Webinar "Real world evidence and real world data (RWE/RWD) in rare diseases"

Date of the event: 18 April 2025

09:00 – 09:10
Opening of the webinar

Kolbin Alexey Sergeevich
MD, Professor, Head of the Department of Clinical Pharmacology and Evidence-Based Medicine, FSBEI HE "First St. Petersburg State Medical University named after I.P. Pavlova, Association of Specialists in Healthcare Technology Assessment

Musina Nuria Zagitovna
Ph.D., Associate Professor, Executive Director of the CIS Orphan Consortium

09:10 – 09:30
Greetings from the CIS Orphan Consortium

Rumyantsev Alexander Grigorievich
MD, Professor, Academician of the Russian Academy of Sciences, Chairman of the Expert Council on Rare (Orphan) Diseases of the State Duma of the Federal Assembly of the Russian Federation and the Expert Council of the Circle of Good Foundation, President of the NMIC DGOI im. Dmitry Rogachev

Aripova Tamara Uktamovna
MD, Academician of the Academy of Sciences of the Republic of Uzbekistan, Director of the Institute of Human Immunology and Genomics of the Academy of Sciences of the Republic of Uzbekistan, Vice-President of the Orphan Consortium of the CIS Countries

09:30 – 09:50
The paradigm of the RCP in the world. Basic definitions, concepts, areas of application and role in modern healthcare. The concept of the RCP in the EAEU

Kolbin Aleksey Sergeevich
MD, Professor, Head of the Department of Clinical Pharmacology and Evidence-Based Medicine, FSBEI HE "First St. Petersburg State Medical University named after I.P. Pavlova"

09:50 – 10:05
Current status of development of the legal basis for the use of real clinical practice in the framework of regulation of circulation of medicines in the EAEU

Kalinichenko Vadim Valentinovich
Department for regulation of circulation of medicines and medical devices of the Ministry of Health of the Russian Federation

10:05 – 10:20
Planning of non-interventional studies with elements of RCP

Niyazov Ravil Rashidovich
Candidate of Medical Sciences, specialist in the development and registration of drugs, LLC "Center for Scientific consulting», RWD working group under the EAEU

10:20 – 10:35
Issues of biostatistical analysis of data for rare diseases in real clinical practice

Solodovnikov Alexander Gennadievich
PhD, Associate Professor of RUDN, Associate Professor of USMU, Director of Quality Management at Statendox LLC

10:35 – 10:55
Rare diseases and modern challenges

Zyryanov Sergey Kensarinovich
Dr. Head of the Department of General and Clinical Pharmacology, RUDN University, Deputy Chief Physician, City Clinical Hospital No. 24, Health Department
Report supported by Sobi

10:55 – 11:10
Methodological approaches to RWE analysis in orphan diseases. Review of best practices

Fedyaeva Vlada Konstantinovna
Federal State Autonomous Educational Institution of Higher Education, First Moscow State Medical University named after I.M. Sechenov, Institute of Translational Medicine and Biotechnology

11:10 – 11:25
Current need for RWE/RWD for assessing medical technologies in rare diseases

Romanova Irina Sergeevna
PhD, Associate Professor, Head of the Department of Clinical Pharmacology, Belarusian State Medical University

Kozhanova Irina Nikolaevna
Dr. of Medicine, Associate Professor, Professor of the Department of Clinical Pharmacology, Belarusian State Medical University

Zhevneronok Irina Vladimirovna
Head of the 2nd Department of Children's Diseases, Belarusian State Medical University, Chief Freelance specialist of the Ministry of Health of the Republic of Belarus on hereditary neuromuscular diseases

11:25 – 11:40
Patient-reported outcomes (PRO) data as part of the RWE ecosystem

Nuriya Zagitovna Musina
PhD, Associate Professor, Executive Director of the CIS Orphan Consortium

11:40 – 11:55
Methods for Improving the Efficiency of Drug Development for Orphan Diseases

Kirill Vitalievich Peskov
Head of the Center for Mathematical Modeling in Drug Development, Sechenov University

11:55 – 12:10
Application of RCP data in orphan diseases: good practice and historical control

Kirill Vladimirovich Zhudenkov
PhD, Associate Professor, Department of Higher Mathematics, Mechanics and Mathematical Modeling, First Moscow State Medical University named after I.M. Sechenov

12:10 – 12:25
Practical tools and methodologies for collecting and structuring RCP

Alexander Naslednikov
Founder and Director of Business Development, RVD Marketplace LLC

12:25 – 12:40
Telemedicine in orphan diseases

Boris Valentinovich Zingerman
General Director of ARPIIM – “National Base of Medical Knowledge”

12:40 – 12:55
Personalized gene therapy in rare diseases: the role of RCT data

Denis Vladimirovich Rebrikov
Doctor of Biological Sciences, Vice-Rector for Research, Pirogov Russian National Research Medical University, Director of the Institute of Translational Medicine, V.I. Kulakov National Medical Research Center

12:55 – 13:10
Kazakhstan's experience of working with RCT data

Adlet Berikbolovich Tabarov
Deputy Chairman of the Board, NNC Healthcare Development named after Salidat Kairbekova, Ministry of Health of the Republic of Kazakhstan

13:10 – 13:25
Russian registries of rare diseases as a source of RWD

Ivanov Alexander Viktorovich
Head of Strategic Consulting Department, Aston Health

Schedule

09:00 – 09:10

Opening of the webinar

Kolbin Alexey Sergeevich

Professor, Head of the Department of Clinical Pharmacology and Evidence-Based Medicine, Federal State Educational Institution of Higher Education “First St. Petersburg State Medical University named after Academician I.P. Pavlov”.

Musina Nuria Zagitovna

Executive Director of the CIS Orphan Consortium, Director of International Cooperation of NAERES

09:10 – 09:30

Greetings from the CIS Orphan Consortium

Rumyantsev Alexander Grigorievich

President of the CIS Orphan Consortium, President of the Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center for Pediatric Hematology and Oncology" of the Ministry of Health of the Russian Federation, Academician of the Russian Academy of Sciences

Aripova Tamara Uktamovna

Doctor of Medical Sciences, Academician of the Academy of Sciences of the Republic of Uzbekistan, Director of the Institute of Human Immunology and Genomics of the Academy of Sciences of the Republic of Uzbekistan, Vice President of the Orphan Consortium of the CIS Countries

09:30 – 09:50

The paradigm of the RCP in the world. Basic definitions, concepts, areas of application and role in modern healthcare. The concept of the RCP in the EAEU

Kolbin Alexey Sergeevich

09:50 – 10:05

Current status of development of the legal basis for the use of real clinical practice in the framework of regulation of circulation of medicines in the EAEU

Kalinichenko Vadim Valentinovich

Департамент регулирования обращения лекарственных средств и медицинских изделий Минздрава России

10:05 – 10:20

Planning of non-interventional studies with elements of RCT

Niyazov Ravil Rashidovich

Specialist in regulatory issues of drug development. LLC "Center for Scientific Consulting"

10:20 – 10:35

Issues of biostatistical data analysis for rare diseases in real clinical practice

Solodovnikov Alexander Gennadievich

PhD, Associate Professor of RUDN, Associate Professor of USMU, Director of Quality Management of Statendoks LLC

10:35 – 10:55

Rare diseases and modern challenges

Zyryanov Sergey Kensarinovich

Doctor of Medical Sciences, Head of the Department of General and Clinical Pharmacology of RUDN University, Deputy Chief Physician of City Clinical Hospital No. 24 DZ Report supported by the company "Sobi"

10:55 – 11:10

Methodological approaches to RWE analysis in orphan diseases. A review of best practices

Fedyaeva Vlada Konstantinovna

Head of the Department of Methodology for Development and Expert Evaluation of Clinical Guidelines of the Federal State Budgetary Institution Center for Expertise and Qualification of Medical Care of the Ministry of Health of the Russian Federation

11:10 – 11:25

The current need for RWE/RWD for medical technology assessment in rare diseases

Romanova Irina Sergeevna

PhD, Associate Professor, Head of the Department of Clinical Pharmacology, Belarusian State Medical University

Kozhanova Irina Nikolaevna

Doctor of Medical Sciences, Associate Professor, Chief Specialist of the Main Healthcare Department of the Minsk Regional Executive Committee for Clinical Pharmacology, Professor of the Department of Clinical Pharmacology of the Belarusian State Medical University

Zhevneronok Irina Vladimirovna

Head of the 2nd Department of Children's Diseases of the Belarusian State Medical University, Chief Specialist of the Ministry of Health of the Republic of Belarus for Hereditary Neuromuscular Diseases in Children, Head of the Republican Center for Hereditary Neuromuscular Diseases in Children

11:25 – 11:40

Patient-reported outcomes (PRO) data as part of the RWE ecosystem

Musina Nuria Zagitovna

Executive Director of the CIS Orphan Consortium, Director of International Cooperation of NAERES

11:40 – 11:55

Methods to improve the efficiency of drug development for orphan diseases

Peskov Kirill Vitalievich

Head of the Center for Mathematical Modeling in Drug Development at Sechenov University

11:55 – 12:10

Application of RCT data in orphan diseases: good practice and historical control

Zhudenkov Kirill Vladimirovich

PhD, Associate Professor, Department of Higher Mathematics, Mechanics and Mathematical Modeling, I.M. Sechenov First Moscow State Medical University

12:10 – 12:25

Practical tools and methodologies for collecting and structuring RCP

Alexander Naslednikov

Founder and Director of Business Development of RVD Marketplace LLC

12:25 – 12:40

Telemedicine in Orphan Diseases

Zingerman Boris Valentinovich

Association of Developers and Users of Artificial Intelligence in Medicine, Medsenger.AI Remote Monitoring Platform

12:40 – 12:55

Personalized Gene Therapy in Rare Diseases: The Role of RCT Data

Rebrikov Denis Vladimirovich

D.B.N., Vice-Rector for Research at the N.I. Pirogov Russian National Research Medical University, Director of the Institute of Translational Medicine at the V.I. Kulakov National Medical Research Center

12:55 – 13:10

Kazakhstan's experience of working with RCP data

Tabarov Adlet Berikbolovich

Deputy Chairman of the Board of the Republican State Enterprise on the Right of Economic Management "National Scientific Center for Healthcare Development named after Salidat Kairbekova" of the Ministry of Healthcare of the Republic of Kazakhstan

13:10 – 13:25

Evaluation of real clinical practice of therapy of patients with primary immunodeficiencies (PID) within the framework of provision by the Circle of Goodness foundation: experience of data collection and analysis

Mukhina Anna Alexandrovna

PhD, research fellow, Department of Epidemiology and Monitoring of Primary Immunodeficiencies, Dmitry Rogachev National Medical Research Center for Pediatric Hematology and Oncology, Ministry of Health of the Russian Federation, curator of the PID NAEPID registry, allergist-immunologist, Dmitry Rogachev National Medical Research Center for Pediatric Hematology and Oncology, Ministry of Health of the Russian Federation

13:25 – 13:40

Russian Rare Disease Registries as a Source of RWD