Changing Policy, Saving Lives

ANNUAL REPORT

Table of Contents

Responding to a Crisis 2 EveryLife Foundation Team 3 Changing Policy, Saving Lives 4

Greater than Covid-19 5- 6 Advocates in Action 7 Lifting Every Voice 8 Every Voice Matters 9- 10

Engaging New Patient Communities 11- 12

2020 Income and Expenses 13- 14 EveryLife Foundation Board of Directors 14

everylifefoundation.org @EveryLifeOrg 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.

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

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 Elissa Taylor Associate Director of Alliance Development etaylor@everylifefoundation.org

Policy, Advocacy, and Patient Engagement

Kylie Barber Medical Foods Policy Associate kbarber@everylifefoundation.org Annie Kennedy Chief of Policy and Advocacy akennedy@everylifefoundation.org

Katelyn Laws RDLA Program Coordinator klaws@everylifefoundation.org

Liesl Broadbridge Policy Fellow lbroadbridge@everylifefoundation.org

Lindsey Cundiff Associate Director of Patient Engagement lcundiff@everylifefoundation.org

Swapna Kakani RDLA State Advocacy Fellow skakani@everylifefoundation.org Jack Meloro Community Congress Program Coordinator jmeloro@everylifefoundation.org

Adrian Palau-Tejeda Diversity Inclusion Advocacy Fellow Apalau-tejeda@everylifefoundation.org 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

Communications & Marketing

Brenda Colmenares Communications Manager bcolmenares@everylifefoundation.org Britta Dornan Senior Director of Communications and Marketing bdornan@everylifefoundation.org

CHANGING POLICY, SAVING LIVES

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:

Close the innovation gap

Eliminate the diagnostic odyssey

Improve the regulatory process

Ensure patient access to therapies and cures

Empower the patient voice 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.

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.

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.

Information Hub

Launched an online COVID-19 Action Center in March to provide resources and information to the community.

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.

Rare Disease Congressional Caucus Briefing

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.

TELEHEALTH

IS IMPORTANT

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

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

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

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. Scientific Workshop

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.