Innovators in Rare Disease Diagnosis and Care Compete for The Amgen Prize and $150,0000 in Prize Funding at the Concordia Annual Summit

Pictured: Rick V., who lives with Thyroid Eye Disease (TED), and his wife and care partner Debby

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  • Over 400 million people around the world are affected by rare diseases. Receiving a diagnosis can take years, if at all. Even if diagnosis is possible, treatment can be challenging due to the lack of FDA-approved medicine in the United States. This all leads to daily burdens including symptom management, complicated treatment, limited access to transportation, frequent absences from school or work, high out-of-pocket expenses, stigma, and heavy emotional burdens for both patients and their caretakers. These challenges persist in all countries at various income levels around the world.
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  • This is why Amgen, a Core Programming Sponsor of the 2024 Concordia Annual Summit, has collaborated with MIT Solve to launch The Amgen Prize (formerly The Horizon Prize). The Amgen Prize is a global award recognizing non-profit organizations that develop innovative solutions to improve the journey of patients with rare diseases, from diagnosis to holistic care. This includes solutions that address critical areas such as reducing the time, cost, and resources involved in the diagnostic process, optimizing comprehensive care for patients and caregivers, fostering community and connection among those affected by rare diseases, and enhancing daily care management. The recipient of The Amgen Prize will be awarded $150,000 in funding to further scale their innovative solution.
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• This year’s finalists will pitch their solutions to a panel of distinguished judges on the Concordia stage on September 24 in New York City. The Amgen Prize recipient will be announced live at Concordia the following day. Finalists include:
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• Care Management for the HPS Network “Pop-Up” Clinics in Puerto Rico—pitched by Donna Appell, Executive Director of Hermansky-Pudlak Syndrome Network Inc.

• A cohesive care management system to facilitate improved health outcomes for this under-resourced rare community.

  Data Coordination Center for Individualized Treatments—pitched by Winston Yan, Founding President of The N=1 Collaborative (N1C)

• Introduces open data sharing across individualized, N-of-1 therapeutic programs to improve safety and efficacy for today’s and tomorrow’s rare disease patients.

  Factor H La Luz Caregivers Programme—pitched by Ignacio Muñoz-Sanjuan, President of Factor H

• A practical, financial, and emotional support programme for the caregivers of people with Huntington’s Disease in Venezuela.

  SARA-Q—pitched by Mincheol Song, Director of Research at Voice of Calling NPO

• A tool for quantitative and extensive data collection in the assessment of patients with rare disease ataxia. 

  The Odylia Collective—pitched by Ashley Winslow, CEO and CSO of Odylia Therapeutics

• A centralized marketplace that ensures drug development resources are accessible to patient groups and the rare disease research community. 

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• These groundbreaking innovations exemplify the essential role of partnerships in combating rare diseases. Concordia is proud to continue to support The Amgen Prize that brings attention to innovation for patients with rare diseases and is honored to showcase the work of these impactful organizations in collaboration with Amgen and MIT Solve at the 2024 Annual Summit.

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• Finalist pitches will take place during the Concordia Annual Summit on September 24 from 1:30pm- 2:30pm EDT and The Amgen Prize winner announcement will take place on September 25 at 11:15 am EDT.
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• For more information on Annual Summit sessions and speakers, please view the agenda. 
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