Clinical Development/ Regulatory

Clinical Development & Regulatory, Monday 24 August 2020

last published: 27/Mar/20 18:15

Clinical Development & Regulatory, Tuesday 25 August 2020

Ellen Coleman

08:25

Chairperson’s opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors

Emil Kakkis

08:30

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

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical

Mark Rothera

08:55

Sponsored Keynote Address: Gene therapy: the future is now

Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics

Christopher Austin

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)

Neena Nizar

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

Anthony Quinn

Clinical Development & Regulatory

11:30

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

11:50

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

12:10

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

12:30

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

Jonathan Kornstein, Executive Director, Program Strategy, Premier Research

Thomas Abbott

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

Jan Nielsen

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

Rachel Sher

14:20

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

Rachel Sher, Vice President Policy And Regulatory Affairs, NORD

Alexander Natz

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)

Robert Long

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

Abby Sandler

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

Andrea Furia-Helms

14:20

ROUNDTABLE 15: Title to be announced

Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)

Sara Nochur

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

Annie Kennedy

14:20

ROUNDTABLE 17: Economics of Rare – Description to be announced

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

Debbie Drell

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)

Nataliya Shandarovska

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

Scott Schliebner

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

Chelsea Catsburg

14:20

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

Chelsea Catsburg, Engagement Manager, BluePrint Orphan

Scott Gray

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

Anne Cropp

14:20

ROUNDTABLE 4: Early Access – effective measures for successful EAPs

Anne Cropp, Chief Scientific Officer, Early Access Care

Meagan Spychala

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

Amanda Finlayson

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

Michelle Berg

14:20

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

Michelle Berg, President, Gmp Nucleic Acids, Aldevron

David Lapidus

14:20

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

David Lapidus, President, Lapidus Data

Becky Thompson

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

Luke Rosen

Clinical Development & Regulatory

15:25

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

15:45

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

16:05

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

16:25

Following 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 - ADVOCACY LOUNGE: Networking break panel discussion in advocacy lounge

Michael Hanley

16:50

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

Michael Hanley, Chief Commercial Officer, Aeglea Biotherapeutics

Nick Page

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

James Miskin

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