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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

7:30

Registration and Continental Breakfast

8:30

Chairman’s Welcome & Opening Remarks for Shared Sessions with the FDA/CMS Summit

Nielsen Hobbs
Editor
Pink Sheet

8:45

OPENING KEYNOTE ADDRESS

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

9:15

CDER KEYNOTE ADDRESS 2019 and the Year Ahead — Strategic Plans and Priorities

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

10:00

FIRESIDE CHAT Updates on the Innovation and Investment Summit

Nielsen Hobbs
Editor
Pink Sheet

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

10:30

Networking and Refreshment Break

11:00

CDER OFFICE OF NEW DRUGS UPDATE 2019 Update, 2020 Strategic Plans and Priorities

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)

11:45

Main Conference Registration

12:45

Chairperson’s Afternoon Remarks

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

4:30

Conference Chair’s Opening Remarks

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

4:35

CASE STUDY Designing a Patient-Centric Rare Disease Pivotal Trial

Ron Cooper
President & CEO
Albireo Pharma

5:05

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)

5:50

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

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

9:15

CASE STUDYDevelop Holistic Collaborations Between Advocates and Drug Developers

Brett Kopelan
Executive Director
debra of America

John Maslowski
CEO
Fibrocell

9:45

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

Candace Lerman
Founder
Rare Candace

10:15

Networking and Refreshment Break

ELEVATE EXPANDED
ACCESS PROGRAMS FOR
RARE DISEASE THERAPIES

10:45

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

MODERATOR:

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

PANELISTS:

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

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

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

11:30

Strengthen Rare Disease Clinical Research by Leveraging Integrated Technology and In-Home Support to Enhance Patient Recruitment and Retention

MODERATOR:

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

CONVERSATION CONTRIBUTORS:

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

Wes Michael
President & Founder
Rare Patient Voice, LLC

12:15

Networking Luncheon

1:15

FDA Perspective — Decentralized Clinical Trials in Rare Diseases – Key Principles

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

RWE AND DATA-DRIVEN
STRATEGIES DRIVING
CLINICAL PROGRESS

2:00

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

2:45

Networking and Refreshment Break

3:15

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

PANELISTS:

Christian Rubio
Vice President Community Development & Engagement
Global Genes

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

Katherine Leon
Co-Founder and Board Chair
SCAD Alliance

Steve Roberds
Chief Scientific Officer
Tuberous Sclerosis Alliance

4:00

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

4:30

Close of Conference

Agenda

Want more agenda details? Download the brochure.

Want more agenda details? Download the brochure.

DAY ONE TUESDAY, DECEMBER 3, 2019

7:30

Registration and Continental Breakfast

8:30

Chairman’s Welcome & Opening Remarks for Shared Sessions with the FDA/CMS Summit

Nielsen Hobbs
Editor
Pink Sheet

8:45

OPENING KEYNOTE ADDRESS

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

9:15

CDER KEYNOTE ADDRESS 2019 and the Year Ahead — Strategic Plans and Priorities

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

10:00

FIRESIDE CHAT Updates on the Innovation and Investment Summit

Nielsen Hobbs
Editor
Pink Sheet

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

10:30

Networking and Refreshment Break

11:00

CDER OFFICE OF NEW DRUGS UPDATE 2019 Update, 2020 Strategic Plans and Priorities

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)

11:45

Main Conference Registration

12:45

Chairperson’s Afternoon Remarks

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

4:30

Conference Chair’s Opening Remarks

Ali Mohamadi
Executive Director, Patient Advocacy
BioMarin Pharmaceutical

4:35

CASE STUDY Designing a Patient-Centric Rare Disease Pivotal Trial

Ron Cooper
President & CEO
Albireo Pharma

5:05

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)

5:50

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

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

9:15

CASE STUDYDevelop Holistic Collaborations Between Advocates and Drug Developers

Brett Kopelan
Executive Director
debra of America

John Maslowski
CEO
Fibrocell

9:45

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

Candace Lerman
Founder
Rare Candace

10:15

Networking and Refreshment Break

ELEVATE EXPANDED
ACCESS PROGRAMS FOR
RARE DISEASE THERAPIES

10:45

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

MODERATOR:

Jaswinder Singh Khera
Managing Director Americas
WEP Clinical

PANELISTS:

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

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

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

11:30

Strengthen Rare Disease Clinical Research by Leveraging Integrated Technology and In-Home Support to Enhance Patient Recruitment and Retention

MODERATOR:

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

CONVERSATION CONTRIBUTORS:

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

Wes Michael
President & Founder
Rare Patient Voice, LLC

12:15

Networking Luncheon

1:15

FDA Perspective — Decentralized Clinical Trials in Rare Diseases – Key Principles

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

RWE AND DATA-DRIVEN
STRATEGIES DRIVING
CLINICAL PROGRESS

2:00

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

2:45

Networking and Refreshment Break

3:15

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

PANELISTS:

Christian Rubio
Vice President Community Development & Engagement
Global Genes

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

Katherine Leon
Co-Founder and Board Chair
SCAD Alliance

Steve Roberds
Chief Scientific Officer
Tuberous Sclerosis Alliance

4:00

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

4:30

Close of Conference