RARE Daily

SCD Foundation Enters Digital Health Collaboration with FDA to Bridge Health Care Gaps

June 5, 2024

Rare Daily Staff

The Sickle Cell Disease Foundation entered a research collaboration with the U.S. Food and Drug Administration’s Center for Devices and Radiological Health to develop best practices for the safe and effective use of artificial intelligence and machine learning systems and digitally-derived measures that matter to patients.

Specifically, the collaboration will evaluate how digital health technologies can be used to better understand patients’ real-world experiences and evaluate meaningful benefit to those living with rare diseases, like sickle cell disease, for medical product development and public health purposes.

“Despite the recent advancements in treatment, individuals living with sickle cell disease continue to face significant challenges when receiving care. Inadequate knowledge of the disease among health care professionals, lack of research, and poor care coordination, among a few, lead to significant disparities in health outcomes,” said Mary Brown, president and CEO, Sickle Cell Disease Foundation.

The collaboration between the FDA and SCDF will lay the groundwork for best practices in the safe and effective use of AI and ML approaches for the development of novel digitally-derived endpoints. By analyzing longitudinal, de-identified patient-generated health data (i.e. granular biometric and self-reported symptoms data), socio-environmental data, and health information, collected from Institutional Review Board-approved studies led by SCDF in partnership with the Center for Inherited Blood Disorders (CIBD) and The Hills Tandem, the collaboration will develop a framework and software solutions to deploy on the data to help predict outcomes from lived experiences data. In addition, the research may provide insight into the use of real-world data to better design post-approval studies and labeling.

“Collaborating with SCDF will help us to better understand and measure what matters most to patients and advance health equity, with the goal of improving the lives of those living with sickle cell and other rare diseases,” said Vinay Pai, digital health specialist in the Digital Health Center of Excellence at the FDA.

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