A Program of the EveryLife Foundation for Rare Diseases
2016 Rare Disease Week – Legislative Conference
More than 250 advocates attended the 2016 Legislative Conference on March 1st to learn about the legislative process, legislation currently under consideration by Congress, and effective advocacy techniques to build a relationship with Members of Congress and staff. The conference was crafted to educate advocates with little to no political experience and to provide useful updates to those with a history of political action to ensure that everyone was prepared for a successful Lobby Day.
Welcome and Overview
Moderator: Dean Suhr, Parent Advocate and President, MLD Foundation
Election Madness: Campaign Promises and Legislative Realities
The Appropriations Process and Federal Support for Medical Research: Caitlin Leach, Director of Policy and Advocacy, Research!America
What to Expect from Congress in Election Year:
Democratic Perspective: Emily Shetty, Consultant, the Stanton Park Group
Republican Perspective: Cheryl Jaeger, Principal, Williams & Jensen
Impact of Election Year Politics on Legislative Business and Congress
Importance of Congressional Committee
The Promise of Agency Partnership
How Patients Can Engage with Food and Drug Administration (FDA): Gayatri Rao, M.D., J.D., Director for the Office of Orphan Products Development, FDA
Prescription Drug User Fee Act (PDUFA): Eric Gascho, Vice President of Government Affairs, National Health Council (NHC)
Patient Focused Drug Development: Johanna Gray, Senior Vice President, CRD Associates
How Patients Can Engage with National Institutes of Health (NIH): Petra Kaufmann, M.D., M.Sc., Director of the Office of Rare Diseases Research and Division of Clinical Innovation, National Center for Advancing Translational Sciences, NIH
Rare Disease Legislation: A Historical Primer
Orphan Drug Act: Paul Melmeyer, Associate Director of Public Policy, National Organization for Rare Disorders (NORD)
Priority Review Vouchers: Ronald Bartek, Co-Founder/Founding President, Friedreich’s Ataxia Research Alliance (FARA)
What Comes First? Priorities for Future Legislative Efforts
Moderator: Max Bronstein, Senior Director of Public Policy and Government Relations, EveryLife Foundation for Rare Diseases
Rare Disease Caucus: Molly McDonnell, Legislative Assistant, Office of Representative Leonard Lance (NJ-7)
Orphan Product Extensions Now, Accelerating Cures and Treatments, OPEN Act (H.R. 971/S. 1421): Saul Hernandez, Deputy Chief of Staff and Legislative Director, Office of Representative G.K. Butterfield (NC-1)
Patient Focused Impact Assessment Act, PFIA Act (S. 1597): Annie Kennedy, Senior Vice President of Legislation and Public Policy, Parent Project Muscular Dystrophy
Rare Disease Fund Act of 2015 (H.R. 3731): Scott Hinkle, Legislative Director, Office of Representative Juan Vargas (C-51)
Putting Your Best Foot Forward: Tips and Tricks for a Successful Meeting
Building a Relationship with Members of Congress: Lisa Schill, Vice President, RASopathies Network USA
Sharing Your Two Minute Personal Story: Joseph LaMountain, Vice President, Reingold, Inc.
Congressional Score Cards, Hill Asks and Leave Behinds: Vignesh Ganapathy, J.D., Associate Director of Advocacy and Government Relations, EveryLife Foundation for Rare Diseases
Being an Effective Advocate: Stephanie Vance, Owner and Founder of Advocacy Associates
Scenes from the Hill: A Mock Legislative Meeting
Member of Congress: Jennifer Bernstein, Executive Vice President, Horizon Government Affairs
Legislative Aide: Taylor Gilliam, Principal, Inside Pitch Government Affairs
Advocate #1: Sarah Kucharski, CEO/Chairman and Founder, FMD Chat
Advocate #2: Robert Ostrea, Co-Founder and Board Member, Little Miss Hannah Foundation
Advocate #3: Michael Illions, Vice President and Director of Advocacy, Pediatric Hydrocephalus Foundation
Next Steps: Your Path to Success
Continuing Positive Engagement: Julia Jenkins, Executive Director, EveryLife Foundation for Rare Diseases
Effectively Utilizing Media and Social Media: Steve Weiss, Senior Vice President of Health at Edelman and Board Member, Charcot-Marie-Tooth Association
Multiple Paths to Success: Working with Rare Disease Partners
Brett Kopelan and Barbara Wuebbels - How to Work with Industry
Brett Kopelan Executive Director, dEBra of America, and Barbara Wuebbels, RN, MS, Vice President of Patient Advocacy, Audentes Therapeutics
What are the do’s and don’ts when trying to working with industry? In this session, learn how to create effective partnerships.
Stephanie Fischer - Advocacy on Social Media
Stephanie Fischer Senior Director of Patient Engagement and Communications, EveryLife Foundation for Rare Diseases
How can you use social media in just 15 minutes a day to raise awareness, make connections and advocate for or against legislation?
Jeff Allen, Ph.D. - Understanding FDA Review Pathways
Jeff Allen, Ph.D. Executive Director, Friends of Cancer Research
Accelerated Approval, biomarkers, clinical vs. surrogate endpoints, Fast Track designation, orphan designation, and Breakthrough Therapy designation– What does this all mean?
Rare Disease Issues, State by State
Kristin Parde - Modernizing Step Therapy
Kristin Parde, Deputy Vice President, State Advocacy – Policy, Pharmaceutical Research and Manufacturers of America (PhRMA)
Step Therapy requires that patients try a less costly treatment first before their insurance company will cover a more expensive therapy. What are the implications for patients?
Judith Mayer - Rare Disease State Advisory Council
Judith Mayer, Board Member, Taylor’s Tale
What is a Rare Disease State Advisory Council? How can you get one in your state?
Max Bronstein - Newborn Screening
Max Bronstein, Senior Director of Public Policy and Government Relations, EveryLife Foundation for Rare Diseases
Learn why newborn screening is important, who recommends which tests to perform, and how the current state-by-state system could be improved.