C OMMUNITY C ONGRESS M EETING November 13, 2014 Washington, DC 1 - - PowerPoint PPT Presentation

INAUGURAL “COMMUNITY CONGRESS” MEETING

November 13, 2014 Washington, DC

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Agenda

I. Introductions II. 15 Year Delay in Treatment Development & Approval Steve Smith, MPS IV Parent Advocate

• III. Challenges in Treatment Development for Ultra-

Rare Patients Matt Might, NGLY1 Parent Advocate

• IV. Mission & Scope of the EveryLife Foundation

Emil Kakkis, EveryLife Foundation President V. EveryLife’s Community Congress Launch Julia Jenkins, Foundation, Executive Director

• VI. Questions & Discussion from Attendees

(45 minutes)

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Foundation Staff

• Julia Jenkins, Executive Director
• Sue Colton, Director of Operations and Development
• Sara Kowalczyk, Associate Director of Regulatory Affairs

& Medical Writing

• Andy Russell, Associate Director of Advocacy &

Government Relations

• Grant Kerber, Associate Director of Public Policy &

Communications

• Max Bronstein, Advocacy & Government Relations

Consultant

• 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

– 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

• r “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 qualify for Fast Track Designation: serious or

life-threatening disease

• Must obtain data to place the new rare disease

indication on the label

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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, currently under review by Orphanet Journal of Rare Diseases

• Endpoints in Orphan and Accelerated Approval

Designations, an editorial planned submission to Nature Biotechnology

• Editorial on IND-Enabling Safety and Toxicology

Issues (February)

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Rare Disease Legislative Advocates

• 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
• rganizations 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 Representative Leonard Lance

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• 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

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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?

• Of the many reasons for forming the Community

Congress, one particularly driving force to ensure that the EveryLife Foundation is meeting the urgent scientific and policy needs of the rare disease community.

• Additionally, there is no other permanent forum

that brings all community stakeholders together to create pertinent connections and partnerships.

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Membership Levels

$20,000 Industry Leader (3 participants) $10,000 Mid-Cap Bio (2 participants) $ 5,000 Emerging Bio (1 participant) $ 2,500 Start-up Bio (1 participant) $ 1,000 Healthcare/Policy Consultant, CRO, Trade Org, (1 participant) $ 500 Academic/Medical Organizations (1) $ 0 Patient Org/Government Agency (1)

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How will the Congress work?

• Each Fall at the Community Congress annual

meeting, which will be 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 valuable insight on prioritizing future initiatives.

• Issue-based working groups will meet via

conference call/webinar 2 to 3 times per year &

• nce in person at the annual meeting to provide

an opportunity for continued engagement on specific issues to drive policies forward.

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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
• Other?

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Opportunities: 21st Century Cures Initiative

• Implementation of the Cure The Process 2 goals

has already begun with the 21st Century Cures Initiative

• Submitted White Paper comments on first series of

21st Century Cures Roundtables

• PDF of comments available on EveryLife Foundation Website
• Working closely with Energy & Commerce Committee

Leadership

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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

• Other?

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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 across

disease organization that advocates for screening of diseases not recommended by the Secretary Advisory Committee

• We believe some diseases will never be treated

unless we can identify patients first through NBS

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Q&A/Discussion

• Help drive the Community Congress Agenda
• Ensure the Foundation is meeting the urgent

scientific, regulatory, & policy needs of the community.

• We want to hear from you!

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