1st Patient Data Platform Seeks to Boost Care, Treatment Development

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The HypoPARAthyroidism Association has launched the first-of-its-kind Patient-Powered Data Platform, a repository to help understand the disease journey and develop better treatments for people with hypoparathyroidism.

Interested participants — patients, caregivers, or those who have lost a loved one to the rare disease — can enter patient data about common symptoms of hypoparthyroidism, medical history, and quality of life, which will become available to researchers in their clinical trials and to clinicians to help them with treatment decisions.

“Few clinicians and healthcare providers have direct experience treating patients living with this rare disease, and so few understand its impact,” Dolores Shoback, MD, medical adviser at the HypoPARAthyroidism Association, said in a press release.

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“The Patient-Powered Data Platform is a new tool that we are certain will become an essential community-driven resource for advancing collective understanding of HypoPARA, supporting the safe and rapid development of new therapies and better treatment paradigms to improve the health and quality of life for patients,” Shoback said.

“I hope those within the medical and healthcare communities who have contact with these patients will encourage them to learn more about the Patient-Powered Data Platform and consider participating in it,” she added.

The program is the result of a collaboration with RARE-X, a nonprofit that aims to accelerate rare disease research and treatments. RARE-X leverages technology developed by the Broad Institute of MIT, Harvard, and other partners to create a hub for patients, clinicians, and researchers.

“The Patient-Powered Data Platform will also make it easier to identify endocrinologists and other specialists who may have expertise in treating HypoPARA,” said Deb Murphy, director of research and studies, and former president at the HypoPARAthyroidism Association. “Ready access to such information is important for helping to ensure that known or suspected HypoPARA cases are diagnosed accurately and treated effectively on time.”

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The repository is intended to help researchers understand long-term changes in symptoms and responses to therapy. This will improve clinical trial design and data collection strategies, while boosting participation in clinical studies, according to the association. Moreover, it will provide researchers with natural history data — how a disease progresses in the absence of treatment — instead of having to assign patients to a placebo (control group).

This type of platform seeks to speed the development of potential therapies and shorten the time to make them available to patients.

“Understanding the signs, symptoms, diagnostic criteria, causes, and treatment paradigms for HypoPARA is critical for providing patients with optimum care, and systematically collecting such data is critical for enabling the innovation necessary to develop new treatments,” Murphy said.