ANNUAL REPORT
everylifefoundation.org
RESPONDING TO A CRISIS
ANNUAL REPORT
In 2020, COVID-19 brought the world to a halt, but the pandemic has not stopped the advocacy efforts of the rare disease community. It has not even slowed us down. Our community has always had to do things differently, to pivot and reimagine – and this year has been no exception. Before the pandemic struck, more than 900 rare disease advocates traveled from across the country to meet with their Members of Congress during Rare Disease Week on Capitol Hill. Despite its challenges, COVID-19 inspired us to transform our programs and events, enabling us to engage new advocates who would not otherwise have been able to participate. Members of Congress and Congressional staff joined us virtually for Rare Across America and Rare Disease Congressional Caucus Briefings, bringing advocacy to a whole new level. As we expanded our policy scope to address the urgent needs that COVID-19 created for our community, social unrest also highlighted the needs of our underserved minority rare disease patients. The Foundation’s Board of Directors made the decision to expand the Foundation’s mission to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
Table of Contents Responding to a Crisis
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EveryLife Foundation Team
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Changing Policy, Saving Lives
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Greater than Covid-19
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Advocates in Action
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Lifting Every Voice
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Every Voice Matters
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Engaging New Patient Communities
11- 12
2020 Income and Expenses
13- 14
EveryLife Foundation Board of Directors
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Working to fulfill our expanded mission, we were incredibly grateful to be able to increase our outreach and support of the community by: Establishing a Diversity Inclusion Advocacy Fellowship to reach underserved rare disease communities; Launching a State Advocacy Initiative to better support state and regional advocacy and state-based rare disease organizations; Doubling the number of Community Congress patient organization members who, alongside industry partners, are driving the conversation on urgent policy issues and Foundation programs and initiatives; Providing nearly $600,000 – double the amount provided in 2019 - in grants, travel stipends and educational scholarships through our Rare Giving and RAREis Scholarship Fund programs. Without you – the advocates, patient organizations, industry partners, and Congressional champions – none of our work would be possible. Thank you for continuing to advocate with us for diagnostics, treatments and cures. Sincerely,
Julia Jenkins Executive Director, The EveryLife Foundation for Rare Diseases
Mark Dant Chairman of the Board of Directors, The EveryLife Foundation for Rare Diseases
everylifefoundation.org @EveryLifeOrg 1
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EVERYLIFE FOUNDATION FOR RARE DISEASES TEAM Operations Julia Jenkins Executive Director jjenkins@everylifefoundation.org Mary Morlino Patient Engagement Consultant mmorlino@everylifefoundation.org Megan Pinegar Senior Director of Operations and Human Resources mpinegar@everylifefoundation.org Alyssa Terwall Associate Director of Special Events aterwall@everylifefoundation.org
Development Ted Brasfield Senior Director of Development tbrasfield@everylifefoundation.org
Close the innovation gap
Policy, Advocacy, and Patient Engagement Annie Kennedy Chief of Policy and Advocacy akennedy@everylifefoundation.org
Liesl Broadbridge Policy Fellow lbroadbridge@everylifefoundation.org
Katelyn Laws RDLA Program Coordinator klaws@everylifefoundation.org
Lindsey Cundiff Associate Director of Patient Engagement lcundiff@everylifefoundation.org
Jack Meloro Community Congress Program Coordinator jmeloro@everylifefoundation.org
Swapna Kakani RDLA State Advocacy Fellow skakani@everylifefoundation.org
Adrian Palau-Tejeda Diversity Inclusion Advocacy Fellow Apalau-tejeda@everylifefoundation.org
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
The EveryLife Foundation is working to:
Elissa Taylor Associate Director of Alliance Development etaylor@everylifefoundation.org
Kylie Barber Medical Foods Policy Associate kbarber@everylifefoundation.org
CHANGING POLICY, SAVING LIVES
Eliminate the diagnostic odyssey Dylan Simon Newborn Screening and Diagnostics Policy Manager dsimon@everylifefoundation.org Jamie Sullivan Director of Public Policy jsullivan@everylifefoundation.org Shannon von Felden Director of Rare Disease Legislative Advocates svonfelden@everylifefoundation.org
Improve the regulatory process
More than thirty million Americans are living with one or more rare diseases.
The economic burden of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019.
Rare disease patients wait an average of 6.3 years after symptoms present before receiving a confirmed diagnosis.
Ensure patient access to therapies and cures Empower the patient voice
93% - 95% of the more than 7,000 known rare diseases have no U.S. Food and Drug Administrationapproved therapies.
A disease is defined as rare when it affects fewer than 200,000 people in the United States.
Communications & Marketing Brenda Colmenares Communications Manager bcolmenares@everylifefoundation.org
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Britta Dornan Senior Director of Communications and Marketing bdornan@everylifefoundation.org
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GREATER THAN COVID-19
Community Engagement Created a new Ad-Hoc COVID-19 Community Congress Working Group to ensure the needs and experiences of the rare disease community were central in policy discussions related to clinical trials, healthcare access, newborn screening, and diagnostics.
TELEHEALTH IS
IMPORTANT
Information Hub
Telehealth is important. It needs coverage. I should never have to go back in to the doctor for something silly that they can see my kid on the computer screen for; however, it shouldn't be the only option. If you are living with a rare or chronic condition you should have the opportunity to go in to the hospital or physician if you need to. Jenn McNary Rare Disease Advocate, Community Congress COVID-19 Working Group Co-Chair
Home Health and Telehealth Policies In response to the Center’s for Medicare and Medicaid Services’ (CMS) interim final rule, the Foundation asked CMS to ensure rare disease patients can continue to access in-home health care, including injection and infusion services. Joined 340 organizations in signing a letter urging Congressional leaders to make telehealth flexibilities created during the COVID-19 pandemic permanent.
Vaccine Prioritization and Perception
Launched an online COVID-19 Action Center in March to provide resources and information to the community.
Took action to understand the community’s needs and concerns about COVID-19 vaccines and advocate for our high-risk population to have access to safe, accessible and timely vaccines. Conducted the U.S. Rare Disease COVID-19 Vaccine Survey, generating 1,399 responses. Conveyed these survey results in comments to the National Academy of Sciences’ taskforce charged with creating the Preliminary Framework for Equitable Allocation of COVID-19 Vaccine and in a letter to federal COVID-19 leadership and the National Governor’s Association.
Access Barriers Led a sign-on letter that was joined by nearly 200 patient advocacy organizations and sent to leadership in all 50 states aimed at highlighting the unique access barriers created by COVID-19 and temporary policy fixes that could serve to address them.
Scientific Workshop
Rare Disease Congressional Caucus Briefing
Convened more than 160 leaders from the FDA, NIH, CMS, CDC, industry and patient organizations to highlight case studies of COVID-19 innovations, discuss best practices and how to apply the lessons learned to rare disease innovation, and identify unmet needs as the pandemic evolved.
Hosted a briefing in July for nearly 200 Members of Congress, Congressional staff, and advocates entitled ‘Rare Disease Community Experiences with COVID-19 Response Efforts and Future Policy Opportunities’. The briefing featured Jenn McNary, a rare disease mom and advocate who highlighted the extreme burden the pandemic has placed on rare disease families.
Meeting Evolving Needs Joined with other patient organizations and Community Congress members to highlight the unique needs of the rare disease community as states began to reopen and again as the winter surge took hold.
Empowering Advocates Empowered 500 advocates in 48 states during Rare Across America meetings with advocacy tools to advocate to extend home health and telehealth policies beyond the public health emergency.
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This has been a terrible moment for our country and the world, let's make no mistake about it, but this is also a singular moment for the rare disease community and we can't let it go to waste. Christopher P. Austin Former Director National Center for Advancing Translational Sciences National Institues of Health
SINGULAR MOMENT
FOR THE
COMMUNITY 6
LIFTING EVERY VOICE
ADVOCATES IN ACTION Rare Disease Legislative Advocates (RDLA) is a program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations. RDLA believes that every voice matters and that patients are the key to changing public policy. 150+ Advocates Engaged in RDLA webinars each month
DIRECT COMMUNITY SUPPORT $590,000 The Foundation's Rare Giving program supports individual rare disease patients as well as organizations that engage patients, caregivers and others in the community in public policy.
182 Rare Disease Congressional Caucus Members 25 Senators and 157 Representatives
8 Diverse Members Established the RDLA Advisory Committee
$286,290 in Grants and Awards Provided to 50 + rare disease organizations
The National Economic Burden of Rare Disease Study The Foundation commissioned the Lewin Group to estimate the economic impact of rare disease in the U.S. in 2019 and assembled a distinguished steering committee of technical advisors and expert contributors representing government, industry, academia, and rare disease communities. The study included one of the largest surveys thus far of multiple rare disease communities, generating 1,399 fully completed responses from individuals representing about 400 rare disease communities.
$118,350 in Travel Stipends Provided to 130 advocates
$185,000 in Scholarships
Emily T. Solve M.E/CFS Organization
Science-driven disease organization working to accelerate the discovery of safe and effective treatments for myalgic encephalomyelitis and chronic fatigue syndrome.
“With this additional support we were able to provide phone and web based meeting tools, spanish translation of materials, an online action center, closed captioning for all events, and many more accommodations to improve accessibility for those disabled by chronic illness.”
Veronica T. RAREis Scholarship Recipient “I am incredibly optimistic that I discovered my heart tumor, so that I can do some wonderful things in my career as a nurse, and your scholarship will help me get there!”
Jessica G. RAREis Scholarship Recipient “I am in the midst of a Public Policy and Administration Masters and the #RAREis Scholarship Fund will go a long way towards helping me financially.”
Provided to 37 rare disease patients, 23 states represented
Newborn Screening Bootcamp The Foundation and Expecting Health at Genetic Alliance hosted a virtual five-part training series in September for 361 newborn screening stakeholders.
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Thanks to the support of Horizon Therapeutics, the EveryLife Foundation established the #RAREis Scholarship Fund – to enrich the lives of adults living with rare diseases by providing support for their educational pursuits.
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EVERY VOICE MATTERS We do not speak for patients. We provide the training, education, resources and opportunities to make their voices heard. By activating the patient advocate, we can change public policy and save lives.
The Foundation’s Rare Disease Legislative Advocates (RDLA) program convened a record-breaking number of advocates during Rare Disease Week on Capitol Hill and during August recess for meetings held virtually in Members' district offices.
900 Advocates
Participated in Rare Disease Week on Capitol Hill
227 Patient Organizations Represented at Rare Disease Week on Capitol Hill
540 Advocates Participated in Rare Across America
393 Meetings Confirmed with Members of Congress or their staff
Letter to the FDA 14 Members of Congress sent a letter (led by the Rare Disease Congressional Caucus Co-Chairs and in response to advocate outreach) to the FDA in support of an FDA Rare Disease Center of Excellence
286 Meetings Confirmed with Members of Congress or their staff
“There is purpose and passion in our advocacy! We had an amazing, unforgettable experience representing our community and joining more than 900 advocates for Rare Disease Week on Capitol Hill.”
Kids Conquering SCD Advocate Orlando, Florida
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48 States Represented in Rare Across America
“Participating in Rare Across America has been an integral part of our rare journey. Having the opportunity to share our story with legislators has heightened our efforts of advocacy and awareness. Our stories may not be the same, but our united voices empower the rare disease community."
Sarita E. (pictured above) Huntsville, Alabama
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ENGAGING NEW PATIENT COMMUNITIES The Foundation’s Community Congress program is a strategic advisory council that brings patient organizations and industry together to provide advice and insight on urgent policy issues. In 2020, the program doubled its patient organization membership, expanded its focus into access policy, provided critical community leadership in response to the COVID-19 crisis, and continued to develop policy solutions that will impact the rare disease community for generations to come.
Total Member Organizations: 277 Public Policy
TIP SHEET
Diversity and Inclusion Initiative The Foundation established a Diversity Inclusion Advocacy Fellowship to ensure that minority and underserved rare disease patients have an opportunity to make their voices heard by policy makers.
Developing a diverse staff and board that includes underserved communities is imperative and necessary for improving diversity and inclusion. With 79% percent of new board members for S&P 500 companies coming from a diverse background, the need for diversity in leadership and outreach is being realized now more than ever. Reasons include:
Diverse individuals understand cultural and linguistic barriers, enabling better outreach to underserved communities
With a limited patient population for rare diseases, and reduced recruiting for clinical trials among diverse groups, the need for diversity advocacy is imperative. Oncology
Black or African American 3% (1,415 participants)
Some Other Race 97% (7,480 participants)
Some Other Race 97% (55,118 participants)
source: Multi-Region Clinical Trials Center Advancing Ethnical Research 2018 National Conference
Webinar sign-ups Signature or yearly outreach events Events involving outside organizations and work groups Benchmarking and Targeting
Importance, Keeping Track, and Tools To ensure that diversity goals are achieved, it’s important to keep measuring the race and ethnicity of program participants after initial sampling. Tools organizations can use to improve diversity include: Offering multi-lingual language options for event and project materials
The Rare Disease Diversity Coalition The Foundation serves on the streering committefor the Rare Disease Diversity Coalition. Launched by the Black Women's Health Imperative, the Coalition addresses the extraordinary challenges faced by the rare disease patients of color.
NBS and Diagnostics
Membership: 92 Industry 25% / Patient Organizations 74%
Focused on ensuring patients receive earliest access to lifesaving diagnostic opportunities through newborn screening.
Press outreach to minority and non-English language newspapers Engaging LBGTQ, Minority Health, Ethnically Biased Disease Organizations in increasing engagement with underserved communities U.S. National Demographic Estimates (2019)*
Following data collection, organizations can compare how diverse their programs are to national demographic estimates for minority and underserved communities (i.e. US census data).
American Indian and Alaska Native 1.3%
Asian 5.9%
Access
Native Hawaiian and Other Pacific Islander 0.2% Two or More Races 2.8%
Strategies can then be developed that incorporate tools to overcome possible cultural, linguistic, and corporate barriers Benchmarks should be short-term improvement plans with long-term goals and yearly benchmarks that are attainable for underserved participation
Membership: 88 Industry 42% / Patient Organizations 56%
Focused on improving the regulatory process and advancing regulatory science for rare disease therapies.
Cardiovascular Disease Black or African American 3% (211 participants)
Surveying the Field Before embarking on any diversity initiatives, it’s important to understand how well your organization is performing with underserved communities. In order to build diversity goals and benchmarks, it’s necessary to collect ethnicity, racial, and gender data from all programs. Ethnicity responses in registrations should take cues from census groups, with room for additional ethnicity options as needed. Find links and advocacy tools for data collection at https://rareadvocates.org/ advocacy-tools. Information gathering opportunities include:
Regulatory
Participation of Black/African Americans in Clinical Trials for Oncology and Cardiology
Diversity in background in the boardroom creates diversity in ideas and in resolving challenges
Membership: 140 Industry 39% / Patient Organizations 60%
Focused on closing the innovation gap for the 93% - 95% of rare diseases that have no FDA-approved treatment and ensuring patients receive early access to treatment opportunities.
Diversity and Inclusion
Diversity at Home
Current Working Groups include:
Membership: 106 Industry 45% / Patient Organizations 49%
Focused on accelerating patients’ access to approved therapies.
Hispanic or Latinx 18.5% White (not Hispanic or Latinx) 60.1%
Black or African American 13.4%
* Total may exceed 100% because Hispanic or Lantinx may include multiple races.
Rare Disease Legislative Advocates (RDLA) is a program of the EveryLife Foundation for Rare Diseases to support the advocacy efforts of all rare disease patients and organization. RDLA is committed to growing diverse patient advocacy communities and working collaboratively. For additional assistance with Diversity and Inclusion advocacy, contact Shannon von Felden, RDLA Program Director, at svonfelden@everylifefoundation.org.
(202) 697-7273 (RARE)
RAREADVOCATES.ORG
Ad-Hoc COVID-19
@RAREADVOCATES
Membership: 85 Industry 42% / Patient Organizations 55%
Focused on ensuring that the needs and experiences of the rare disease community are heard in relation to healthcare decisions made during the current pandemic.
State Advocacy Initiative The Foundation established a State Advocacy Fellowship and launched the RDLA State Advocacy Hub to better support state and regional rare disease organizations and advocates.
100 Young Adults Paticipated in the new YARR Leadership Summer Series Webinars 11
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2020 INCOME AND EXPENSES
EVERYLIFE FOUNDATION BOARD OF DIRECTORS
The Foundation receives funding from philanthropic organizations, government, individual donors, and corporations to support its activities. Sponsors are offered recognition for their support, but do not influence Foundation policy priorities or event program content. To view our complete funding policy, visit: everylifefoundation.org/fundingpolicy
The Foundation Board of Directors is comprised of individuals with decades of experience in the government, nonprofit, finance, science, medicine, industry, and academic sectors. Several board members are the parents of children with a rare disease, enabling them to offer firsthand knowledge of the challenges facing the rare disease community.
Mark Dant, Chair Executive Director, Ryan Foundation
Frank Sasinowski, MS, MPH, JD, Vice Chair Director, Hyman, Phelps & McNamara P.C.
Julia Jenkins, Secretary Executive Director, EveryLife Foundation
Vicki Seyfert-Margolis, PhD, Treasurer Founder and CEO, MyOwnMed
Ritu Baral, Member Managing Director Senior Biotechnology Analyst, Cowen and Company
THANK YOU TO OUR 2020 SUPPORTERS $200,000 and Over Chan Zuckerberg Initiative Emil D Kakkis Horizon Therapeutics Sanofi Genzyme
$100,000 - $199,000 Alexion Pharmaceuticals Genentech Mallinckrodt Pharmaceuticals Sarepta Therapeutics Travere Therapeutics Takeda Pharmaceutical Vertex Pharmaceuticals 13
$55,000 - $99,000 AMGEN Ritu Baral Centerview Partners Sobi, Inc. Amicus Therapeutics Amryt Pharma Spark Therapeutics UCB Chiesi Global Rare Diseases AveXis/Novartis AVROBIO Ultragenyx Pharmaceutical Daiichi Sankyo United Therapeutics Biogen Biotechnology Innovation William & Carolyn Aliski Enzyvant Therapeutics BioMarin Pharmaceuticals Organization WCG Clinical Eversana Greenwich Biosciences bluebird bio Immunovant Inc $10, 000- $24,000 GlaxoSmithKline Bridge Bio Intercept Pharmaceuticals Actelion Pharmaceuticals/ Ipsen Cowen Group, Inc. Invitae Corporation Johnson & Johnson Jazz Pharmaceuticals National PKU Alliance Gilead Sciences Applied Therapeutics JP Morgan Neurocrine Biosciences Harmony Biosciences Audentes Therapeutics Merck Pfizer Inc Innovation Policy Solutions PTC Therapeutics Ionis Pharmaceuticals Blueprint Medicines Ovid Therapeutics Boehringer-Ingelheim PassageBio Mitsubishi Tanabe USA Bristol Meyers Squibb Perkin Elmer Foundation Orchard Therapeutics $25,000- 54,000 PhRMA Burroughs Wellcome Fund Acceleron Pharmaceuticals Parent Project Muscular Dystrophy Castle Creek Biosciences Recordati Rare Diseases Alnylam Pharmaceuticals REGENXBIO, Inc.
Jennifer Bernstein, Member Executive Vice President, Horizon Government Affairs Richard S. Finkel, Member Director of Experimental Neurotherapeutics in Translational Neuroscience Program, St. Jude Children's Research Hospital Amrit Ray, MD, MBA, Member Senior Advisor, Bain Capital Life Sciences
Emil Kakkis, MD, PhD, Founder President/CEO, Ultragenyx
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501(c)(3) nonprofit organization (Tax ID 26-4614274) 1012 14th St, NW, Suite 500, Washington, D.C. 20005 (202) 697-RARE (7273) info@everylifefoundation.org
everylifefoundation.org @EveryLifeOrg
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.
