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Presentation on theme: "C OMMUNITY C ONGRESS I NTRODUCTORY W EBINAR Tuesday, January 27, 2015 1."— Presentation transcript:


2 Agenda I. I.Introductions a. Foundation Team II. II.Foundation Overview a.Policy Initiatives b.Patient Support Programs III. III.Community Congress Overview IV. IV.Questions & Discussion 2

3 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 3

4 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 4

5 Consulting & Lobbying Support Lobbyists for the OPEN ACT Lobbyists for the OPEN ACT – Harry Sporidis, Polsinelli PC – Tim Perrin, Polsinelli PC Consulting for RDLA & Coordinating the Rare Disease Congressional Caucus Consulting for RDLA & Coordinating the Rare Disease Congressional Caucus – Jen Bernstein, Horizon Government Affairs 5

6 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 6

7 Foundation Initiatives Science Based Policy Goals: Science Based Policy Goals: – CureTheProcess Campaign – 2 New Legislation – OPEN ACT – Annual Scientific Workshop – Current & Planned Publications Patient & Community Support Programs Patient & Community Support Programs – Rare Disease Legislative Advocates – RareGiving – RareArtist – SIMD/NAMA 7

8 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. 8

9 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. 9

10 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 10

11 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 21 st Century Cures 11

12 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 12

13 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) 13

14 14 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 organizations to use when working in DC Rare Disease Congressional Score Card Clearinghouse of all legislation that affects rare disease patients

15 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. 15

16 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 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 16

17 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. 17

18 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 to have policy impact 18

19 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. 19 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.

20 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 20

21 Membership Levels $20,000 Industry Leader (3 participants) $10,000 Mid-Cap Bio (2 participants) $ 5,000 Emerging Bio (1 participant) $ 2,500Start-up Bio (1 participant) $ 1,000Healthcare/Policy Consultant, CRO, Trade Org, (1 participant) $ 500 Academic/Medical Organizations (1) FREEPatient Org/Government Agency (1) 21

22 How will the Congress work? 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 22

23 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

24 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 2 nd & August 4 th In-person full Community Congress meeting (Washington DC): November 4 th 24

25 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 25

26 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 26

27 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 27

28 What’s an Achievable/ Tangible Goal? Educational Patient/Stakeholder Event Educational Patient/Stakeholder Event White paper or editorial publication White paper or editorial publication Congressional Briefing Congressional Briefing Draft Regulatory Guidance Draft Regulatory Guidance Sign-on Letter for Policy Change Sign-on Letter for Policy Change Policy Summit Policy Summit A National or Strategic Plan A National or Strategic Plan Propose or improve legislative language Propose or improve legislative language Influence the implementation of legislation Influence the implementation of legislation 28

29 Sign-up form 29

30 Confirmed Members 30 Public PolicyFDA Regulatory Science Newborn Screening NGLY1.orgPPMDShire Dercums Foundation Sarcoidosis of Long Island Inc. Hunter Syndrome Foundation Stiff Person Syndrome Action Network RASopathies Network USA Adrenal Insufficiency United Sarcoma Foundation ShireNational MPS Society Parent Project MD Vasculitis Patient- Powered Research Network

31 Q&A/Discussion Help drive the Community Congress Agenda, complete the sign-up formsign-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: 31

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