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Expanded Access 3.0 Agenda

The Expanded Access Summit has a new format! Day 1’s Policy and Practice Workshop leads off with a thorough primer of the space. Day 2 will continue the Expanded Access Summit’s annual discussion on the integration of treatment and research. That evening, guests will enjoy the on-site Grand Reception and hear the latest industry developments at the Innovators Showcase. Day 3 is an interactive forum aimed at delivering modern guidance to industry and policymakers.

WHERE

National Press Club
Washington D.C.

WHEN

Monday through Wednesday
January 27-29, 2020

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DOWNLOAD THE AGENDA

Click here to download the EA 3.0 Conference Agenda in PDF form.

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Monday, Jan. 27 - EVENING (Primer / Workshop)

Day 1 - Evening

January 27, 2020
6:00PM - 9:00PM

SESSION A: The Pre-Workshop

Global Regulation and Industry Best Practices

6:00 – 6:30PM

Evening Check-in and Networking
  • Meet the event directors and speakers
  • Hors d’oeuvres, beverages

6:30 - 6:40PM

Introduction: Why do we have pre-market access?

PRESENTED BY
Marjorie Speers, PhD Executive Director, Clinical Research Pathways

6:40 – 7:10pm

US Regulation: 30 years of history with recent updates, including RTT and Project Facilitate

PRESENTED BY
Richard Klein Former Director, Patient Liaison Program, FDA
Amy Barone, MD Medical Officer, Oncology Center of Excellence, FDA

7:10 – 7:40pm

Global Regulatory Landscapes

PRESENTED BY
David Schwicker, PhD ORPHA Strategy Consulting

7:40– 8:00pm

BREAK

8:00– 8:20pm

Single Patient EA and Cohort EAPs: Knowing the difference and what each is intended for

PRESENTED BY
David Farber Senior Partner, King & Spalding

8:20 – 8:40pm

The Range of Uses for EA: Pharmaceuticals, devices, research-stage, post-research, new-use, new form,
etc.

PRESENTED BY
Kevin Weatherwax Managing Director, Michigan Institute for Clinical and Health Research, University of Michigan Health System
Karen Frascello Director, Global Medical Affairs, Early Access Programs

8:40 – 9:00pm

Business Agreements with Health Care Providers:

PRESENTED BY
Erika Segear, PhD, RAC Associate Director of Regulatory Affairs, Duke University School of Medicine

Z

9:00PM

Workshop Adjourned

Tuesday, Jan. 28 - MORNING & AFTERNOON

Day 2 - Morning & Afternoon

January 28, 2020
8:00AM - 5:00PM

SESSION 1: The Global Stakeholder Forum

Integrating Expanded Access into the Clinical Development Process

8:15 – 8:30am

Opening Remarks

PRESENTED BY
Jess Rabourn, CFA Executive Producer, Expanded Access Summit | CEO, WideTrial, Inc.

8:30 – 9:30am

Meeting the Original Promise of Expanded Access: How far have we come? Are we on the Right Track?

PRESENTED BY
Steve Usdin Senior Editor, BioCentury (Moderator)
Frank Young, MD, PhD EVP Clinical & Reg Affairs, TissueTech Inc.; Former FDA Commissioner, 1984-1989
Tony Fauci, MD Director, National Institute of Allergy and Infectious Diseases (NIAID) 1984-Present
Stuart Nightingale, MD Consultant on Public Health and Regulatory Affairs, NIH Former FDA Associate Commissioner for Health Affairs
Marjorie Speers, PhD Executive Director, Clinical Research Pathways

9:30 – 10:05am

Morning Keynote: Smarter, more inclusive drug development

PRESENTED BY
Janet Woodcock, MD Director, Center for Drug Evaluation and Research (CDER), FDA

10:05 – 10:30am

BREAK

10:30am – 11:30pm

Getting Past the Simple Narratives: Deeper Looks at the Opportunities, Obstacles, Politics, and Competitive Interests Surrounding Pre-Market Access

PRESENTED BY
Mark Boutin Chief Executive Officer, National Health Council
Anne Cropp, PharmD Chief Science Officer, Early Access Care LLC
Jonathan Darrow, JD Faculty Member, Harvard Medical School
Richard Klein Former Director, Patient Liaison Program, FDA

11:30am – 12:30pm

Frameworks for Reimbursing Exploratory Treatment for Serious & Life-Threatening Health Conditions: Re-Examining “Reasonable and Necessary” Care Options

PRESENTED BY
Jess Rabourn, CFA Executive Producer, Expanded Access Summit | CEO, WideTrial, Inc. (Moderator)
Peter Pitts President, Center for Medicine in the Public Interest
Alexander Natz Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
National Payer Representative, TBA

12:30 – 1:15pm

Buffet Lunch

1:15 – 1:45pm

Afternoon Keynote: Clinical Discovery in More-Representative Ranges of Patients

PRESENTED BY
Chris Austin, MD Director, National Center for Advancing Translational Sciences, NIH

1:45 – 3:00pm

The Integration of Treatment and Clinical Research

PRESENTED BY
Stuart Bell, PhD Vice President, Real World Evidence, Inceptua Medicines Access
Amy Barone, MD Medical Officer, Oncology Center of Excellence, FDA
Paul Aliu, PharmD Global Head of Medical Governance, Novartis Oncology
Michael Kurilla, MD, PhD Director, Clinical and Translational Science Awards (CTSA) Program, NCATS, NIH

3:00 – 3:10pm

BREAK

3:10 – 4:10pm

HCPs and Life Science Companies Partnering for Wider Access

PRESENTED BY
Suanna Bruinooge, MPH Director of Research Strategy, CENTRA, ASCO
Pat Furlong Founding President and CEO, Parent Project Muscular Dystrophy (PPMD)
Erika Segear, PhD, RAC Associate Director of Regulatory Affairs, Duke University School of Medicine
Kevin Weatherwax Managing Director, Michigan Institute for Clinical and Health Research, University of Michigan Health System

4:10 – 5:00pm

Addressing Population Needs: Supply Shortages, New Markets, Contagion, and BioPreparedness

PRESENTED BY
Amar Bhat, PhD Interim Executive Director, Reagan-Udall Foundation, FDA
Stuart Nightingale, M.D Former Associate Commissioner for Health Affairs, FDA (1982-2000)
John Lagus Managing Director of Business Development, Tanner Pharma Group
CDC Representative TBA, pending confirmation

Z

5:00PM

Session Adjourned

Tuesday, Jan. 28 - EVENING

Day 2 - Evening

January 28, 2020
5:15PM - 8:00PM

The Grand Reception & The Innovators Showcase

5:15 - 8:00PM

THE GRAND RECEPTION
  • Gourmet food and beverage
  • Exhibitor Galleries open
  • Music, ping-pong tournament, and additional activities

6:00 - 8:00PM

SESSION B: The Innovators Showcase

Real Solutions and Service Offerings Presented by Leaders in the Field

6:00 - 6:30PM

Set 1
  • Four presenters, schedule TBA
  • 6 minutes each, 1-minute turnaround

6:30 – 6:45pm

BREAK

6:45 – 7:15pm

Set 2
  • Four presenters, schedule TBA
  • 6 minutes each, 1-minute turnaround

7:15 – 7:30pm

BREAK

7:30 – 8:00pm

Set 3
  • Four presenters, schedule TBA
  • 6 minutes each, 1-minute turnaround
Z

8:00PM

Showcase Adjourned

Wednesday, Jan. 29 - MORNING & AFTERNOON

Day 3 - Morning & Afternoon

January 29, 2020
8:30AM - 3:00PM

SESSION 2: The Interactive Forum

Charting the Course for Modern Clinical Development

8:30 – 8:45am

Recap of Day 1

PRESENTED BY
Jess Rabourn, CFA Executive Producer, Expanded Access Summit | CEO, WideTrial, Inc.

8:45 – 10:00am

Inclusivity in Clinical Development: Should all patients be provided the option to participate? If so, how? What are the barriers?

PRESENTED BY
Behtash Bahador Director, Center for Information and Study on Clinical Research Participation (CISCRP)
Naomi Lopez Bauman Director of Healthcare Policy, Goldwater Institute
Christina Hartman, MPH Senior Director of Advocacy, The Assistance Fund
Andrew Shuman, MD, FACS Chief of Clinical Ethics Services, University of Michigan Medical School

10:00 – 10:20am

BREAK

10:20 – 11:00am

Case Analysis: Expanded Access for Spinraza, Elaprase and other high-cost biotherapeutics

PRESENTED BY
Khrystal K. Davis Founder & President of Texas Rare Alliance & Texas Newborn Screening Advisory Committee Member
Jeff Leider President, X2, “Let Them Be Little” Foundation
Karen Frascello Director, Global Medical Affairs, Early Access Programs

11:00am - 12:00pm

Discussion on Funding Access: If Cost Recovery is key to feasible access programs, who should the costs be recovered from?

PRESENTED BY
David Farber, JD Senior Partner, King & Spalding
Mark Boutin Chief Executive Officer, National Health Council
Marjorie Speers, PhD Executive Director, Clinical Research Pathways
National Payer Representative, TBA

12:00 - 12:15pm

Introduction to the interactive challenge

Behtash Bahador Director, Center for Information and Study on Clinical Research Participation (CISCRP)

12:15 - 1:15PM

Box Lunch

1:15 - 2:30PM

The Interactive Challenge: Breakout and Report

PRESENTED BY
Behtash Bahador Director, Center for Information and Study on Clinical Research Participation (CISCRP) (Moderator)

2:30 – 3:00pm

Policy Recommendations / Resolutions: Moderated round-table forum

PRESENTED BY
Steve Usdin Senior Editor, BioCentury
Peter Pitts President, Center for Medicine in the Public Interest
Pat Furlong Founding President and CEO, Parent Project Muscular Dystrophy (PPMD
Paul Aliu, PharmD Global Head of Medical Governance, Novartis Oncology

Z

3:00PM

Expanded Access Summit Adjourned

DOWNLOAD THE AGENDA

Click here to download the EA 3.0 Conference Agenda in PDF form.

We look forward to seeing you in WASHINGTON!

Register now to ensure best price admission and hotel room availability.