orphan centers,” but in practice they perform their functions — concentrating expert diagnostics, patient routing, multidisciplinary follow-up and coordination of drug supply. The development of these institutions as full-fledged orphan centers is one of the key tasks of the regional agenda.

Antonina Alikovna Pak (National Children’s Medical Center of Uzbekistan) reported that the first orphan disease center was created in 2024. At the time of signing the 2026–2030 state program, 2,174 children were already being provided with medicines and therapeutic nutrition from the budget.

In Kazakhstan, according to the electronic registry, there are 87,716 patients with orphan diseases (73,038 adults and 14,678 children); since 2020, the number of registered patients has more than doubled. In Belarus, the registry of patients with neuromuscular diseases grew from 280 to nearly 600 in five years. In Kyrgyzstan, patients with 41 orphan nosologies are seen at the National Center for Maternal and Child Health; a total of 416 children.

“If earlier the main problem of rare diseases concerned diagnosis, now with the introduction of expanded newborn screening, the provision of medical care and follow-up of patients with these rare diagnoses comes to the fore,” — Andrey Petrovich Prodeus, Chief Pediatric Allergist-Immunologist of the Moscow Region Ministry of Health.

“One of the key trends is the transition from palliative care to early pathogenetic therapy, including gene and enzyme replacement therapy, with socially oriented budget financing,” — Irina Vladimirovna Zhevneronok.

Registries of Rare Diseases

Registries, observational studies and digital platforms were considered in the session as a full-fledged digital infrastructure for rare diseases — for clinical practice, pharmacovigilance, therapy effectiveness assessment and management decision-making.

The session was opened by Alexey Sergeevich Kolbin, Head of the Department of Clinical Pharmacology and Evidence-Based Medicine at Pavlov First Saint Petersburg State Medical University. Ravil Rashidovich Niyazov presented the European federated network DARWIN EU, combining data on about 250 million patients and about 40 partners. Mikhail Alexandrovich Getman (HSE University) spoke about the voluntary Russian-language DARNIS registry.

Evgeniya Valerievna Nazarova, Executive Director of NAEHRES, reported on five observational studies launched in 2025–2026: on hemophilia and von Willebrand disease, SMA in adults and children, neuroblastoma and cystic fibrosis. The study on hemophilia and von Willebrand disease has already enrolled 457 patients against a plan of 400.

“Orphan diseases — they are all like separate islands, each with its own genetics, its own clinical picture, its own treatment. But all these islands are still united by the same problems — underdiagnosis, lack of epidemiological data, problems with drug supply, lack of long-term results of expensive treatment.”

Tatyana Viktorovna Shamanskaya (Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology) presented the National Registry for Neuroblastoma: the first patient was included on April 14, 2026; the target is 350 patients. Alexander Viktorovich Ivanov (Aston Health) spoke about the orphan audit, which currently includes about 20 nosologies and more than 20,000 patients.

RWD/RWE — Real-World Data and Real-World Evidence

The final session of the forum showed how real-world data (RWD) and real-world evidence (RWE) are used in the system of care for patients with rare diseases — from diagnosis and follow-up to assessing therapy effectiveness, drug supply, HTA and management decision-making.

Opening the session, Alexey Sergeevich Kolbin formulated the main methodological conclusion:

“For RWD to become evidence, to become RWE, it is necessary to ensure the quality of real-world studies through regulatory framework, a single reproducible methodology for all participants in these studies, and the development of quality standards.”

Country experiences were shared by David Danko (Hungary) — on approaches to financing orphan drugs in Europe; Frederico Sallum (Brazil) — on the role of RWD in reducing uncertainty in HTA; Polina Alekseevna Mukhortova (Federal Center for Healthcare Quality Assessment and Control of the Russian Ministry of Health) — on the development of parameters for assessing the condition of children receiving therapy from the “Circle of Good” foundation. To date, parameters have been developed for 67 diseases, with a total of 257 parameters. In a pilot project for three diseases (cystic fibrosis, SMA, Duchenne muscular dystrophy), data were received from 51 regions of the Russian Federation; therapy was able to be assessed by criteria for more than 70% of patients.

The systems of the Commonwealth countries were presented by Adlet Berikbolovich Tabarov (Kazakhstan), Aida Ashiralievna Zurdinova (Kyrgyzstan), Larisa Nikolaevna Gavrilenko (Belarus) and Albert Mushegovich Martirosyan (Armenia).

Closing of the Forum: New Stage of the CIS Orphan Consortium

Summarizing the results of the forum, Nuriya Zagitovna Musina outlined several strategic directions. This year, the forum expanded beyond the CIS: BRICS, MENA countries and other partners joined the work — more than 20 countries were represented. This creates a new scale for the Consortium and defines the next stage of its development.

Strategic directions for the Consortium’s development:

• Expanding cooperation with BRICS and MENA countries — a practical platform for the development, evaluation, registration and access to orphan drugs.

• Methodological convergence: moving towards joint clinical assessment similar to the Joint Clinical Assessment in the European Union.

• Development of risk-sharing approaches — a necessary condition for high-quality RWD collection and effective drug supply.

• Support for gene and cell technologies through networking and expert coordination: connecting academic developments with potential investors and building international cooperation.

• Health Policy and international cooperation — the main strategic niche of the Consortium while maintaining the clinical direction as a platform for networking.

“We are moving towards BRICS because there is colossal potential there when it comes to drug supply. It is imperative to coordinate our orphan drug development, and then the entire drug lifecycle — that’s a wonderful substrate for BRICS.”

“I am glad that we are creating these bridges between countries, that people are starting to know each other, to recognize each other. And I think this is very important, because the worst thing that can happen is when experts start stewing in their own juice and do not understand what is happening in the world.”

The III CIS Orphan Forum has come to an end: the Commonwealth countries are entering into cooperation with BRICS

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