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EXPANDED ACCESS 2.0 AGENDA

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, Politics, and Law
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

  • A clear review of the rules and practice of pre-approval access in the U.S.
  • Cohort Expanded Access versus Individual “Compassionate Use”
  • Past and present initiatives to address perceived barriers
  • Federal “Right to Try”: big impact or big sideshow?
Global Pre-Market Access Strategies
Paul Aliu PharmD, Global Head of Medical Governance, Novartis
Tom Watson, Executive VP, Bionical Group
Karen Frascello, Vice President, Caligor Coghlan
Richard Moscicki, MD, Chief Medical Officer, PhRMA

  • The core differences between U.S. and ex-U.S. pre-approval access
  • Strategies for engaging markets at the right time and right scale
  • Special considerations for global expanded access campaigns
  • Commercial stage market access strategies
Integrating Expanded Access into the Drug Development Process
Marjorie Speers, PhD, Executive Director, Clinical Research Pathways
Kevin Weatherwax, Adjunct Professor, University of Michigan Health System
Hank Mansbach, MD, VP of Clinical Development, Ultragenyx
Carrie Langlais Furr, PhD RAC, VP of Regulatory Affairs, AmpliPhi BioScience

  • Feasibility factors and best practices established at the 2017 EA Summit
  • Lessons from the first 30 years of regulated Expanded Access
  • Prevailing narratives and trends: harmful or helpful?
  • New frameworks for networked clinics and IRBs
Real World Data: Learning from Patients
Jonathan Darrow, JD, Faculty Member, Harvard Medical School
Amy Barone, MD, Medical Officer -Hematology and Oncology, FDA
Michael Kurilla, MD PhD, Director-Division of Clinical Innovation, NIH
Suanna Bruinooge, MPH, Director of Research -CENTRA, ASCO
Kevin Mayo, PhD, Global Head -Market Access, PTC Therapeutics

  • Designing cohort Expanded Access programs for optimum data capture
  • Expanded Access as a (dominant?) resource for real world insights
  • Adaptive clinical development strategies informed by treatment outcomes
  • The valuation of data, consent, and privacy
The Economics and Marketplace for Exploratory Treatment
Jess Rabourn, CFA, CEO, WideTrial Inc
Richard Klein, Former Division Director, FDA
Peter Pitts, President, Center for Medicine in the Public Interes
Richard Moscicki, MD, Chief Medical Officer, PhRMA

  • Collaborative Expanded Access initiatives, sharing the costs
  • Paying for proven remedy vs. paying for exploratory options
  • “Reasonable and Necessary” with evidence development
  • New approaches to reimbursement
Putting it Together: Emerging Approaches to Expanded Access
Kevin Weatherwax, Adjunct Professor, University of Michigan Health System
Peter Pitts, President, Center for Medicine in the Public Interest
Stuart Bell, PhD, VP, Inceptua Medicines Access
Dennis Akkaya, Director of Corporate Development, MyTomorrows

  • Existing case examples and their uptake by industry and medicine
  • Proposed new business frameworks for manufacturers and health systems
  • A new paradigm for clinical development in difficult areas
  • Roundtable: solving the barriers to wider access and evidence generation

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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

Kate Goodrich, MD, Director, Center for Clinical Standards and Quality, CMS
Steve Usdin, Senior Editor, BioCentury

Early Treatment Access: Benefits, Burdens, Ethical Obligations, and the Duty to Protect – the Common Ground
Richard Payne, MD, John B. Francis Chair, 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 Constitue
Amanda Parrish, PhD, Director of Medical Affairs, Duke Medical S
Lisa Kearns, Senior Research Associate, NYU 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
Greg Koski, MD, PhD, CEO, Alliance for Clinical Research Excellence and Safety

  • 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
Behtash Bahador, Senior Manager, CISCRP
Jeff Leider, Executive Director, Let Them Be Little X2 Foundation

  • 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
Richard Payne, MD, John B. Francis Chair, Center for Practical Bioethics

  • 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.
Richard Payne, MD, Chair of Bioethics, Center for Practical Bioethics

  • A roundtable with all panelists and conference attendees

We look forward to seeing you in WASHINGTON!

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