FOXG1 Research Foundation Co-founder and CEO Nasha Fitter was the first spotlight speaker at the White House Rare Disease Forum hosted by the White House Office of Science and Technology Policy's Health Outcomes Team February 28th ahead of Rare Disease Day 2024.

Nasha Fitter represented the FOXG1 syndrome community as well as the 30 million patients, 15 million children, in the United States living with a rare disease. Nasha spoke about radical funding changes required to support drug development uniquely led by patient organization groups like the FOXG1 Research Foundation.

Following Nasha’s clear and impactful presentation, following speakers from ARPA-H, the Chan Zuckerberg Initiative, and Global Genes cited Nasha’s speech and the work of the FOXG1 Research Foundation to show how efficiently patient organizations can drive drug development if given funding.

Thank you to the White House for giving this platform, listening, and considering how far government funds can go to saving lives and alleviating significant economic burden if distributed among the patient organizations that are swiftly driving drug development because it is their children’s lives at stake

Nasha’s speech has sparked a conversation throughout the rare disease space.

See Nash’s LinkedIn follow up of the forum …

On Wednesday, February 28, as part of the Biden-Harris Administration’s goal to improve health outcomes, the White House Office of Science and Technology Policy will host a forum marking Rare Disease Day and highlighting the Biden-Harris Administration’s commitment to supporting patients and families facing a rare disease and delivering progress against the nearly 10,000 known rare diseases that impact up to 30 million Americans.
 
Leaders from the Biden-Harris Administration and the private and nonprofit sectors will work together to:

• Elevate the experiences and expertise of people and families facing rare diseases;

• Share developments from the Biden-Harris Administration and rare disease community and learn from one another to accelerate progress; and

• Identify opportunities to improve rare disease diagnosis, advance research and access to innovation to deliver effective treatment, and boost support for patients and families.

WHEN: Wednesday, February 28, 5:30 PM – 7:00 PM ET
WHERE: The event will be livestreamed at https://youtube.com/live/uG43oLZzeBE
FEATURED SPEAKERS: 

• Dr. Arati Prabhakar, Assistant to the President for Science and Technology, Director of the White House Office of Science and Technology Policy

• Dr. Danielle Carnival, Deputy Assistant to the President for the Cancer Moonshot, Deputy Director for Health Outcomes, White House Office of Science and Technology Policy

• Dr. Monica Bertagnolli, Director, National Institutes of Health

• Dr. Renee Wegryzn, Director, Advanced Research Projects Agency for Health

• Nasha Fitter, Co-founder, FOXG1 Research Foundation

• Emily Kramer-Golinkoff, Co-founder, Emily’s Entourage

• Dr. David Fajgenbaum, Co-founder, Every Cure

• Shonta Chambers, Executive Vice President-Health Equity Initiatives and Community Engagement at Patient Advocate Foundation

• Dr. Joni Rutter, Director, National Center for Advancing Translational Sciences

• Tamar Thompson, Vice President, Head of Global Corporate Affairs, Alexion, AstraZeneca Rare Disease

• Dr. Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Diseases

• Charlene Son Rigby, Chief Executive Officer, Global Genes

• Tania Simoncelli, Vice President, Science and Society, Chan Zuckerberg Initiative

• Julia Tierney, Deputy Center Director (Strategy, Policy & Legislation), Center for Biologics Evaluation & Research, Food and Drug Administration

• Paul Melmeyer, Vice President, Public Policy and Advocacy, at the Muscular Dystrophy Association

• Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation

• Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement, EveryLife Foundation

CONTACT: For press inquiries related to this event please email press@ostp.eop.gov.
 
BACKGROUND: The Orphan Drug Act defines a rare disease as a disease or condition that affects fewer than 200,000 people. In the United States, rare diseases affect more than 30 million people in total. Of nearly 10,000 known rare diseases, only 5% have a Food and Drug Administration-approved treatment option.
Recent scientific and technological advancements have provided hope for understanding and delivery of new treatments for rare diseases, but significant work remains to address this critical public health issue that effects about 1 in 10 Americans.