A two-day program on the core issues in Expanded Access

Day 1 Sessions – Tuesday, January 22: Best practices for feasibility

The Big Picture: Engaging More Patients in Clinical Development (a fireside chat)

Janet Woodcock, MD, Director, Center for Drug Evaluation and Research FDA
Peter Pitts, President, Center for Medicine in the Public Interest

Early Treatment Access: The Regulation, Laws, and Politics
Starting with a set of exact terminology for use throughout the Summit, this session brings the audience fully up to speed on the origin, regulation, and general practice of pre-approval access. It differentiates single-patient “compassionate use” from traditional group-level “Expanded Access trials”. The panel will examine the politics around recent initiatives, including “Right to Try” and assess their impact on actual regulation and practice.

Steve Usdin, Senior Editor, BioCentury
Richard Klein, Former Division Director, FDA
Anne Cropp, PharmD, Chief Science Officer, Early Access Care LLC
David Farber, JD, Partner, King & Spalding
Beth Roxland, JD, Senior Advisor on Health Policy, Roxland Consultants, LLC

Strategic Application of Pre-Approval Access Programs
Is Expanded Access an option for your company / your therapeutic area / your medical practice? What countries should you run these programs in? When and how do you launch? The panel will map the range of use cases for U.S. and global pre-approval access programs. This session considers commercial feasibility and the range of regulatory channels to accommodate different kinds of sponsors and situations.

Karen Frascello, Vice President, Caligor Coghlan
Paul Aliu PharmD, Global Head of Medical Governance, Novartis
Marcel Van Kuijck, MD, PhD, VP UltraPrograms, Global Medical Affairs, Ultragenyx
Tom Watson, Executive VP, Bionical Group

The New Paradigm for Clinical Development:  Integrating Well-Designed Access Programs
Expanded Access planning is no longer a matter of responding to patients’ requests. It has become an integral part of the clinical development process. Built upon 30 years of case experience, new business frameworks aim to ensure that treatment-use of new medicines is achieved efficiently and in harmony with continued research. This session examines these frameworks from the perspectives of trial sites, health systems, and therapeutics companies.

Chris Beardmore, CEO, Anova LLC
Marjorie Speers, PhD, Executive Director, Clinical Research Pathways
Kevin Weatherwax, Adjunct Professor, University of Michigan Health System
Carrie Langlais Furr, PhD RAC, VP of Regulatory Affairs, AmpliPhi BioScience

Real World Data A:  EAPs -the New Wellspring of Valuable Secondary Data
The opportunities for real-world data and pre-approval access have always overlapped, but now they’re gaining widespread attention. Considering the vast size of past treatment programs in AIDS and cancer, Expanded Access may prove to be the dominant source of all pre-market treatment outcomes information. This session examines the regulatory and commercial value of outcomes data generated by Expanded Access programs.

Jonathan Darrow, JD, Faculty, Harvard Medical School
Dennis Akkaya, Director of Corporate Development, MyTomorrows
Amy Barone, MD, Medical Officer -Hematology and Oncology, FDA
Stuart Bell, PhD, Vice President, Inceptua Medicines Access

Real World Data B: Program Design for Optimal Evidence Generation
Converting data capture into actionable evidence requires design. The panelists will examine heterogeneity in target populations and the use of wider, more representative access programs to potentially reveal response covariates, biomarkers, and other discovery to inform the design of pivotal research studies. Pragmatic trial design may provide lessons for pre-market Expanded Access.

Karen Klein, US Head of Program Management, Clinigen
Michael Kurilla, MD, PhD, Director-Division of Clinical Innovation, NIH
Suanna Bruinooge, MPH, Director of Research -CENTRA, ASCO
Emily Speas, Senior Project Manager, ICON

The Economics and Marketplace for Exploratory Treatment
Are Expanded Access programs economically viable to their sponsors? Can drug companies afford to run them at a large enough size to meaningfully improve the set of options for patients? This session examines the practices of cost recovery and reimbursement, by which health systems, payers, consumers, and charitable collaborations contribute to the funding of scalable access solutions

Richard Klein, Former Division Director, FDA
Jess Rabourn, CFA, CEO, WideTrial Inc
Peter Pitts, President, Center for Medicine in the Public Interest
David Farber, JD, Partner, King & Spalding

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Day 2 Sessions – Wednesday, January 23: Bioethics and Policy

How to Value Exploratory Treatment and the Data it Generates (a fireside chat)

Steve Usdin, Senior Editor, BioCentury
Marc Boutin, JD, CEO, National Health Council

Early Treatment Access: Benefits, Burdens, Ethical Obligations, and the Duty to Protect – the Common Ground

John Carney, President and CEO, Center for Practical Bioethics

  • Protecting patients from harm and false claims
  • Unintended consequences of unregulated pre-market access
  • Right to try and the collection of data
  • Panel: How to ensure societal goods are not compromised

The Ethics of New Regulatory Processes and Pathways

Art Gertel, Partner, MedSciCom LLC
Richard Klein, Former director, FDA Office of Health and Constituent Affairs
Lisa Kearns, Senior Research Associate, NYU School of Medicine
Erika Segear, PhD, RAC, Assoc. Director of Regulatory Affairs, Duke School of Medicine

  • Group Expanded Access, N of 1 Expanded Access, DoD Battlefield Use
  • Transforming Expanded Access to Maximize Support and Study (TEAMSS) 
    NIH-NCATS commitments for expansion
  • Pragmatic design of treatment-use trials
  • Financial and medical risks to the patient
  • New circumstances under “Right to Try”


Methodologies to Provide Patients the Best Options for Treatment

Art Gertel, Partner, MedSciCom LLC
Larry Liberti, PhD, Exec. Director, Centre for Innovation in Regulatory Sciences
Brian Edwards, MD, VP, Alliance for Clinical Research Excellence and Safety 

  • Risk/benefit analysis- Considering disease rarity, disease severity, cost, and other factors in allocation of resources
  • Ethics reflections of experts in Health Technology Assessment
  • International perspectives on social values judgments

The Social Contract of Expanded Access, Part A: Roles and Ethical Responsibilities of Investigators

Andrew Shuman, MD, FACS, Chief of Clinical Ethics Service, University of Michigan Medical School

  • Safeguarding against undue product claims and promotion
  • The role of individual decision making in defining standard of care
  • Exploratory treatment options at non-research centers

The Social Contract of Expanded Access, Part B: Patient Agency

Jeff Leider, Executive Director, Let Them Be Little X2 Foundation
Kelly Ranallo, President, Turner Syndrome Global Alliance

  • Pre-engagement public education on Expanded Access
  • Post-engagement, redefining relationships between subject and researcher
  • “Participatory research” and reshaping the consent process
  • Inclusiveness and diversity in the overall clinical development process

Addressing High Patient Need and High Patient Vulnerability

Art Gertel, Partner, MedSciCom LLC
Naomi Lopez Bauman, Director of Healthcare Policy, Goldwater Institute

  • The impact of privacy protections (GDPR, Policy 70, HIPAA)
  • The valuation of data, consent, and privacy
  • “Participatory research” and reshaping the consent process

Conference Wrap-Up Discussion: The Future Role of Pre-Approval Access Programs in the Clinical Development Process

Jess Rabourn, CFA, CEO, WideTrial Inc.
John Carney, President and CEO, Center for Practical Bioethics

  • A roundtable with all panelists and conference attendees

We look forward to seeing you in WASHINGTON!

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