Rare Disease Advocacy World


Rare Disease Advocacy World, Monday 17 August 2020

last published: 25/Aug/20 17:55


Rare Disease Advocacy World, Monday 24 August 2020

Debra Miller
Rare Disease Advocacy World

Best practices for venture philanthropy and industry partnership

Debra Miller, CEO & Founder, CureDuchenne
Raymond Huml
Rare Disease Advocacy World

Key Role for Patient Advocacy Groups in Rare Disease Drug Development: Making Better Use of This Valuable Resource

Raymond Huml, Vice President of Medical and Scientific Strategy, Head, Health’s Rare Disease Consortium, Syneos Health
Ron Bartek
Rare Disease Advocacy World

PANEL: Stronger Together during COVID-19: Continuing industry and patient partnerships along the full continuum of development, from pre-clinical through commercialization

Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Matt Flesch, Vice President Of Communications And Patient Advocacy, Horizon Therapeutics
Michael Hanley, Chief Commercial Officer, Aeglea BioTherapeutics
Kim Stephens, President, Project Alive
Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)
Jenn Mcnary, Founder, One Rare


Ellen Coleman

Chairperson’s opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Jeremy Levin

Keynote Address: Rare diseases in the time of Covid-19

Jeremy Levin, Chairman and Chief Executive Officer, Chairman, BIO, Ovid Therapeutics
Emil Kakkis

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

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
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 throughou

Neena Nizar, President, The Jansens Foundation
Jessica Scott, Head of R&D Patient Engagement Office,, Takeda Pharmaceuticals
Amy Mckee, Vice President, Regulatory, Parexel Consulting, Parexel International
Dara Richardson-Heron, Chief Patient Officer, Pfizer
Yann Le Cam, Chief Executive Officer, EURORDIS


Annette Bakker
Rare Disease Advocacy World

Non-profit drug discovery enabling platforms vs advocacy organizations - challenges in pharma perception of foundations

Annette Bakker, President, Childrens Tumor Foundation
Brian Loew
Rare Disease Advocacy World

Redefining Rare: Health Journeys for millions of rare disease patients to unlock medical discoveries

Brian Loew, CEO, Inspire
Amy Gray
Rare Disease Advocacy World

PANEL: Moving treatments from lab to trial – what are the current barriers and how are patient organizations overcoming these challenges and collaborating with partners

Amy Gray, Chief Executive Officer, Charcot-Marie-Tooth Association
Sharon Hesterlee, Executive Vice President, Chief Research Officer, Muscular Dystrophy Association
Claudia Hirawat, Executive Chairman, VOZ Advisors
Lianna Orlando, Senior Director Of Research, CureDuchenne


Amanda Finlayson

ROUNDTABLE 1: 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
David Lapidus

ROUNDTABLE 2: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts

David Lapidus, President, Lapidus Data
Sam Lucas

ROUNDTABLE 3: Expanded Access Programs- How EAPs can complement rare disease drug development and optimize commercial launch

Sam Lucas, SVP, Expanded Access Programs, Durbin
Julia Campbell, VP, Client Strategies and Services, Durbin
Todd Galles, VP, US Business Development, EAPs, Durbin
Janet Maynard

ROUNDTABLE 4: FDA updates - Office of Orphan Product Development

Janet Maynard, Director, Office Of Orphan Products Development, Food And Drug Administration, U.S. Food and Drug Administration
Lewis Fermaglich, Medical Advisor, Office of Orphan Products Development,, FDA


Durhane Wong-Rieger
Rare Disease Advocacy World

Rare Disease International: Is it time for global access? If not now, when?

Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders
Jacqueline Erdo
Rare Disease Advocacy World

Payer engagement strategy – relationship building and bridging information gaps to ensure access for cystic fibrosis patients

Jacqueline Erdo, Senior Manager, Policy & Advocacy,, Cystic Fibrosis Foundation
Terry Wilcox
Rare Disease Advocacy World

The importance of patient engagement in the age of ICER

Terry Wilcox, Co-Founder And Executive Director, Patients Rising & Patients Rising
Mindy Cameron
Rare Disease Advocacy World

The burden of access on patients and families

Mindy Cameron, Advocacy Director, Little Hercules Foundation
Oodaye Shukla

Keynote Panel: Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization

Moderator: Oodaye Shukla, Chief Data Officer, HVH Precision Analytics, now EVERSANA
Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma
Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
last published: 25/Aug/20 17:55