08:30 – 10:30
Session 5: Global rare disease policies and programmes
- Introduction: report from Group C
- UN Committee for Rare Diseases – Anders Olauson, Agrenska Foundation, Sweden
- International Rare Diseases Resarch Consortium (IRDiRC) – Christopher Austin, National Institutes of Health, USA
- A global approach to patient registries – Manuel Posada, Instituto de Salud Carlos III, Spain
- Rare Diseases International – Durhane Wong-Rieger, Canadian Organization for Rare Disorders, Canada
- Analysis of rare disease global policies and programmes to advance access to care and treatment – Safiyya Dharssi, Pfizer, USA
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11:00 – 12:45
Session 6: Access to treatment
- Introduction: report from Group D
- Closing the gap: the World Federation of Hemophilia’s experience in responding to the challenges of a global rare disease – Alain Weill, World Federation of Hemophilia, Canada
- Logistical aspects of access to treatment – Jason Blackman, Marken, South Africa
- The need for innovative thinking to treat rare diseases in Africa – Kelly du Plessis, Rare Diseases South Africa, South Africa
- GSK’s graduated approach to patents and intellectual property to widen access to medicines in the world’s poorest countries – Rudy Onia, GSK, South Africa
- The difficulties in receiving access to high priced and orphan drugs: some thoughts – Marc Blockman, University of Cape Town, South Africa
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13:45 – 15:45
Session 7: Research and research funding
- Introduction: report from Group E
- Gaining access to available NIH rare diseases and translational research resources – Christopher Austin, National Institutes of Health, USA
- Catalysing progress from discovery to health benefits through research collaboration – Petra Kaufmann, National Institutes of Health, USA
- Applications to rare diseases from H3 Africa Research Project – Raj Ramesar, University of Cape Town, South Africa
- H3Africa BioNet and opportunities for rare diseases research – Judit Kumuthini, University of Cape Town, South Africa
- In vitro assays for characterisation of novel rare genetic variants – Maja Stojiljkovic, University of Belgrade, Serbia
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15:45 – 16:45
Session 8: Panel Discussion – “Problem Board”
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16:45 – 18:00
Session 9: Poster session
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