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Speakers

Explore Patient-Centric Approaches to Trial Design, Recruitment and Access to Enhanced Outcomes

December 3-4, 2019
  • Arlington, VA

Amy P. Abernethy, MD, PhD
Principal Deputy Commissioner
U.S. Food and Drug Administration (FDA)

Martina Bebin M.D., M.P.A.
Professor of Neurology and Pediatrics
UAB Epilepsy Center

Melanie Blank, MD
Medical Officer, Rare Diseases Program
U.S. Food and Drug Administration (FDA)

Ron Cooper
President & CEO
Albireo Pharma

Michael Eging
Executive Director
Rare Action Access Project

Chris Garabedian
CEO
Xontogeny

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

Cherylann Gregory
CEO & President
Specialty Pharmacy Nursing Network (SPNN), Inc.

Eric D. Hargan
Deputy Secretary
U.S. Department of Health and Human Services

Nielsen Hobbs
Editor
The Pink Sheet

Emil D. Kakkis, MD, PhD
CEO, President and Founder
Ultragenyx Pharmaceutical Inc.

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

Brett Kopelan
Executive Director
debra of America

Katherine Leon
Co-Founder and Board Chair
SCAD Alliance

Candace Lerman
Founder
Rare Candace

Nanxin (Nick) Li, PhD, MBA
Senior Director, Health Economics and Outcomes Research
uniQure

Raymond Mankoski, MD, PhD
Vice President Medical Affairs
Blueprint Medicines

John Maslowski
CEO
Fibrocell

Wes Michael
President & Founder
Rare Patient Voice, LLC

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

Sara Nayeem, MD
Partner
NEA

Adora Ndu, PharmD, JD
Executive Director, Regulatory Affairs, Policy,
Research, Engagement (PRE), & Reg Internationa
BioMarin Pharmaceutical

Nneka Onwudiwe, PharmD, PhD, MBA
Founder and Chief Executive Officer
Pharmaceutical Economics Consultants of America (PECA) LLC

Tejashri Purohit-Sheth, MD
Director, Division of Clinical Evaluation and
Pharmacology/Toxicology (CBER / OTAT / DCEPT)
U.S. Food and Drug Administration (FDA)

Gretchen Randlett
Clinical Trial Commercial Product Strategy and Process
Eli Lilly and Company

Steve Roberds
Chief Scientific Officer
Tuberous Sclerosis Alliance

Isaac R. Rodriguez-Chavez, Ph.D., M.H.Sc., M.Sc.
FDA Officer, Clinical Research Methodology, Regulatory Compliance
and Policy Development, CDER
U.S. Food and Drug Administration

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Khushboo Sharma, MBA, RAC
Deputy Office Director of Operations, Office of New Drugs,
Center for Drug Evaluation and Research (CDER)
U.S. Food and Drug Administration (FDA)

R'Kes D. Starling, RPh, MBA
Founder and Chief Executive Officer
Reveles Clinical Services

Peter Stein
Director, Office of New Drugs, CDER
U.S. Food and Drug Administration (FDA)

Dan Takefman, Ph.D.
Head, Regulatory Affairs
Spark Therapeutics

Kevin Weatherwax
Co-PI, Transforming Expanded Access to Maximize Support
and Study (TEAMSS); Co-Chair, Expanded Access Oversight Committee
University of Michigan Health Systems

Monica Weldon
President, CEO & Founder
Bridge the Gap – SYNGAP Education and Research Foundation

Michael Werner
Co-Founder and Senior Policy Counsel
Alliance for Regenerative Medicine

Celia M. Witten, PhD, MD
Deputy Director, Center for Biologics Evaluation and Research (CBER)
U.S. Food and Drug Administration (FDA)

Janet Woodcock
Director, Center for Drug Evaluation & Research (CDER)
U.S. Food and Drug Administration (FDA)

Amy P. Abernethy, MD, PhD
Principal Deputy Commissioner
U.S. Food and Drug Administration (FDA)

Martina Bebin M.D., M.P.A.
Professor of Neurology and Pediatrics
UAB Epilepsy Center

Melanie Blank, MD
Medical Officer, Rare Diseases Program
U.S. Food and Drug Administration (FDA)

Ron Cooper
President & CEO
Albireo Pharma

Michael Eging
Executive Director
Rare Action Access Project

Chris Garabedian
CEO
Xontogeny

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

Cherylann Gregory
CEO & President
Specialty Pharmacy Nursing Network (SPNN), Inc.

Eric D. Hargan
Deputy Secretary
U.S. Department of Health and Human Services

Nielsen Hobbs
Editor
The Pink Sheet

Emil D. Kakkis, MD, PhD
CEO, President and Founder
Ultragenyx Pharmaceutical Inc.

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

Brett Kopelan
Executive Director
debra of America

Katherine Leon
Co-Founder and Board Chair
SCAD Alliance

Candace Lerman
Founder
Rare Candace

Nanxin (Nick) Li, PhD, MBA
Senior Director, Health Economics and Outcomes Research
uniQure

Raymond Mankoski, MD, PhD
Vice President Medical Affairs
Blueprint Medicines

John Maslowski
CEO
Fibrocell

Wes Michael
President & Founder
Rare Patient Voice, LLC

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

Sara Nayeem, MD
Partner
NEA

Adora Ndu, PharmD, JD
Executive Director, Regulatory Affairs, Policy,
Research, Engagement (PRE), & Reg Internationa
BioMarin Pharmaceutical

Nneka Onwudiwe, PharmD, PhD, MBA
Founder and Chief Executive Officer
Pharmaceutical Economics Consultants of America (PECA) LLC

Tejashri Purohit-Sheth, MD
Director, Division of Clinical Evaluation and
Pharmacology/Toxicology (CBER / OTAT / DCEPT)
U.S. Food and Drug Administration (FDA)

Gretchen Randlett
Clinical Trial Commercial Product Strategy and Process
Eli Lilly and Company

Steve Roberds
Chief Scientific Officer
Tuberous Sclerosis Alliance

Isaac R. Rodriguez-Chavez, Ph.D., M.H.Sc., M.Sc.
FDA Officer, Clinical Research Methodology, Regulatory Compliance
and Policy Development, CDER
U.S. Food and Drug Administration

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Khushboo Sharma, MBA, RAC
Deputy Office Director of Operations, Office of New Drugs,
Center for Drug Evaluation and Research (CDER)
U.S. Food and Drug Administration (FDA)

R'Kes D. Starling, RPh, MBA
Founder and Chief Executive Officer
Reveles Clinical Services

Peter Stein
Director, Office of New Drugs, CDER
U.S. Food and Drug Administration (FDA)

Dan Takefman, Ph.D.
Head, Regulatory Affairs
Spark Therapeutics

Kevin Weatherwax
Co-PI, Transforming Expanded Access to Maximize Support
and Study (TEAMSS); Co-Chair, Expanded Access Oversight Committee
University of Michigan Health Systems

Monica Weldon
President, CEO & Founder
Bridge the Gap – SYNGAP Education and Research Foundation

Michael Werner
Co-Founder and Senior Policy Counsel
Alliance for Regenerative Medicine

Celia M. Witten, PhD, MD
Deputy Director, Center for Biologics Evaluation and Research (CBER)
U.S. Food and Drug Administration (FDA)

Janet Woodcock
Director, Center for Drug Evaluation & Research (CDER)
U.S. Food and Drug Administration (FDA)