Benefit-Risk Boot Camp

bootcampBuilding a Robust Patient Profile of Benefit-Risk in Your Community

September 23, 2014 | 8:45 a.m. - 5:30 p.m.
Milken Institute School of Public Health at George Washington University | Washington, D.C.

This one-day event was designed specifically for patient organization research and engagement staff members, patient advocates, policy makers, and other stakeholders to hear directly from experts about how to participate effectively in research and policy activities that can enhance patient-centered decision-making about medical products in development, under regulatory review, and on the market.

Read more about our speakers


tumblr inline nd8h1xPjMn1rgq43d**Click the session titles for panel summaries

Session 1   Understanding Benefit-Risk Assessment in Medical Product R&D

Objective: Describe how benefits and risks are assessed by pharmaceutical companies and regulators as a product is being developed, during clinical trials, and across the life cycle for approved therapies.

  • Robert Metcalf, PhD, Vice President, Global Regulatory Affairs, Eli Lilly & Company – Overview of lifecycle approach to benefit-risk assessment
  • Rebecca Noel, DrPH, MPH, Senior Research Scientist, Global Patient Safety, Eli Lilly & Company – Benefit-risk assessment in early and late-stage development
  • Frank Rockhold, PhD, Senior Vice President, Global Clinical Safety and Pharmacovigilance, GlaxoSmithKline – Benefit-risk assessment post-marketing approval

 Moderated Question & Answer Session

  • Kristin Van Goor, PhD, RAC, Assistant Vice President of Scientific and Regulatory Affairs, Pharmaceutical Research and Manufacturers of America



tumblr inline nd8h7k0Yor1rgq43dSession 2  The Science of Eliciting Patient Preferences in Benefit-Risk

Objective: Provide a common understanding of methods used to understand patient preferences for benefit expectations and risk tolerance, how preferences can inform product development, and current gaps in the knowledge base.

  • John F.P. Bridges, PhD, Associate Professor, Johns Hopkins Bloomberg School of Public Health
  • Bennett Levitan, MD, PhD, Director, Quantitative Safety Research, Janssen R&D of Johnson & Johnson
  • Bill Murray, President & CEO, Medical Device Innovation Consortium

  Moderated Question & Answer Session

  • Marilyn Metcalf, PhD, Senior Director, Benefit-Risk Evaluation, GSK



tumblr inline ndehvuaOFz1rgq43dSession 3  Post-Market Applications of Benefit-Risk Assessment

Objective: Describe how information about benefits and risks is used to evaluate safety, effectiveness, and value by patients, prescribers, and payers.

Moderated Question & Answer Session

  • Lona L. Vincent, MPH, Senior Associate Director, Michael J. Fox Foundation for Parkinson’s Research



tumblr inline ndehkkLdQf1rgq43dSession 4   Key FDA Programs

Objective: Describe FDA initiatives at CDER and CDRH to incorporate patient perspectives in decision-making and to articulate assessment of benefit-risk in a consistent manner across divisions in each center, as well as FDA’s Patient Representative Program.

  • Steven Immergut, MPH, Associate Commissioner for External Affairs, Office of the Commissioner – Patient Representative Program
  • Jeffrey E. Shuren, MD, JD, Director, Center for Devices and Radiologic Health – Patient Preference Initiative
  • Janet Woodcock, MD, Director, Center for Drug Evaluation and Research – Structured benefit-risk assessment and Patient-Focused Drug Development
  • Moderator – Margaret Anderson, MA, Executive Director, FasterCures



tumblr inline ndei5tj2tt1rgq43dSession 5   Successful Patient Engagement in Drug Development

Objective: Describe current trends in patient engagement and ways in which patient perspectives are influencing drug development.

  • Marc Boutin, JD, Executive Vice President & Chief Operating Officer, National Health Council
  • Bray Patrick-Lake, Director of Stakeholder Management, Clinical Trials Transformation Initiative
  • Anthony Yanni, MD, MBA, Head, Patient Value and Strategy, Sanofi
  • Moderator – Sue Sheridan, MBA, MIM, Director of Patient Engagement, Patient-Centered Outcomes Research Institute



tumblr inline ndeifmzBFG1rgq43dSession 6   Case Study: Duchenne Muscular Dystrophy

Objective: Illustrate a pioneering collaboration among patient advocates, academics, and policy experts to advance drug development for this serious and ultimately fatal genetic disorder.

  • John F.P. Bridges, PhD, Associate Professor, Johns Hopkins Bloomberg School of Public Health
  • Timothy Franson, MD, Chief Medical Officer, YourEncore
  • Holly Peay, MS, CGC, Vice President of Outreach & Education, Parent Project Muscular Dystrophy
  • Moderator – K. Kimberly McCleary, Director of Strategic Initiatives, FasterCures



tumblr inline ndeirqSAWx1rgq43dSession 7   Regulatory Policy 101

Objective: Provide an overview of ways that industry and patient organizations can influence Congressional action and agency policy that leads to better access to treatments patients value.

  • Diane Dorman, Vice President of Public Policy, National Organization for Rare Diseases
  • Andrew Emmett, MPH, Managing Director, Science & Regulatory Affairs, Biotechnology Industry Organization
  • Sascha Haverfield, PhD, Vice President, Scientific & Regulatory Affairs, Pharmaceutical Research and Manufacturers of America
  • Annie Kennedy, Senior Vice President – Legislation & Public Policy, Parent Project Muscular Dystrophy
  • Moderator – Robert Metcalf, PhD, Vice President, GlobalRegulatory Affairs, Eli Lilly & Company