Stronger Together: The Importance of Collaboration in Rare Disease Advocacy

Quick Overview

Annie Kennedy, the keynote speaker at the 2023 FAST Science Summit, emphasized the importance of collaboration in rare disease advocacy. She discussed the work of the Every Life Foundation for Rare Diseases, which brings together patient advocacy organizations, biopharmaceutical partners, and other coalitions. Kennedy highlighted the need to protect and enhance regulatory incentives for therapeutic development in rare diseases. She also discussed the role of patient experience data in drug development and the importance of centralizing rare disease expertise at the FDA. Kennedy urged the rare disease community to continue advocating for increased funding and support for rare diseases.

Introduction

I am honoured to be here today to talk about the importance of collaboration in rare disease advocacy. As the keynote speaker, I want to share my story and the work of the Every Life Foundation for Rare Diseases. Together, we can make a difference and show that rare is not rare when we come together.

The Every Life Foundation for Rare Diseases

The Every Life Foundation for Rare Diseases is a rare disease organization that brings together patient advocacy organizations, biopharmaceutical partners, and other coalitions. Our goal is to work collectively to advocate for the rare disease community. We have two arms: our policy arm, known as our community congress, and our advocacy arm, known as rare disease legislative advocates.

Collaboration with FAST

FAST has been a valuable partner in our advocacy efforts. They have actively participated in initiatives such as Rare Across America and have been leaders within our advocacy arm. We are grateful for their engagement and support.

Policy Priorities

Our policy priorities are shaped by our community partners, including FAST. These priorities include:

1. Eliminating the diagnostic odyssey in rare diseases
2. Expediting therapeutic development for rare diseases
3. Informing access and value decisions in a proactive manner

By working together, we can ensure that the voices of the 30 million Americans living with rare diseases are heard by Congress.

The Economic Impact of Rare Diseases

Rare diseases have a significant economic impact, with an estimated close to a trillion dollars spent on living with rare diseases in the US in 2019. However, 60% of these costs are borne by families and society. It is crucial that we raise awareness of the economic burden of rare diseases and advocate for increased funding and support.

Tools and Resources for Rare Disease Advocacy

We have developed various tools and resources to support rare disease advocacy. These include:

• The ICD Code Roadmap: A roadmap to help organizations navigate the process of obtaining an ICD code for their rare disease.
• The Guide to Patient Involvement in Rare Disease Therapeutic Development: A comprehensive guide that provides insights and case studies on how patient communities can be involved in the development of therapies.
• Legislative Policy Grid: A tool that helps us prioritize legislative issues and track their progress on Capitol Hill.

Ensuring Equitable Access and Support

We are committed to ensuring that all members of the rare disease community receive equitable access and support. This includes advocating for the needs of smaller rare populations and pushing for the inclusion of rare diseases in newborn screening programs.

Advocacy Opportunities

We encourage individuals and organizations to get involved in rare disease advocacy. Opportunities include participating in Rare Disease Week, engaging in Rare Across America, and taking advantage of our advocacy programs and resources.

Conclusion

Collaboration is key in rare disease advocacy. By working together, we can amplify our voices, raise awareness, and advocate for the needs of the rare disease community. Let us continue to support one another and strive for a future where rare is not rare.