3 Key Takeaways From the 2024 #RAREis White Paper

Addressing solutions in the rare disease community for more than 400 million people worldwide has been a significant challenge for industry, advocacy groups and rare disease families alike.

In 2023, Amgen collaborated closely with 44 rare disease organizations who were recipients of the #RAREis Global Advocate Grant program, representing 14 countries across the globe. Through a survey and a live summit, the group shared the biggest issues their communities face as well as opportunities to accelerate progress. The new white paper sharing their feedback was published today and can be downloaded here.

The 2024 #RAREis White Paper highlights the key issues facing rare disease advocates and demonstrates how a global approach to collaborations and insights sharing across disease states can fuel progress for the rare disease community at large.

While many challenges in rare disease are specific to the rare condition itself, other critical issues face many rare disease states, including:

• Access to adequate care attributed to the location of care, lack of provider education and financial burdens of care
• Organizational funding limitations impacting championing attention, providing comprehensive patient education and resources and the ability to attract and retain staff and volunteers at advocacy organizations
• Impact of mental health for both rare disease individuals and caregivers leading to feelings of isolation, desperation and limited resources for dealing with a life-altering experience navigating a rare disease diagnosis
• Diversity, Equity and Inclusion challenges surrounding inclusive patient care for rare disease families and equity in clinical research around the world due to historical marginalization and underrepresentation of diverse rare disease communities in research efforts
• Transitions of care faced by young rare disease individuals and connections to established care pathways for aging into adulthood

The themes identified above are all areas of opportunity and ripe for change. The advocate participants identified meaningful learnings that can be implemented by anyone supporting the rare disease community to make a difference:

1. Rethink local activation for global impact – By sharing insights and opportunities, the community can foster accelerated momentum through continued like-minded efforts and information sharing.
2. Community leaders shouldn’t approach problems alone – We’re better together and collaborations should be true partnerships that leverage the strengths and contributions of all partners involved while addressing mutual goals to advance the state of care.
3. Think longer term – It’s critical to focus on multi-year and ongoing commitments to maximize and accelerate impact overall.

The launch of the white paper also coincides with the kickoff of the 2024 #RAREis Global Advocate Grant application opening. Applications for this year’s grant program are open until May 31.

Learn more about how the #RAREis Global Advocate Grant program has engaged advocates from around the world, providing grant support at the micro level to generate macro momentum and have your advocacy organization apply for funding today!