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Commercial, Monday 11 July 2022

last published: 20/May/22 23:45
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Commercial, Tuesday 12 July 2022

08:30

Organizer Opening Remarks

08:35

Chair Opening Remarks

Emil Kakkis
08:40

Keynote Address: Drug Development in the 21st Century - The use of primary disease activity biomarkers to establish efficacy

Keynote speaker to be announced
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Shannon Resetich
09:00

Keynote Fireside-chat: State of global rare ecosystem – leveraging public private partnerships to further shape the future or rare diseases around the world

Shannon Resetich, Global Franchise Head, Rare Diseases, Sanofi Genzyme
Fumie Griego, Deputy Director General And Chief Operating Officer, International Federation of Pharmaceutical Manufacturers and Associations
Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Disorders
09:35

Keynote Address: Reserved for FDA Commissioner

Panel discussion
09:45

Keynote Panel: Advancing cell and gene therapies for rare diseases: What challenges face developers in pre-commercialization and how can all stakeholders work to get therapies to rare patients faster?

Moderator: Izaskun Elorza, Senior Medical Director,, Parexel
Janet Lynch Lambert, Chief Executive Officer, Alliance for Regenerative Medicine
Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics
Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD)
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Therapeutics
Richa Poddar
Commercial
11:15

Launch strategies: Considerations for natural history, assistance programs and improving diagnosis

Richa Poddar, Chief Commercial Officer, Agios Pharmaceuticals
Maria Kirsch
Commercial
11:35

Fireside Chat: The Digital Hub -balancing high-yech and high-touch solutions to eliminate access, affordability and adherence barriers of rare disease patients

Maria Kirsch, General Manager, Patient Services, EVERSANA
Grant Castor
Commercial
11:55

Responsible marketing for rare diseases

Grant Castor, Vice President Commercial Operations, Sentynl Therapeutics
Daniel Donovan
Commercial
12:15

Successful commercialization of products for rare diseases at a fraction of the cost

Daniel Donovan, Founder & CEO, rareLife solutions
Panel discussion
Commercial
15:15

PANEL: Key levers for successful launch of rare disease therapies

Moderator: Paul Nash, Senior Director, Simon-Kucher & Partners
Brad Martin, Vice President, Market Access, Taysha Gene Therapies
Simone Seiter, Partner, Simon-Kucher & Partners
Peter Shadday, Chief Commercial Officer US, Global Head of Marketing Specialties, Board Member,, Zambon
Commercial
15:55

Fireside Chat: Patient finding - understanding patient populations

Sponsored by EVERSANA
Panel discussion
16:50

Data & Innovation Keynote Panel: Use of Real World Data & AI for rare diseases - where are key areas of opportunity in product development, patient access and commercialization?

Sponsored by EVERSANA
Moderator: Rachele Hendricks-Sturrup, Research Director, Real World Evidence, Duke Margolis Center for Health Policy
Christopher Moxham, Chief Scientific Officer, Rarebase, PBC
Samer Ansari, Head of Data, Digital & Technology, Takeda Oncology, Takeda
Scott Snyder, Chief Digital Officer, EVERSANA
last published: 20/May/22 23:45
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