Rare Oncology
Rare Oncology, Monday 24 August 2020
last published: 27/Mar/20 18:15
Rare Oncology, Tuesday 25 August 2020
08:25
Chairperson’s opening remarks
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
08:30
Keynote Address: The future of rare diseases: developing and accessing the next generation of therapies
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
08:55
Sponsored Keynote Address: Gene therapy: the future is now
Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics
09:20
Keynote Panel: The moment is now for rare diseases, but how do we advance scientific innovation, data sharing, and the commercial landscape to expedite treatments for the remaining 6,000+ diseases
Moderator: Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH)
Peter Marks, Director, Center For Biologics Evaluation And Research (Cber), U.S. Food and Drug Administration
Janet Woodcock, Director Of Drug Evaluation And Research Center, Food and Drug Administration (FDA)
09:50
Keynote Panel: Patient involvement in drug development for rare diseases
Examining how patients are driving meaningful development and what companies are doing to incorporate patient input throughout clinical trials and beyond
Neena Nizar, President, The Jansens Foundation
Sebastien Martel, Global Head of Rare Diseases, Sanofi
Wolfram Nothaft, Chief Medical Officer, Takeda
Yann Le Cam, Chief Executive Officer, EURORDIS
10:30
10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK
13:00
Lunch Keynote Panel: Examining industry data strategies and use of AI to advance rare disease patient identification, R&D, and commercialization
Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma
Oodaye Shukla, Chief Data Officer, HVH Precision Analytics
Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
14:20
ROUNDTABLE 10: Patient and provider experiences - A tailored tech + talent approach to enhance the journey
Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions
14:20
ROUNDTABLE 11: Orphan Drug Act-assessing the impact since 1983 and looking forward
Rachel Sher, Vice President Policy And Regulatory Affairs, NORD
14:20
ROUNDTABLE 12: Real World Evidence – examining recent cases of real world evidence for approvals and reimbursement in Europe
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
14:20
ROUNDTABLE 13: Patient Perspective – methods for including patient perspectives in pre-clinical, clinical, and access
Robert Long, Executive Director, Uplifting Athletes
Seth Rotberg, Co Founder And President, Our Odyssey
14:20
ROUNDTABLE 14: Rare Cancer – advancing best practices and partnerships for rare cancer diagnosis and drug
Abby Sandler, Executive Director, Mypart, Center For Cancer Research,, National Institutes of Health
Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation
14:20
ROUNDTABLE 15: Title to be announced
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)
14:20
ROUNDTABLE 16: Diversity & inclusion - The importance of D&I in a global company - exploring its impact on employee morale, engagement and retention of workforce, and also the need for D&I in clinical
Sara Nochur, Senior Vice President Regulatory Affairs, Alnylam Pharaceuticals
Bert Bruce, Vice President, Rare Disease Commercial Development, Pfizer
14:20
ROUNDTABLE 17: Economics of Rare – Description to be announced
Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases
14:20
ROUNDTABLE 18: Splinter Groups - How to advance your organization, unify communities and focus on your mission while navigating challenges with personalities, emotions and leadership differences among
Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)
14:20
ROUNDTABLE 19: Diagnostic access programs – advancing access to rare disease diagnosis in emerging markets
Nataliya Shandarovska, Patient Advocate, National Alliance for Rare Diseases Support - Malta
Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria
14:20
ROUNDTABLE 1: Reducing study burden – approaches for assessing and reducing the burden of clinical trial participation for patients
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
14:20
ROUNDTABLE 23: Gene therapy education – best practices for educate patient populations on the science and implications of gene therapy
14:20
ROUNDTABLE 2: Synthetic control arms - using RWE to streamline the regulatory process
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
14:20
ROUNDTABLE 3: The patient journey - improving clinical trial performance and delivering better study outcomes through a personalized approach to the patient experience
Scott Gray, CEO, Clincierge
14:20
ROUNDTABLE 4: Early Access – effective measures for successful EAPs
Anne Cropp, Chief Scientific Officer, Early Access Care
14:20
ROUNDTABLE 5: Patient-Focused Drug Development – how to get started
Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc
14:20
ROUNDTABLE 6: Balancing the Patient Experience & ROI – understanding when to incorporate patient accommodations into study programs and balancing short and long term financial goals
Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge
14:20
ROUNDTABLE 7: Gene therapy CMC – early pDNA considerations for late stage success
Michelle Berg, President, Gmp Nucleic Acids, Aldevron
14:20
ROUNDTABLE 8: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts
David Lapidus, President, Lapidus Data
14:20
ROUNDTABLE 9: Access Programs - discussion on the value of rare and orphan access programs, next generation registries and post marketing surveillance for products
Becky Thompson, Director Integrated Solutions, Parexel
ROUNDTABLE SESSIONS (1-hour roundtable sessions split between 2 rounds of 30min) – Sign up for two roundtables by registration
3:20 END OF ROUNDTABLES
16:45
4:45 AFTERNOON NETWORKING BREAK - ADVOCACY LOUNGE: Networking break panel discussion in advocacy lounge
16:50
PANEL: Stronger Together: The Power in Aligning our Objectives around the Patient
Michael Hanley, Chief Commercial Officer, Aeglea Biotherapeutics
17:25
Keynote Address: KYMRIAH case study: what it takes to bring an organization from clinical to commercial scale for vector manufacture
Nick Page, Chief Operations Officer, Oxford Biomedica
17:35
Keynote Panel: Enabling delivery of cell and gene therapies to patients: resolving continued manufacturing bottlenecks
Moderator: James Miskin, Chief Technical Officer, Oxford Biomedica
Ran Zheng, Chief Technology Officer, Orchard Therapeutics
Jan Thirkettle, Chief Development Officer, Freeline Therapeutics
Yong Dai, Chief Technology Officer, Prevail Therapeutics
Mayo Pujols, Vice President, Head Of Global Cell And Gene Technical Development And Manufacturing, Novartis
18:05
End of Conference Day 1
Networking Hour sponsored by Orchard Therapeutics
last published: 27/Mar/20 18:15
