Frauke Naumann-Winter | Head of Unit
BfArM

Day 2 - Thursday 24 October @ 11:10

Panel: What are the impacts of rare designation?

Moderator: Daniel O'Connor, Director, Regulatory Policy & Early Access, ABPI

Frauke Naumann-Winter, Head of Unit, BfArM

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Frauke Naumann-Winter / Panel: What are the impacts of rare designation?

Frauke Naumann-Winter, Head of Unit,
BfArM

Frauke Naumann-Winter is a molecular biologist and epidemiologist by training and holds a PhD in Genetics. Frauke Naumann-Winter is currently Head of Unit for Paediatric and Orphan Medicinal Products in the Federal Institute for Drugs and Medical devices and is the German delegate and Vice-Chair at ...

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Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International

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Durhane Wong-Rieger / Panel: What are the impacts of rare designation?

Durhane Wong-Rieger, President, Chief Executive Officer,
Canadian Organisation for Rare Disorder & Rare Diseases International

DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...

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Jonathan Kornstein, Vice President, Rare Disease and Pediatrics, CAIDYA

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Jonathan Kornstein / Panel: What are the impacts of rare designation?

Jonathan Kornstein, Vice President, Rare Disease and Pediatrics,
CAIDYA

Jonathan Kornstein is Vice President, Rare Disease and Pediatrics at Caidya and has over 29 years of expereince in the Pharmaceutical and CRO industry. He has demonstrated a depth of project management and clinical operations experience in a multitude of therapeutic areas with a focus on rare and...

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Day 3 - Friday 25th October @ 09:45

The use of RWD in orphan drug development and access pathways

-Where does real world data fit in the evidence hierarchy for developing orphan medicines?

-What are the opportunities and challenges for using real world data generated from rare disease patients?

-How can innovative approaches help to integrate real world evidence in supporting effective decision making by different stakeholders?

-Is there good alignment between decision makers on the value of real world data?

Moderator: Daniel O'Connor, Director, Regulatory Policy & Early Access, ABPI

Alison Cave, Chief Safety Officer, Medicines and Healthcare products Regulatory Agency

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Alison Cave / The use of RWD in orphan drug development and access pathways

Alison Cave, Chief Safety Officer,
Medicines and Healthcare products Regulatory Agency

Alison Cave is Chief Safety Officer at the Medicines and Healthcare products Regulatory Agency. She holds a BSc Honours degree and PhD and has significant academic research and regulatory experience with roles at Kings College London, the Wellcome Trust, UK Research and Innovation and the European...

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Frauke Naumann-Winter, Head of Unit, BfArM

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Frauke Naumann-Winter / The use of RWD in orphan drug development and access pathways

Frauke Naumann-Winter, Head of Unit,
BfArM

Frauke Naumann-Winter is a molecular biologist and epidemiologist by training and holds a PhD in Genetics. Frauke Naumann-Winter is currently Head of Unit for Paediatric and Orphan Medicinal Products in the Federal Institute for Drugs and Medical devices and is the German delegate and Vice-Chair at ...

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Sicily Mburu, International Federation Of Psoriasis Associations

Annika Jodicke, Senior Researcher in Pharmacoepidemiology, University of Oxford NDORMS