Over 400 million people around the world are affected by rare diseases. Approximately half are undiagnosed, and receiving a diagnosis takes an average of five years. Even after diagnosis, treatment can be challenging: 95% of rare diseases lack an FDA-approved medicine in the U.S. The daily burdens of symptom management and treatment can be complicated by various obstacles, including limited access to transportation for appointments, frequent absences from school or work, significant out-of-pocket expenses, and bureaucratic barriers or inadequate reimbursement options. Caregivers, who play a critical role for many patients (especially pediatric patients), are affected by heavy emotional, financial and social burdens. Both patients and their caregivers often experience loneliness, isolation, and stigma. These challenges persist in all countries at various income levels around the world.

Nonprofits and nongovernmental organizations (NGOs) - especially patient advocacy groups - are uniquely poised to help address these unmet needs. Through their direct engagement with patients, caregivers, and other stakeholders, patient advocacy groups foster a sense of community, advocate for their needs, and empower individuals to take agency over their health. Additionally, they serve as drivers of research, treatment advancements, and broader initiatives focused on quality of life.

Innovations driven by NGOs and nonprofits have an important role to play in improving the experience and wellbeing of patients with rare diseases. Patient registries and databases can facilitate collaborative research and accelerate new treatments. Educational tools can help patients and their caretakers better understand their diagnosis and treatments. Biomedical research approached in new and creative ways can make cures and personalized medicine more accessible. All innovations will need an ecosystem to scale, linking researchers, clinicians, and communities in pursuit of better serving patients.

The Amgen Prize (formerly The Horizon Prize) seeks innovative solutions from nonprofit organizations that improve the journey for patients with rare diseases, from diagnosis to treatment and holistic care. The Amgen Prize has a particular interest in solutions that: 

• Improve the rare disease diagnostic journey – reducing the time, cost, resources, and duplicative travel and testing for patients and caregivers.

• Optimize holistic care for people with rare diseases and their caretakers—including physical, mental, social, and other types of support.

• Promote community and connection among patients with rare diseases and their caregivers.

• Support daily care management for patients and/or their caregivers.

$150,000 in prize funding is available for one winning solution team for The Amgen Prize.

Glossary of Key Terms:

• Nonprofit/Non-governmental organizations/NGO:
  • While the term “NGO” is widely used, there are also many other overlapping terms used such as “non-profit,” “voluntary,” and “civil society” organizations.  NGOs are self-governing, private, not-for-profit organizations. Please note that this Prize is open to nonprofit tax-exempt organizations in the U.S., or its equivalent outside of the U.S. as determined by Amgen. Eligibility for this award is subject to Amgen’s sole determination. Please see the Eligibility Criteria here.
• Innovation

  • Includes a wide variety of approaches that lead to new products, processes, or services, or improvements to existing ones. Innovation can come from new technologies, new applications of existing technologies, new organizational models, or something else. Adapting existing work to reach new stakeholders or communities, particularly those who have less access to affordable healthcare, is also innovative.