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
Breakout Sessions
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.
