Clinical Development/ Regulatory


Clinical Development & Regulatory, Monday 24 August 2020

last published: 19/May/20 00:46


Clinical Development & Regulatory, Tuesday 25 August 2020

Ellen Coleman

Chairperson’s opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Emil Kakkis

Keynote Address: The future of rare diseases: developing and accessing the next generation of therapies

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Bobby Gaspar

Sponsored Keynote Address: Gene therapy: the future is now

Bobby Gaspar, Chief Executive Officer, Orchard Therapeutics
Christopher Austin

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)
Neena Nizar

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


Trial Design

Anthony Quinn
Clinical Development & Regulatory

Next generation human enzymes: Innovative solutions for patients with rare diseases

Anthony Quinn, President And Chief Executive Officer, Aeglea Biotherapeutics
Peter Stein
Clinical Development & Regulatory

Updates 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)
Lauren Neighbours
Clinical Development & Regulatory

Using an integrated digital model for patient finding in rare disease trials

Lauren Neighbours, VP, Regulatory Affairs & Program Management, Optum
Jonathan Kornstein
Clinical Development & Regulatory

A partnership that lasts - key considerations when working with CROs on rare disease studies

Jonathan Kornstein, Executive Director, Program Strategy, Premier Research
Thomas Abbott

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
Jan Nielsen

ROUNDTABLE 10: Patient and provider experiences - A tailored tech + talent approach to enhance the journey

Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions
Rachel Sher

ROUNDTABLE 11: Orphan Drug Act-assessing the impact since 1983 and looking forward

Rachel Sher, Vice President Policy And Regulatory Affairs, NORD
Alexander Natz

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)
Seth Rotberg

ROUNDTABLE 13: Patient Perspective – methods for including patient perspectives in pre-clinical, clinical, and access

Seth Rotberg, Co Founder And President, Our Odyssey
Robert Long, Executive Director, Uplifting Athletes
Kristin Anthony

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
Andrea Furia-Helms

ROUNDTABLE 15: Title to be announced

Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration
Sara Nochur

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 Pharmaceuticals
Bert Bruce, Vice President, Rare Disease Commercial Development, Pfizer
Annie Kennedy

ROUNDTABLE 17: Economics of Rare – Description to be announced

Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases
Debbie Drell

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)
Nataliya Shandarovska

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
Scott Schliebner

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

ROUNDTABLE 23: Gene therapy education – best practices for educate patient populations on the science and implications of gene therapy

Chelsea Catsburg

ROUNDTABLE 2: Synthetic control arms - using RWE to streamline the regulatory process

Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Scott Gray

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
Anne Cropp

ROUNDTABLE 4: Early Access – effective measures for successful EAPs

Anne Cropp, Chief Scientific Officer, Early Access Care
Meagan Spychala

ROUNDTABLE 5: Patient-Focused Drug Development – how to get started

Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc
Amanda Finlayson

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
Michelle Berg

ROUNDTABLE 7: Gene therapy CMC – early pDNA considerations for late stage success

Michelle Berg, President, Gmp Nucleic Acids, Aldevron
David Lapidus

ROUNDTABLE 8: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts

David Lapidus, President, Lapidus Data
Becky Thompson

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


Patient Voice

Luke Rosen
Clinical Development & Regulatory

Science-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
Mary Mcgowan
Clinical Development & Regulatory

Case Study: Transforming training protocols to incorporate patients and caregivers’ voice into all aspects of clinical development

Mary Mcgowan, Executive Director, The Myositis Association
Leo Russo
Clinical Development & Regulatory

Patients 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
Andrew Ecob
Clinical Development & Regulatory

Following patients with rare and orphan diseases from clinical trials to market

Andrew Ecob, Vice President Of Real World Evidence Strategy, Parexel

4:45 AFTERNOON NETWORKING BREAK - ADVOCACY LOUNGE: Networking break panel discussion in advocacy lounge

Michael Hanley

PANEL: Stronger Together: The Power in Aligning our Objectives around the Patient

Michael Hanley, Chief Commercial Officer, Aeglea Biotherapeutics
Nick Page

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
James Miskin

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
Yong Dai, Chief Technology Officer, Prevail Therapeutics
Jan Thirkettle, Chief Development Officer, Freeline Therapeutics
Mayo Pujols, Vice President, Head Of Global Cell And Gene Technical Development And Manufacturing, Novartis

End of Conference Day 1

Networking Hour sponsored by Orchard Therapeutics
last published: 19/May/20 00:46