Rare Answers™: A System to Improve Patient Outcomes

Feb 25, 2021 10:00 AM ET

At Alexion, Corporate Social Responsibility (CSR) is an inherent part of everything we do and supports our mission to transform the lives of people affected by rare diseases and devastating conditions. In addition to developing and delivering life-changing medicines, this mission drives us to seek opportunities to advance innovative solutions to system-wide challenges faced by rare disease patients, such as the “diagnostic odyssey.”

For the 400 million people around the world who are affected by a rare disease, half of whom are children, it can often take years to receive an accurate diagnosis and many never receive one at all. For these patients, every minute counts and a fast and accurate diagnosis would create a pathway to care that is critically needed. Yet, with more than 7,000 known rare diseases and more medical data available to us than ever before, it can be difficult for physicians to correctly diagnosis a patient and guide them to the best available care plan.

Rare Answers™ is a system of innovative and sustainable tools designed to help shorten the diagnostic journey and time to initiate treatment for children with a rare disease. Developed in collaboration with leading children’s hospitals and technology and data-science companies, Rare Answers tools are designed to deliver actionable data to physicians by analyzing phenotypic and genomic data and incorporating precision software, medical information, and clinical insights.

Pilots of this approach have achieved world records in reducing the time to diagnosis and treatment of children in NICUs and PICUs. Because Rare Answers is both personalized and scalable, it has the potential to be easily accessed by patients and physicians regardless of geography.

Alexion has acted as steward of this program for the past five years, investing as part of our commitment to meeting the needs of all rare disease patients.

For more information on Rare Answers tools, download the fact sheet and FAQs, read the press releases announcing Alexion’s collaboration with Rady Children’s Hospital and The Manton Center at Boston Children’s Hospital, or visit the Research and Development page of Alexion.com.

To learn more about how Alexion is redefining what it means to live with a rare disease, visit Alexion.com