Clinical Development & Regulatory

 

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Clinical Development & Regulatory, Monday 11 July 2022

last published: 20/May/22 23:45
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Clinical Development & Regulatory, 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
Panel discussion
Clinical Development & Regulatory
11:35

Patient recruitment in a competitive environment: challenges and opportunities of enrolling in rare/ultra rare conditions

Sponsored by Premier Research
Jackie Brown, Vice President, Rare Diseases, Therapeutic and Device Program Management, Premier Research
Nicole Carswell, Executive Director, Patient Engagement and Recruitment,, Premier Research
Katherine Capperella
Clinical Development & Regulatory
11:55

Fireside Chat: Diversity in clinical trials – Description pending

Sponsored by Janseen
Katherine Capperella, VP of Global Patient Engagement, Janssen Pharmaceutical companies of Johnson & Johnson
Michael Murphy
Clinical Development & Regulatory
12:15

Endpoints - where are we now and how does the development process need to evolve?

Michael Murphy, Chief Medical & Scientific Officer, WorldWide Clinical Trials
Clinical Development & Regulatory
12:35

VIDEO: Updates from FDA

FDA representative to be announced
Irina Antonijevic
Clinical Development & Regulatory
15:15

Importance of Natural History studies and real world data to support clinical and biomarker development

Irina Antonijevic, Chief Medical Officer, Head of R&D, Triplet Therapeutics
Will Maier
Clinical Development & Regulatory
15:35

Effective use of rare disease RWE for external controls in drug development

Will Maier, Vice President, Rare Disease, Drug Development Sciences, ICON plc
Kristin Van Goor
Clinical Development & Regulatory
15:55

Retrospective 6 years out from the Cures Act - where are we on Patient Experience Data & Updates from FDA on Real World Evidence

Moderator: Kristin Van Goor, Lead, North America Regulatory Policy, Vertex Pharmaceuticals
Violeta Stoyanova-Beninska
Clinical Development & Regulatory
16:35

Updates on use of Real World Evidence from EMA

Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products, Medicines Evaluation Board, Medicines Evaluation Board
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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