Transcript April 23rd, 2014

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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
 Andy
Taylor, Legislative Director
Office of Congressman Michael McCaul
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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FDA issued Draft Guidance on Expedited Approval in June 2013:
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Fast Track
Accelerated Approval (ULTRA/FAST)
Priority Review Vouchers (Creating Hope Act)
Breakthrough Therapy Designation
Did not address issues specific to rare diseases
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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/GuidanceComplianceRe
gulatoryInformation/Guidances/UCM358301.pdf
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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
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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
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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
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RDLA will help you schedule meetings & prepare for your
In-District Meetings!
Register Here:
http://www.congressplus.com/events/index.cfm?action=Re
gistration_Form&eventcode=ZpDAaE&bypass=true
Important Dates:
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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
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July 8th: Building Relationships with Members of Congress
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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
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
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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
RareVoice Awards 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.
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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
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.
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RDLA’s In–District Lobby Days
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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
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If there are agenda items that you would like to see covered in future
RDLA meetings, please email: [email protected]
(No RDLA meetings/Calls for July & August due to Aug Recess)