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

Day 1 - Evening

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

MODULE A: The Pre-Workshop

Global Regulation and Industry Best Practices

6:00 – 6:15PM

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

6:15 - 6:30PM

Why we have pre-market access to medicines: An introduction

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

6:30 – 7:00pm

U.S. Expanded Access Regulation: 30+ years of regulation, plus recent updates including "Right to Try" and Project Facilitate

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

7:00 – 7:45pm

Global Regulatory Landscapes: Europe, Americas, Asia

PRESENTED BY
David Schwicker, PhD Principal and Founder, ORPHA Strategy Consulting
Connie Coulomb MBA Managing Partner, Couloumb Strategy Consulting
Georges Van Baelan President, Arctyrion, Inc.

7:45 – 7:55pm

Regulatory Q&A Review

PRESENTED BY
All Speakers

7:55– 8:10pm

BREAK

8:10 – 8:35pm

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

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

8:35 – 9:00pm

Business Agreements and IRB Coordination with Health Care Providers

PRESENTED BY
Erika Segear, PhD, RAC Associate Director of Regulatory Affairs, Duke University School of Medicine
Chris Beardmore CEO, Anova LLC

Z

9:00PM

Workshop Adjourned

Tuesday, Jan. 28 - MORNING & AFTERNOON

Day 2 - Morning & Afternoon

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

MODULE 1: The Global Stakeholder Forum

Integrating Expanded Access into the Clinical Development Process

8:20 – 8:30am

Opening Remarks

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

8:30 – 9:30am

Session 1

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
Anthony Fauci, MD Director, National Institute of Allergy and Infectious Diseases (NIAID) 1984-Present
Peter Barton Hutt, JD Senior Counsel, Covington & Burling LLP
Stuart Nightingale, MD Former FDA Associate Commissioner for Health Affairs  (1982 – 2000)
Marjorie Speers, PhD Executive Director, Clinical Research Pathways

9:30 – 10:05am

Session 2

Morning Keynote Address: Smarter, more inclusive drug development

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

10:05 – 10:30am

BREAK

10:30 – 11:30am

Session 3

Getting Past the Simple Narratives: Deeper looks at the opportunities, obstacles, politics, and competitive interests surrounding pre-market access

PRESENTED BY
Marc Boutin* Chief Executive Officer, National Health Council
Anne Cropp, PharmD Chief Science Officer, Early Access Care LLC
Jonathan Darrow, S.J.D. Faculty Member, Harvard Medical School
Richard Klein Former Director, Officer of Health and Constituent Affairs, FDA (1990-1997)
Tobias Polak PhD Candidate, Erasmus University

11:30am – 12:30pm

Session 4

Frameworks for Reimbursing Exploratory Treatment for Serious & Life-Threatening Health Conditions: Re-examining “Reasonable and Necessary” care options; coverage with evidence generation

PRESENTED BY
Jess Rabourn, CFA* Executive Producer, Expanded Access Summit | CEO, WideTrial, Inc.
Alexander Natz Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
Edmund Pezalla, MD, MPH Former National Medical Director and VP, Aetna (2007 – 2016)
Peter Pitts President, Center for Medicine in the Public Interest

12:30 – 1:15pm

Buffet Lunch

1:15 – 1:45pm

Session 5

Afternoon Keynote Address: Clinical discovery through treatment of wider ranges of patients

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

1:45 – 3:00pm

Session 6

The Integration of Treatment and Clinical Research: Informing and strengthening the clinical development of novel regulated products through data-generating treatment programs

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

3:00 – 3:10pm

BREAK

3:10 – 4:10pm

Session 7

Health Care Providers and Life Science Companies Partnering for Wider Access: Identifying the beneficiaries of pre-market access, and designing appropriate, sustainable partnerships

PRESENTED BY
Chris Beardmore* CEO, Anova LLC
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

Session 8

Addressing Population Needs: Pre-market access -- including Emergency Use Authorization -- to address supply shortages, underserved markets, contagion preparedness, and national defense

PRESENTED BY
Amar Bhat, PhD* Interim Executive Director, Reagan-Udall Foundation, FDA
John Lagus Managing Director of Business Development, Tanner Pharma Group
Hilary Marston, MD Medical Officer, National Institute of Allergy and Infectious Diseases (NIAID), NIH
Robert Walker, MD Director, Division of Clinical Development, Biomedical Advanced Research and Development Authority (BARDA), HHS

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 and Poster Session
  • Music, ping-pong tournament, and additional activities

6:00 - 8:00PM

MODULE B: The Innovators Showcase

Real Solutions 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

MODULE 2: The Interactive Forum

Charting the Course for Modern Clinical Development

8:30 – 8:40am

Recap of Day 1

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

8:40 – 9:45am

Session 1

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)
Christina Hartman, MPH Senior Director of Advocacy, The Assistance Fund
Naomi Lopez Bauman Director of Healthcare Policy, Goldwater Institute
Andrew Shuman, MD, FACS Chief of Clinical Ethics Services, University of Michigan Medical School

9:45 – 10:00am

BREAK

10:00 – 11:00am

Session 2

Expanded Access for Modern High-cost Therapeutics: New challenges with cutting edge biologics, including cell & gene therapies, nucleotide, and novel antibodies

PRESENTED BY
Khrystal K. Davis, JD* Founder & President of Texas Rare Alliance, Texas Newborn Screening Advisory Committee
Karen Frascello Director, Global Medical Affairs, Early Access Programs, Alnylam Pharmaceuticals
Jeff Leider President, X2, “Let Them Be Little” Foundation
Douglas Sproule, MD Vice President, Spinal Muscular Atrophy Therapeutic Area Head, AveXis

11:00am - 12:00pm

Session 3

Who Should Pay for Pre-Market Treatment Access Programs: If Cost Recovery is key to feasible access programs, then who should the costs be recovered from and under what circumstances?

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

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

Session 4

The Interactive Challenge; Breakout and Report
Assigned seating at tables representing model stakeholder organizations. Delegates present findings.

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

2:30 – 3:00pm

Session 5

Policy Recommendations / Resolutions: A moderated roundtable discussion with audience

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 Medical Governance, Chief Medical Office, Novartis Pharmaceuticals

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.