June 17, 2014

1. June 17, 2014

2. Agenda • Rare Action Network - Lisa Phelps, NORD • House Committee requests a GAO report on Compassionate Access  - Andy Taylor, Legislative Director - Office of Congressman Michael McCaul • S. 323/H.R. 1428 Comprehensive Immunosuppressive Drug Coverage Act of 2013 - Michelle Davis, PKD Foundation • CAL Undiagnosed - Taylor Gilliam, Legislative Assistant - Office of Congressman John Carter • FDA Finalizes Expedited Approval Process - Julia Jenkins, EveryLife Foundation Updates From RDLA: • RDLA's In-District Lobby Days • Abbey Nominations Open for the 2014 RareVoice Awards Gala 

3. House Committee Requests a GAO Report on Compassionate Access  • Andy Taylor, Legislative Director Office of Congressman Michael McCaul

4. FDA’s Guidance on Expedited Programs for Serious Conditions — Drugs and Biologics • FDASIA signed by the President in 2012 included ULTRA/FAST Legislation, which required the FDA to issue a guidance on Accelerated Approval that specifically addresses rare diseases drug development issues. • FDA issued Draft Guidance on Expedited Approval in June 2013: • Fast Track • Accelerated Approval (ULTRA/FAST) • Priority Review Vouchers (Creating Hope Act) • Breakthrough Therapy Designation • Did not address issues specific to rare diseases

5. FDA Finalizes Expedited Approval Guidance • 109 Members of Congress sent a letter to FDA asking them to revise the guidance to fulfil the intent of FDASIA & give Rare Disease Access to the Accelerated Approval Pathway • EveryLife Foundation drafted a White Paper that could be used to help create the scientific framework to give rare diseases access to Accelerated Approval • Submitted for Publishing with Orpha.Net • FDA Issued Final Guidance: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf

6. FDA Finalizes Expedited Approval Guidance • Guidance is improved but still does not provide scientific framework for using Accelerated Approval • Recent FDA decisions on Duchene treatments signal FDA may be moving in a positive direction • FDA-agricultural funding bill included language expressing concerned the FDA is under utilizing its Accelerated Approval authority given to the agency under FDASIA & directs FDA to report on the way it has used this authority since 2012, its plans to use it in the future, and a justification for using this authority for diseases that are not life-ending

7. FDA Finalizes Expedited Approval Guidance • Improvements • Revisions to the Section VII Accelerated Approval introduction • New development of Section 3 under Section VII Part A • Surrogate and Intermediate clinical endpoint examples provided • Not Included: • To Systematically Collect Patient Data or not? • Individual Case Considerations/ Lack of a Clearly Articulated Process and Requirements No Biomarker Qualification Request Form and Process

8. NEW THIS YEAR! RDLA’s In-District Lobby DaysCongress comes to you during August Recess • Meeting with your Member of Congress once a year is not enough! • Strengthen relationships with Relationships with your Members of Congress • Build on the Success of Rare Disease Day Lobby Days in Washington DC • Allows those who can’t make it to D.C. to have an opportunity to meet with their Members in their Community

9. RDLA’s In-District Lobby Days • RDLA will help you schedule meetings & prepare for your In-District Meetings! • Register Here: http://www.congressplus.com/events/index.cfm?action=Registration_Form&eventcode=ZpDAaE&bypass=true • Important Dates: • July 8th Webinar: Building Relationships with Members of Congress • July 11th Deadline to Register to Participate • July 22nd Webinar: How to Have a Successful Meeting • August 4th – September 5th Congressional Recess In-District Meetings

10. RDLA’s In-District Lobby Days • July 8th:  Building Relationships with Members of Congress • How a Fly Out works & Scheduling meetings– Advocacy Associates • Getting to know your Member of Congress • How to attend a town Hall/Campaign Event • Engaging your Member of Congress on Social media • July 22nd: How to Have a Successful Meeting • Materials for you meeting • Talking Points – how to tell your personal story in 2 minutes • Issue Training • Rare Disease Congressional Caucus • America Heals Act • Following up for Success

11. 2014 RareVoice Awards Gala • 3rd Annual Celebration to Honor Advocates who • Give Rare Disease Patients A Voice on Capitol Hill Thursday, November 13, 2014 6:00 pm Champagne Cocktail Reception 8:00 pm Awards Ceremony 9:00 pm After-Party Celebration Arena Stage, Washington DC This is a widely attended public event There is not cost to attend www.RareVoiceAwards.org

12. The “Abbey” • The “Abbey” represents the "rare voice" speaking on behalf of patients, especially the children, who might not otherwise be heard • The award, with her blessing, was named for Abbey Meyers, founder of NORD • Mrs. Meyers received the lifetime achievement award at the inaugural RareVoiceAwards Gala for her vital role in the passage of the Orphan Drug Act The statue was commissioned for the RareVoice Awards Gala from the renowned sculptor Nobe who specializes in bronzes. Nobe's brilliant technique and original approach endow his figures with a sense of mystery and wonder that permeates the physical essence of the sculptures and brings each piece to life.  

13. Abbey Nominations Open for the 2014 RareVoice Awards Gala  • We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  Deadline to submit nominations is Friday August 29, 2014 • If you know of someone who has been a “Voice” for the rare disease community and should be honored for their work, please complete the application at the link below for one of the three categories: http://rarevoiceawards.org/program/nominate/ • All nominees will be honored at the Gala • Abbey recipients will be announced live at the Gala

14. Abbey Nominations Categories: 1.   Congressional Staff We honor both Members of Congress and Congressional Staff as we recognize that Congressional Staff are some of rare disease patients’ best advocates.  While staff usually work tirelessly behind the scenes, this Gala pays tribute to their vital role in the legislative process.  The Congressional Staffer who wins the award also wins an award for their Member of Congress. 2.  Patient Advocate or Patient Organization Patient organizations and individual advocates have many roles and responsibilities in advocating for rare disease patient.  This award is meant to recognize those who go above and beyond to become policy leaders and political advocates. 3.  Government Agency Leaders Staff from the National Institutes of Health (NIH), Food and Drug Administration (FDA), Social Security Administration (SSA) and other Government Agencies play a large role in influencing and implementing rare disease policy.  This new award is meant to highlight their important work and thank them for being a rare disease advocate.

15. Save the Date! • RDLA’s In–District Lobby Days • July 8th Webinar: Building Relationships with Members of Congress • July 22nd Webinar: How to Have a Successful Meeting • August Recess In-District Meetings August 4th – September 5th • September 17th Rare Disease Congressional Caucus Briefing on FDASIA Implementation • Sept. 18th RDLA in Person Meeting • If there are agenda items that you would like to see covered infuture RDLA meetings, please email: arussell@everylifefoundation.org • (No RDLA meetings/Calls for July & August due to Aug Recess)