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Agenda

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

December 3-4, 2019
  • Arlington, VA

Agenda

Want more agenda details? Download the brochure.

DAY ONE TUESDAY, DECEMBER 3, 2019

11:45

Main Conference Registration

12:45

Opening Remarks for Shared Regulatory Sessions
at The Rare Disease and FDA CMS Summits

Nielsen Hobbs
Editor
The Pink Sheet

REGULATORY SPOTLIGHT

1:00

INTERACTIVE Q&A SESSION Advanced Therapeutics and Cell and Gene Therapies

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

1:45

INTERACTIVE Q&A SESSION Innovations, Trends and Insights

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

2:30

Networking and Refreshment Break

3:00

Evolve the Rare Disease Drug Review Process

PANELISTS:

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

Ron Cooper
President & CEO
Albireo Pharma

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

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

3:45

PANEL DISCUSSION Understand the Shifting Regulatory Policy Landscapefor Cell and
Gene Therapies

PANELISTS:

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

Dan Takefman, PhD
Head, Regulatory Affairs
Spark Therapeutics

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

4:15

Brief Refreshment Break

4:25

Conference Chair’s Opening Remarks

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

4:30

CASE STUDY Designing a Patient-Centric Rare Disease Pivotal Trial

Ron Cooper
President & CEO
Albireo Pharma

5:00

FIRESIDE CHAT Navigate Regulatory Strategies for Rare Disease Clinical Development

MODERATOR:

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

CONVERSATION CONTRIBUTORS:

Chris Garabedian
CEO
Xontogeny

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

5:45

Close of Day One

Networking, Wine and
Cheese Reception
immediately following the final session on day one

DAY TWO WEDNESDAY, DECEMBER 4, 2019

7:30

Continental Breakfast

8:00

Conference Chair’s Review of Day One

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

OPTIMIZE CLINICAL COLLABORATIONS AND
TRIAL DESIGN

8:15

Opportunities to Accelerate and De-Risk Rare Disease Drug Development

Sara Nayeem, MD
Partner
NEA

8:45

Elevate Clinical Collaborations — The PREVeNT Case

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

9:30

CASE STUDYDevelop Holistic Collaborations Between Advocates and Drug Developers

Brett Kopelan
Executive Director
debra of America

John Maslowski
CEO
Fibrocell

10:15

Networking and Refreshment Break

10:45

Develop Data-Driven Approaches to Optimize Clinical Recruitment and Patient Identification

Candace Lerman
Founder
Rare Candace

ELEVATE EXPANDED
ACCESS PROGRAMS FOR
RARE DISEASE THERAPIES

11:15

Optimize Planning for Expanded Access Programs (EAPs) During R&D

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

12:00

Networking Luncheon

1:00

Explore the RMAT Designation and Current Policy Initiatives

Lyndsey Scull
Vice President Communications
Alliance for Regenerative Medicine

RWE AND DATA-DRIVEN
STRATEGIES DRIVING
CLINICAL PROGRESS

1:45

PANEL DISCUSSIONOptimize Reimbursement and Access Through Proactive Planning in Clinical Development

MODERATOR:

Michael Eging
Executive Director
Rare Action Access Project

PANELISTS:

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

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

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

2:30

Networking and Refreshment Break

3:00

PANEL DISCUSSIONAddress Roadblocks in Successful Utilization of Registry and
Other Patient Data

PANELISTS:

Len Rosenberg
Head of Clinical Operations
Beat AML & Leukemia and Lymphoma Society

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Monica Weldon
CEO
Bridge the Gap – SYNGAP Education and Research Foundation

3:45

Drive Quality in Real in World Data (RWD) to Optimize Regulatory Approvals and Product Value Propositions

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

5:00

Close of Conference

Agenda

Want more agenda details? Download the brochure.

Want more agenda details? Download the brochure.

DAY ONE TUESDAY, DECEMBER 3, 2019

11:45

Main Conference Registration

12:45

Opening Remarks for Shared Regulatory Sessions
at The Rare Disease and FDA CMS Summits

Nielsen Hobbs
Editor
The Pink Sheet

REGULATORY SPOTLIGHT

1:00

INTERACTIVE Q&A SESSION Advanced Therapeutics and Cell and Gene Therapies

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

1:45

INTERACTIVE Q&A SESSION Innovations, Trends and Insights

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

2:30

Networking and Refreshment Break

3:00

Evolve the Rare Disease Drug Review Process

PANELISTS:

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

Ron Cooper
President & CEO
Albireo Pharma

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

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

3:45

PANEL DISCUSSION Understand the Shifting Regulatory Policy Landscapefor Cell and
Gene Therapies

PANELISTS:

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

Dan Takefman, PhD
Head, Regulatory Affairs
Spark Therapeutics

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

4:15

Brief Refreshment Break

4:25

Conference Chair’s Opening Remarks

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

4:30

CASE STUDY Designing a Patient-Centric Rare Disease Pivotal Trial

Ron Cooper
President & CEO
Albireo Pharma

5:00

FIRESIDE CHAT Navigate Regulatory Strategies for Rare Disease Clinical Development

MODERATOR:

Derrick Gingery
Senior Writer
The Pink Sheet and Scrip

CONVERSATION CONTRIBUTORS:

Chris Garabedian
CEO
Xontogeny

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

5:45

Close of Day One

Networking, Wine and
Cheese Reception
immediately following the final session on day one

DAY TWO WEDNESDAY, DECEMBER 4, 2019

7:30

Continental Breakfast

8:00

Conference Chair’s Review of Day One

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

OPTIMIZE CLINICAL COLLABORATIONS AND
TRIAL DESIGN

8:15

Opportunities to Accelerate and De-Risk Rare Disease Drug Development

Sara Nayeem, MD
Partner
NEA

8:45

Elevate Clinical Collaborations — The PREVeNT Case

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

9:30

CASE STUDYDevelop Holistic Collaborations Between Advocates and Drug Developers

Brett Kopelan
Executive Director
debra of America

John Maslowski
CEO
Fibrocell

10:15

Networking and Refreshment Break

10:45

Develop Data-Driven Approaches to Optimize Clinical Recruitment and Patient Identification

Candace Lerman
Founder
Rare Candace

ELEVATE EXPANDED
ACCESS PROGRAMS FOR
RARE DISEASE THERAPIES

11:15

Optimize Planning for Expanded Access Programs (EAPs) During R&D

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

12:00

Networking Luncheon

1:00

Explore the RMAT Designation and Current Policy Initiatives

Lyndsey Scull
Vice President Communications
Alliance for Regenerative Medicine

RWE AND DATA-DRIVEN
STRATEGIES DRIVING
CLINICAL PROGRESS

1:45

PANEL DISCUSSIONOptimize Reimbursement and Access Through Proactive Planning in Clinical Development

MODERATOR:

Michael Eging
Executive Director
Rare Action Access Project

PANELISTS:

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

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

Kari Rosbeck
President & CEO
Tuberous Sclerosis Alliance

2:30

Networking and Refreshment Break

3:00

PANEL DISCUSSIONAddress Roadblocks in Successful Utilization of Registry and
Other Patient Data

PANELISTS:

Len Rosenberg
Head of Clinical Operations
Beat AML & Leukemia and Lymphoma Society

Christian Rubio
Vice President Community Development & Engagement
Global Genes

Monica Weldon
CEO
Bridge the Gap – SYNGAP Education and Research Foundation

3:45

Drive Quality in Real in World Data (RWD) to Optimize Regulatory Approvals and Product Value Propositions

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

5:00

Close of Conference