COMMUNITY CONGRESS
INTRODUCTORY WEBINAR
Tuesday, January 27, 2015
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Agenda
I. Introductions
a. Foundation Team
II. Foundation Overview
a. Policy Initiatives
b. Patient Support Programs
III. Community Congress Overview
IV. Questions & Discussion
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Foundation Staff
• Julia Jenkins, Executive Director
• Max Bronstein, Senior Director, Public & Government
Relations
• Sue Colton, Director of Operations and Development
• Andy Russell, Associate Director of Advocacy &
Government Relations
• Grant Kerber, Associate Director of Public Policy &
Communications
• Deborah Walter, Manager of Finance & HR
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Board Members
• President & Founder Emil D. Kakkis, MD, PhD, President/CEO,
Ultragenyx
• Secretary Julia Jenkins, Executive Director, EveryLife
Foundation
• Treasurer John Klock, MD, CEO & Managing Director, QT
Ultrasound, LLC
• Ritu Baral, Managing Director/Senior Biotechnology Analyst,
Cowen & Company
• Mark Dant, Executive Director, Ryan Foundation for MPS
• David Rowitch, MD, PhD, Chief of Neonatology, UCSF
• Jenny Soriano, MD, OB/GYN, Kaiser Permanente
• Matt Wilsey, NGLY1 Parent Advocate
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Consulting & Lobbying Support
• Lobbyists for the OPEN ACT
– Harry Sporidis, Polsinelli PC
– Tim Perrin, Polsinelli PC
• Consulting for RDLA & Coordinating the
Rare Disease Congressional Caucus
– Jen Bernstein, Horizon Government
Affairs
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About the Foundation:
• Dedicated to Accelerating Biotechnology
Innovation for Rare Disease Treatments
• Advocating practical and scientifically sound
change in policy and law to increase the
predictability of the regulatory process through
scientific analysis and dialogue, grassroots
support & expert-led workshops.
• Foundation Mission:
– No disease is too rare to deserve treatment
– All treatments should be safe & effective
– We could be doing more with the science we
already have
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Foundation Initiatives
• Science Based Policy Goals:
– CureTheProcess Campaign – 2
• New Legislation – OPEN ACT
– Annual Scientific Workshop
– Current & Planned Publications
• Patient & Community Support Programs
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Rare Disease Legislative Advocates
RareGiving
RareArtist
SIMD/NAMA
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• CTP-2 is a grassroots, patient driven advocacy
campaign to remove the roadblocks in the drug
development process.
• The campaign educations patients advocates,
Congressional allies and the public about the policy
changes needed to the spur the development &
increase the availability of lifesavings treatments.
• Together we can bring needed treatments to the
nearly 30 million Americans affected by rare
diseases.
www.CureTheProcess.org
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Campaign Goals
• Rationalize: Encourage the FDA to accommodate a
more scientifically rational and flexible application of
safety data to allow US patients to have access to early
stage clinical trials.
• Incentivize: Enact legislation encouraging industry to
repurpose major market drugs for rare disease patents
or “rare-purpose” drugs to ensure patients have access
to safe, effective and affordable treatments.
• Specialize: Encourage the FDA to create more
specialized drug review divisions and allow reviewers
access to the latest science to facilitate a better
understanding of the diseases they are reviewing.
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Orphan Product Extensions Now
Accelerating Cures & Treatments (OPEN ACT)
Incentivize the Investment in Rare Disease Drug Development
• Sponsor receives FDA approval for their major market
drug or has a current approved drug still under patent
protection
• Sponsor could seek rare disease indications to extend
its patent life & protect revenue from market
competition for 6 months
• Must be a Rare Disease – under 200,000 patients in
the US
• Must obtain data to place the new rare disease
indication on the label
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Support for OPEN Growing
• Potential for hundreds of safe, effective
affordable treatments available for rare disease
patients
• Introduced last Congress by 4 bipartisan original
co-sponsors
– Leads: Energy & Commerce Committee Members
Reps Bilirakis (R-FL) & Butterfield (D-NC)
• 95 patient organizations supporting
• To be reintroduced next month
• Reps DeGette & Upton are interested
– May be included as a piece of 21st Century Cures
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Annual Scientific Workshop
• 2014: Rationalizing Safety Testing to Enable Clinical
Studies and Approval in the US for Rare Disease
Treatments
• Save the Date: Incorporating the Patient
Perspective in the Selection of Endpoints and
Clinical Trial Design & Analysis
– September 15, 2015, Washington DC
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Upcoming Publications
• Recommendations for the Development of Rare
Disease Drugs using the Accelerated Approval
Pathway and for Qualifying Biomarkers as
Primary Endpoints, being published in Orphanet
Journal of Rare Diseases
• Endpoints in Orphan and Accelerated Approval
Designations, an editorial planned submission to
Nature Biotechnology in February
• Editorial on IND-Enabling Safety and Toxicology
Issues (Spring)
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Rare Disease
Legislative Advocates
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Legislative and grassroots strategies
Online advocacy tools to contact Congress
Coalition building and support for legislation
Monthly Conference calls/meetings to learn
about legislation & Action Alerts
• DC Office with workstation & meeting space for
organizations to use when working in DC
• Rare Disease Congressional Score Card
• RareAdvocates.org Clearinghouse of all
legislation that affects rare disease patients
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RareGiving
• RareGiving is an expansion of the Foundation’s
Text to Donate program
• Provides funding to support individual rare
disease patients and patient organizations
• Provides travel scholarships for patient
advocates to attend Capitol Hill and FDA
advocacy events.
• Foundation awards grants for patient and
physician education events and academic
conferences that facilitate collaboration across
diseases.
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• Exhibits the unique gifts of individuals affected by
rare diseases and promotes the expression of their
stories through art.
• Artists are encouraged to submit their work in our
annual EveryLife Art Contest
• RareArtist.org is a permanent space where “rare
artists” are celebrated and the public can view
their work
• 2015 Rare Artist Reception
– During Rare Disease Week on Capitol Hill
– Hosted by Rare Disease Caucus & Art Caucus Co-Chair
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Representative Leonard Lance
• The North American Metabolic Academy (NAMA) is
an educational program sponsored by the Society
for Inborn Metabolic Disorders (SIMD)
• Created to address the urgent need for specialized
clinical training in the treatment of inborn errors of
metabolism.
• NAMA encourages trainees to pursue a career as
biochemical geneticists.
• The Foundation helps secure grants to ensure this
essential program has funding & works to promote
the program so it continues to grow.
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• To educate Rare Disease Community Stakeholders about
the scientific and policy goals of the Foundation
• Create a formal group and process to receive input from
Industry, patient organizations and other stakeholders to
ensure the Foundation advocacy efforts are addressing
the most pressing needs of the rare disease community
• Help prioritize the Foundation’s plans for the coming year
• Create working groups to address urgent policy issues
• Set tangible and achievable policy goals, devise strategies
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to have policy impact
RareCongress.org
• The Community Congress is a membership-based
program of the EveryLife Foundation dedicated to
bringing patient organizations, industry leaders,
and other rare disease stakeholders together.
Who can be a member of the
Community Congress?
• Pharmaceutical companies, patient organizations,
and other stakeholder organizations are welcome
to become members of the Community Congress.
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Why did the Foundation
create the Community Congress?
• To ensure the EveryLife Foundation is meeting
the urgent scientific and policy needs of the rare
disease community.
• To create a permanent forum that brings all
community stakeholders together to create
pertinent connections and partnerships.
• To educate the Rare Disease Community about
the work of the Foundation
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Membership Levels
$20,000
$10,000
$ 5,000
$ 2,500
$ 1,000
$ 500
FREE
Industry Leader (3 participants)
Mid-Cap Bio (2 participants)
Emerging Bio (1 participant)
Start-up Bio (1 participant)
Healthcare/Policy Consultant, CRO,
Trade Org, (1 participant)
Academic/Medical Organizations (1)
Patient Org/Government Agency (1)
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How will the Congress work?
• Each Fall the Foundation will host the
Community Congress annual meeting, held the
day of the RareVoice Awards Gala in Washington
DC
– Members will learn about the Foundation’s
scientific and policy goals, and help provide
insight on prioritizing future initiatives.
• Members can help drive the policy changes in
working groups or just participate as supporting
members
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Working Groups
• Issue-based working groups will meet via
conference call/webinar 2 to 3 times per year &
once in person at the annual meeting to provide an
opportunity for continued engagement on specific
issues to drive policies forward. Foundation staff
will provide support to working groups.
• Each working group will have two co-chairs, one
from a patient group and another from industry
• Co-chairs will be nominated by working group
participants
• Members will vote on what issue is chosen & how
best to make a policy impact
Preliminary Timeline, 2015
• Informational Webinar: January 27
• First working group calls/webinars commence:
Week of March 23 - Working group formation
and issue selection
• Ongoing engagement among working group
members
• Additional calls/webinars: June 2nd & August 4th
• In-person full Community Congress meeting
(Washington DC): November 4th
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Public Policy Working Group
Prioritize one policy issue to focus on for
2015 – Pick Something Achievable that will
have an impact
Topics to consider:
• Expanded/Experimental Access
• Reimbursement
• Access & Affordability
• Incentivizing drug development
• Medical foods
• New Funding models for NIH & FDA
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Regulatory/Scientific Policy
Working Group
Prioritize One Goal for the Year – Event or
Publication – something achievable
• Use natural history to reduce placebo control groups
• Inborn Errors of Metabolism patient focused meeting
• Regulatory training for patient advocates similar to Eurordis
Summer School program
• Analysis of 6-Minute Walk Test and Other Endpoints
• Incorporating the Multi-domain Responder Index Analysis
Technique into Rare Disease Analysis Strategy
• The Effective Use of Responder Analyses and Minimally Important
Differences in Rare Disease Studies
• Event: Regulatory training for patient advocates
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NBS Working Group
• Advocating for NBS State by State, Disease by Disease is an
unacceptable burden to place on patient organizations &
individual advocates
• We need to bring stakeholders together to create a
comprehensive state & federal strategy to increase newborn
screening for rare diseases
• The EveryLife Foundation is the only cross
disease organization that advocates for screening of
diseases not recommended by the Secretary’s Advisory
Committee
• We believe some diseases will never be treated unless we
can identify patients first through NBS
• Event: National Summit for all NBS stakeholders
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What’s an Achievable/
Tangible Goal?
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Educational Patient/Stakeholder Event
White paper or editorial publication
Congressional Briefing
Draft Regulatory Guidance
Sign-on Letter for Policy Change
Policy Summit
A National or Strategic Plan
Propose or improve legislative language
Influence the implementation of legislation
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Sign-up form
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Confirmed Members
Public Policy
FDA Regulatory
Science
Newborn
Screening
NGLY1.org
PPMD
Shire
Dercums
Foundation
Sarcoidosis of Long
Island Inc.
Hunter Syndrome
Foundation
Stiff Person
Syndrome Action
Network
RASopathies
Network USA
Adrenal
Insufficiency United
Sarcoma
Foundation
Shire
National MPS
Society
Parent Project MD
Vasculitis PatientPowered Research
Network
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Q&A/Discussion
• Help drive the Community Congress Agenda,
complete the sign-up form
• Ensure the Foundation is meeting the urgent
scientific, regulatory, & policy needs of the
community.
• We want to hear from you!
• Additional questions? Email Max Bronstein:
mbronstein@everylifefoundation.org
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