Documentary Screening

BREAKOUT SESSIONS

2:00 PM ET

LEGISLATIVE HOT TOPICS

• • • • Moderator: Liesl Broadbridge, ScM, CGC, Rutgers University Ryan Fisher, Parent Project Muscular Dystrophy John Richardson, National Society of Genetic Counselors Annie Kennedy, EveryLife Foundation for Rare Diseases

YOUNG ADULT REPRESENTATIVES OF RDLA (YARR): DEEP POLICY DIVE

• • • Moderator: Courtney Felle, EveryLife Foundation for Rare Diseases Christina Brundage, YARR Laura Romano, YARR

BREAKOUT SESSIONS

3:00 PM ET

HOW CONGRESS CAN WORK TO REDUCE BARRIERS TO CARE AND HEALTH DISPARITIES

• • • • • Introductions: Tamar Thompson, Alexion Pharmaceuticals Moderator: Linda Blount, Black Women’s Health Imperative Lauren Citron, Office of Representative Bobby Rush Shayne Woods, Office of Senator Tim Scott Daisy Kim, Asian and Pacific Islander American Health Forum

Presented by:

HOW DATA CAN POWER RARE DISEASE ADVOCACY

• • • • • • Moderator: Jamie Sullivan, EveryLife Foundation Eric Sid, National Center for Advancing Translational Sciences Melissa Haendel, University of Colorado Anschutz School of Medicine Ryan Shay, Faegre Drinker Marc Yale, International Pemphigus and Pemphigoid Foundation

NETWORKING HAPPY HOUR ON ADVOCACY AND POLICY TOPICS

4:00 P.M. ET

•State Advocacy • Moderators: Rachelle Raudes and Shannon von Felden, EveryLife Foundation for Rare Diseases

•How to Engage with State DURs and P&T Committees • Moderators: Baillie McGowan and Jack Meloro, EveryLife Foundation for Rare Diseases

•Newborn Screening RUSP Alignment • Moderators: Dylan Simon and Claire Ellis, EveryLife Foundation for Rare Diseases

•Starting Your Advocacy Journey as a New Rare Disease Advocate • Moderators: Sarah Tompkins; Rare Disease Week Chair, Sarita Edwards; Rare Disease Week Vice Chair and Katelyn Laws, EveryLife Foundation for Rare Diseasesw

YOUNG ADULT REPRESENTATIVES OF RDLA (YARR) MEETUP

5:15 P.M. ET

YARR Members will gather following the Legislative Conference to discuss the day’s events. Anyone 16-30 years old is welcome to attend whether or not they are a member of YARR.

Hill Day Issue Information

Legislative Ask #1

Cosponsor the Speeding Therapy Access Today Act of 2021, H.R. 1730/S. 670 STAT Act

SPEEDING THERAPY ACCESS TODAY

TALKING POINTS

 More than 30 million Americans are living with one or more rare disease.  Between 93% and 95% of the more than 7,000 known rare diseases have no U.S. Food and Drug Administration approved therapy.  The development process for a rare disease drug takes an average of 15 years.  Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together.  When new therapies for rare diseases are approved, patients often face unnecessary delays and barriers to access, resulting in avoidable deterioration in health.

STAT ACT VISION

 The STAT Act will enact targeted, impactful, and attainable policy reforms at the Food and Drug Administration (FDA) to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies.  Improve rare disease coordination, stakeholder engagement, and policy development within FDA by expanding existing authority to create a Rare Disease Center of Excellence.  Inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee.  Fund regulatory science and related activities to support development of therapies to treat very small rare disease populations.  Strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of pay.