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Agenda

Design Sustainable Early Access Programs to Manage Global Complexities and Advance Real World Evidence

March 28-29, 2018
  • Washington, DC

Agenda

Want more agenda details? Download the brochure.

DAY ONE Wednesday, March 28, 2018

7:30

Registration and Continental Breakfast

8:30

Chairperson’s Welcome and Opening Remarks

Beverly Harrison
Head of Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

8:45

WASHINGTON UPDATE PANEL SESSION
Multi-Stakeholder Perspectives on Legislation Impacting the Future of
Expanded Access

MODERATOR:

Ramana Sonty
Director, Global Medical Organization
Johnson & Johnson

PANELISTS:

Cartier Esham
Executive Vice President, Emerging Companies,
Vice President, Science and Regulatory Affairs
Biotechnology Innovation Organization (BIO)

Elena Gerasimov, MA, MPH
Director of Programs
Kids v Cancer

Richard Klein
Former Director, Patient Liaison
FDA

Emma James, PhD
Senior Director, Medical Affairs & Health Outcomes
Audentes Therapeutics, Inc.

9:45

Navigate the Landscape for U.S. and Global Pre-Approval Access

Paul Aliu, PharmD, MBA
Global Head, Medical Governance, Chief Medical Office
Novartis Pharma AG

10:30

Networking and Refreshment Break

11:00

Evolving the Scope of the Expanded Access Navigator

June Wasser
Executive Director/CEO
Reagan-Udall Foundation

11:45

Networking Luncheon

1:00

Clarifying the Regulatory Framework for Expanded Access —
Addressing Barriers to Access

Richard Klein
Former Director, Patient Liaison
FDA

2:00

Making an Impact with EAPs

Anne B. Cropp
Chief Scientific Officer
Early Access Care

2:45

Integrate Expanded Access into Your Drug Development Process

Kristin Van Goor
Senior Director, Global Regulatory Policy, US Lead
Biogen

3:30

Networking and Refreshment Break

4:00

CO-PRESENTATION
Removing Barriers to Access —
Understand the Clinician’s Perspective on Single-Patient Access and Group EAPs

Kevin Weatherwax
Director IND/IDE Assistance Program
University of Michigan Health System

Erika Segear Johnson, PhD, RAC
Associate Director, Regulatory Affairs, Office of Regulatory Affairs and Quality
Duke University School of Medicine

5:00

Close of Day One

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

DAY TWO Thursday, March 29, 2018

7:30

Continental Breakfast

8:15

Chairperson’s Review of Day One

Beverly Harrison
Head of Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

8:30

PANEL DISCUSSION
Managing Ethical Considerations for Expanded Access Programs

MODERATOR:

Alison Bateman-House
Research Assistant Professor, Division of Medical Ethics
NYU

PANELISTS:

Myra R. Herrle, PhD, RPh, RAC
Senior Director, Global Regulatory Strategy (GRS)
AbbVie Inc.

Christine MacCracken
Director, Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

Paul Aliu, PharmD, MBA
Global Head Medical Governance, Chief Medical Office
Novartis Pharma AG

Paul Melmeyer
Director, Federal Policy
National Organization of Rare Disorders

9:30

Launching and Sustaining Expanded Access Programs —
Pitfalls and Lessons Learned

Hank Mansbach
Vice President, Clinical Development
Ultragenyx Pharmaceutical Inc.

10:15

Networking and Refreshment Break

10:45

Demystify Global Complexities in Expanded Access

Mariangela Demarcus, PhD
Associate Director, Managed Access Programs, Ph IV & Medical Strategy
Vertex Pharmaceuticals (Europe) Limited

11:30

CONCURRENT WORKING SESSIONS Select one session for an interactive, deep-dive, facilitated discussion

A Operationalizing Global Expanded Access Programs

Gretchen M. Randlett
Consultant – Clinical Trial Commercial Product Strategy and Process
Eli Lilly and Company

B Multi-Stakeholder Perspectives on Pricing and Reimbursement of Unapproved Medicines

Brenda Gleason
President, M2 Health Care Consulting;
Professorial Lecturer, GWU School of Public Health

12:30

Networking Luncheon

1:30

Economic Sustainability and Expanded Access Programs

Jess Rabourn
Co-founder and Managing Director
WideTrial

2:15

PANEL DISCUSSION Define the Parameters of Data Collection for Expanded Access — Advancing the Generation of Real-World Evidence to Address Multi-Stakeholder Needs

MODERATOR:

Speaker TBA

PANELISTS:

Kristin Van Goor
Senior Director, Global Regulatory Policy, US Lead
Biogen

Hank Mansbach
Vice President, Clinical Development
Ultragenyx Pharmaceutical Inc.

Jess Rabourn
Co-founder and Managing Director
WideTrial

Jennifer McNary
Board Member, Beauhawks Foundation; and Manager
JMcNary Consulting

3:00

Close of Conference

Agenda

Want more agenda details? Download the brochure.
Want more agenda details? Download the brochure.

DAY ONE Wednesday, March 28, 2018

7:30

Registration and Continental Breakfast

8:30

Chairperson’s Welcome and Opening Remarks

Beverly Harrison
Head of Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

8:45

WASHINGTON UPDATE PANEL SESSION
Multi-Stakeholder Perspectives on Legislation Impacting the Future of
Expanded Access

MODERATOR:

Ramana Sonty
Director, Global Medical Organization
Johnson & Johnson

PANELISTS:

Cartier Esham
Executive Vice President, Emerging Companies,
Vice President, Science and Regulatory Affairs
Biotechnology Innovation Organization (BIO)

Elena Gerasimov, MA, MPH
Director of Programs
Kids v Cancer

Richard Klein
Former Director, Patient Liaison
FDA

Emma James, PhD
Senior Director, Medical Affairs & Health Outcomes
Audentes Therapeutics, Inc.

9:45

Navigate the Landscape for U.S. and Global Pre-Approval Access

Paul Aliu, PharmD, MBA
Global Head, Medical Governance, Chief Medical Office
Novartis Pharma AG

10:30

Networking and Refreshment Break

11:00

Evolving the Scope of the Expanded Access Navigator

June Wasser
Executive Director/CEO
Reagan-Udall Foundation

11:45

Networking Luncheon

1:00

Clarifying the Regulatory Framework for Expanded Access —
Addressing Barriers to Access

Richard Klein
Former Director, Patient Liaison
FDA

2:00

Making an Impact with EAPs

Anne B. Cropp
Chief Scientific Officer
Early Access Care

2:45

Integrate Expanded Access into Your Drug Development Process

Kristin Van Goor
Senior Director, Global Regulatory Policy, US Lead
Biogen

3:30

Networking and Refreshment Break

4:00

CO-PRESENTATION
Removing Barriers to Access —
Understand the Clinician’s Perspective on Single-Patient Access and Group EAPs

Kevin Weatherwax
Director IND/IDE Assistance Program
University of Michigan Health System

Erika Segear Johnson, PhD, RAC
Associate Director, Regulatory Affairs, Office of Regulatory Affairs and Quality
Duke University School of Medicine

5:00

Close of Day One

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

DAY TWO Thursday, March 29, 2018

7:30

Continental Breakfast

8:15

Chairperson’s Review of Day One

Beverly Harrison
Head of Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

8:30

PANEL DISCUSSION
Managing Ethical Considerations for Expanded Access Programs

MODERATOR:

Alison Bateman-House
Research Assistant Professor, Division of Medical Ethics
NYU

PANELISTS:

Myra R. Herrle, PhD, RPh, RAC
Senior Director, Global Regulatory Strategy (GRS)
AbbVie Inc.

Christine MacCracken
Director, Patient Support, Office of the Chief Medical Officer
Johnson & Johnson

Paul Aliu, PharmD, MBA
Global Head Medical Governance, Chief Medical Office
Novartis Pharma AG

Paul Melmeyer
Director, Federal Policy
National Organization of Rare Disorders

9:30

Launching and Sustaining Expanded Access Programs —
Pitfalls and Lessons Learned

Hank Mansbach
Vice President, Clinical Development
Ultragenyx Pharmaceutical Inc.

10:15

Networking and Refreshment Break

10:45

Demystify Global Complexities in Expanded Access

Mariangela Demarcus, PhD
Associate Director, Managed Access Programs, Ph IV & Medical Strategy
Vertex Pharmaceuticals (Europe) Limited

11:30

CONCURRENT WORKING SESSIONS Select one session for an interactive, deep-dive, facilitated discussion

A Operationalizing Global Expanded Access Programs

Gretchen M. Randlett
Consultant – Clinical Trial Commercial Product Strategy and Process
Eli Lilly and Company

B Multi-Stakeholder Perspectives on Pricing and Reimbursement of Unapproved Medicines

Brenda Gleason
President, M2 Health Care Consulting;
Professorial Lecturer, GWU School of Public Health

12:30

Networking Luncheon

1:30

Economic Sustainability and Expanded Access Programs

Jess Rabourn
Co-founder and Managing Director
WideTrial

2:15

PANEL DISCUSSION Define the Parameters of Data Collection for Expanded Access — Advancing the Generation of Real-World Evidence to Address Multi-Stakeholder Needs

MODERATOR:

Speaker TBA

PANELISTS:

Kristin Van Goor
Senior Director, Global Regulatory Policy, US Lead
Biogen

Hank Mansbach
Vice President, Clinical Development
Ultragenyx Pharmaceutical Inc.

Jess Rabourn
Co-founder and Managing Director
WideTrial

Jennifer McNary
Board Member, Beauhawks Foundation; and Manager
JMcNary Consulting

3:00

Close of Conference