| 08:30 | Registration | O'Reilly Hall, University College Dublin |
|---|---|---|
09:00 | Opening Address | Professors Rachel Crowley & Cormac McCarthy Co-Leads, Rare Disease Clinical Trial Network |
SESSION ONE | EUROPEAN REFERENCE NETWORK REGISTRIES | - |
09:10 | Rare Disease Research Catalyst Data Overview | Mr Daniel Mikula, Rare Disease Research Catalyst Consortium |
09:20 | European Registries for Rare Endocrine and Bone Conditions | Professor Natasha Appelman-Dijkstra, Leiden University Medical Centre |
09:35 | Epidermolysis Bullosa (EB) Registry | Dr Sinéad Hickey, Debra Ireland Ms Lara Cutlar, NISR Solutions CLG |
09:50 | Rare Neurological Disease Registry | Professor Sinéad Murphy, Tallaght University Hospital, Trinity College Dublin |
10:05 | ERN Lung Registries and Academy | Ms Elisabeth Humbert-Dorfmüller, ERN Lung |
10:20 | Panel Discussion | Chaired by: Dr Fiona Browne, Children’s Health Ireland/National & International Skin Registries |
10:40 | Clinical Trial Spotlight: Autoimmune Pulmonary Alveolar Proteinosis-IMPALA 2 | Professor Elisabeth Bendstrup, Aarhus University |
10:55 | PPI & Seed Funding Presentations | - |
11:00 | Poster Viewing & Networking | - |
11:45 | Clinical Trial Spotlight: Rare Bone Disorders - TOPaZ/ZiPP-LTE | Professor Stuart Ralston, University of Edinburgh |
SESSION TWO | RARE DISEASE CLINICAL TRIALS - STAKEHOLDERS PERSPECTIVES | - |
12:00 | Regulatory Perspective | Ms Emer Cooke, European Medicines Agency |
12:15 | Public Patient Involvement (PPI) Perspective | Ms Mary Vasseghi, Trinity College Dublin |
12:30 | Industry Perspective | Mr Killian MacDonald, Gilead Sciences Ireland & Clinical Research Exchange Group, IPHA |
12:45 | Panel Discussion | Chaired by: Professor Elisabeth Bendstrup, Aarhus University |
13:00 | Lunch & Poster Viewing | - |
14:00 | Clinical Trial Spotlight: Duchenne Muscular Dystrophy – DYNE-DELIVER | Dr Declan O’Rourke, Children’s Health Ireland, Temple St. |
SESSION THREE | TRIALS METHODOLOGY & TRANSLATION | - |
14:15 | Translating Basic Science into Meaningful Therapies - New therapeutic approaches in pulmonary arterial hypertension | Professor Marc Humbert, Université Paris-Saclay |
14:45 | Platform Trials | Dr Leanne Hays, HRB PRIME Ireland, Irish Critical Care-CTN |
15:00 | Study Within a Trial (SWAT) | Professor Valerie Smith, UCD |
15:15 | Panel Discussion | Chaired by: Dr Linda O’Neill, UCD |
15:30 | Poster Viewing & Networking | - |
16:00 | Clinical Trial Identification Project Overview | Ms Sarah Forde, Rare Disease Clinical Trial Network |
16:15 | KEYNOTE LECTURE Clinical Trial Designs: New Options for the Study of Rare Diseases | Professor Jeffrey Krischer, University of South Florida, USA |
17:15 | Poster Prize Presentations | - |
17:20 | Closing Address Followed by Drinks Reception | Professor Michael Keane, Head of School, School of Medicine, UCD |
