RWE in Orphan Diseases: International Dialogue and Joint Decisions
On September 25, the 6th conference with international participation “Real Clinical Practice” was held in Moscow. Possible and Realistic."
During the plenary session, President of the CIS Orphan Consortium, Academician Alexander Rumyantsev emphasized:
"Cooperative observational studies are a tool for collecting data from real-world clinical practice, which allows for a deeper understanding of the efficacy and safety of innovative drugs, timely adjustments to clinical approaches, and the reliability of decisions."
A separate conference session, "Data from Real-World Clinical Practice in Orphan Diseases," organized by the CIS Orphan Consortium, became a platform for the exchange of practical initiatives.
Moderator — Executive Director of the CIS Orphan Consortium Nuriya Musina.
Keynotes:
The CIS Orphan Consortium currently unites 25 organizations from 8 countries, including the Association of Specialists in Health Technology Assessment (A.S. Kolbin).
This collaborative effort aims to develop the systematic use of real-world clinical practice data and expand access to innovative therapies for patients with rare diseases.
During the plenary session, President of the CIS Orphan Consortium, Academician Alexander Rumyantsev emphasized:
"Cooperative observational studies are a tool for collecting data from real-world clinical practice, which allows for a deeper understanding of the efficacy and safety of innovative drugs, timely adjustments to clinical approaches, and the reliability of decisions."
A separate conference session, "Data from Real-World Clinical Practice in Orphan Diseases," organized by the CIS Orphan Consortium, became a platform for the exchange of practical initiatives.
Moderator — Executive Director of the CIS Orphan Consortium Nuriya Musina.
Keynotes:
- Yernas Tleutaevich Tuleutayev (Kazakhstan, Director of the Republican Center for Coordination of Orphan Diseases of the University Medical Center Corporate Foundation) presented a draft national registry of patients with rare diseases. The country is creating a digital registration system integrated with medical information modules and interdepartmental exchange mechanisms. These tools should form the basis for forecasting needs, improving patient routing, and planning drug provision.
- Evgenia Valeryevna Nazarova, Executive Director of NAERES (https://t.me/naerez), spoke about a large-scale project of cooperative observational studies, which is creating a unified database of real-world clinical practice data for patients with rare diseases. The initiative, implemented by NAERES, brings together leading Russian federal centers and enables the integration of domestic experience into the international context.
- Ekaterina Iosifovna Alekseeva, Chief Pediatric Rheumatologist of the Russian Ministry of Health, Director of the N.F. Filatov Clinical Institute of Child Health at Sechenov University, and Corresponding Member of the Russian Academy of Sciences, presented a digital ecosystem for monitoring childhood rheumatic diseases. The system integrates clinical and PRO (patient-reported outcomes) data, enabling the assessment of quality of care, adherence to clinical guidelines, treatment efficacy and safety, and forecasting healthcare system needs.
The CIS Orphan Consortium currently unites 25 organizations from 8 countries, including the Association of Specialists in Health Technology Assessment (A.S. Kolbin).
This collaborative effort aims to develop the systematic use of real-world clinical practice data and expand access to innovative therapies for patients with rare diseases.
