Clinical Development/ Regulatory
Clinical Development & Regulatory, Wednesday 29 April 2020
last published: 31/Jan/20 21:55
Clinical Development & Regulatory, Thursday 30 April 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)
10:30
10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK
Trial Design
Clinical Development & Regulatory
11:30Next generation human enzymes: Innovative solutions for patients with rare diseases
Anthony Quinn, President And Chief Executive Officer, Aeglea Biotherapeutics
Clinical Development & Regulatory
11:50Updates from FDA on Rare Disease Clinical Trial Design and incorporation of patient voice
Peter Stein, Director, Office Of New Drugs (Ond), Cder, Food and Drug Administration (FDA)
Clinical Development & Regulatory
12:10Running rare disease registries in the digital age
Lauren Neighbours, Neighbours, Senior Director, Regulatory Affairs, Optum Digital Research Network, Optum
Clinical Development & Regulatory
12:30A partnership that lasts - key considerations when working with CROs on rare disease studies
Jonathan Kornstein, Executive Director, Program Strategy, Premier Research
13:00
Lunch Keynote Panel: Examining industry data strategies and use of AI to advance rare disease patient identification, R&D, and commercialization
Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders (NORD)
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma
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 14: Rare Cancer – advancing best practices and partnerships for rare cancer diagnosis and drug
Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation
Abby Sandler, Executive Director, Mypart, Center For Cancer Research,, National Institutes of Health
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
14:20
ROUNDTABLE 17: Internal Patient Perspective – blocking and tackling actionable implementation of patient voice throughout company functions
Jennifer Helfer, Director, Global Patient Advocacy Lead, bluebird bio
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, Chief Executive Officer, 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
Patient Voice
Clinical Development & Regulatory
15:25Science-Driven and Patient-Focused: Rare Disease Patient and Caregiver Inclusion in Early Therapeutic Discovery
Luke Rosen, Head Of Patient Engagement And Digital Health Experience, Ovid Therapeutics
Clinical Development & Regulatory
15:45Case Study: Transforming training protocols to incorporate patients and caregivers’ voice into all aspects of clinical development
Mary Mcgowan, Executive Director, The Myositis Association
Clinical Development & Regulatory
16:05Patients preference studies and multi-criteria decision analyses - their value and impact for Rare Diseases regulatory submissions
Leo Russo, Senior Director, Epidemiology Worldwide Safety And Regulatory, Pfizer
Clinical Development & Regulatory
16:25Following patients with rare and orphan diseases from clinical trials to market
Andrew Ecob, Vice President Of Real World Evidence Strategy, PAREXEL
16:45
4:45 AFTERNOON NETWORKING BREAK
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 FOLLOWED BY NETWORKING DRINKS RECEPTION
last published: 31/Jan/20 21:55
