International Conference on Rare Diseases 2022

Presentations

Building a Pathway from Diagnosis to Access


“SESSION 2 | EUROPEAN vs NATIONAL RD ACTION PLANS”

Prof. Birute TUMIENE, Associate Professor at Vilnius University, Faculty of Medicine, Head of Unit, Center for Medical Genetics, Vilnius University Hospital Santaros Clinics, National Coordinator, Orphanet International, Panel of Experts Member, WHO Collaborative Global Network for Rare Diseases, Diagnostics Scientific Committee Member, International Rare Diseases Research Consortium (IRDiRC)
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“Screen4Care NBS IMI Project”

Nicolas GARNIER, Senior Director, Patient Advocacy Lead, Global Product Development, Pfizer Rare Disease
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“NBS Pilot Austria”

Vassiliki KONSTANTOPOULOU, Inborn Errors of Metabolism, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Newborn screening and Laboratory for metabolic disorders
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“DARWIN EU (Data Analytics and Real World Interrogation Network)”

Alexandra PACURARIU, Scientific Administrator, Data Analytics and Methods Task Force, European Medicines Agency (EMA)
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“Rare Diseases: Think Sustainable, Think Digital, Think Patient!”

Norbert COUESPEL, Chair, Future-Proofing Health Systems Committee, European Health Parliament, 7th edition
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“B. NEW ACCESS MODELS FOR RD MEDICINES”

Nerea BLANQUÉ-CATALINA, Senior Vice President Market Access and Pricing, Alira Health
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“D. NEW FUNDING MODELS FOR RD DRUG DEVELOPMENT”

Devin ROSENTHAL, PhD. Vice President NovaQuest Capital Management
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“SESSION 6 | RARE DISEASES AS A NATIONAL PRIORITY IN GREECE”

Michael HIMONAS, General Manager of the Hellenic Association of Pharmaceutical Companies (SFEE)
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