Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research.

The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the London-based company said.

Xperiome works by using machine learning — a method of data analysis — and behavioral science to collect real-world experiences from patients that may then help scientists and companies reach their goals of patient involvement.

With an online community of more than 11,000 patients and caregivers, Xperiome connects those living with a wide variety of rare diseases, which are defined as disorders affecting less than 200,000 people in the U.S. The European Union defines a disease or condition as rare if it affects fewer than 1 in 2,000 people within the general population.

Examples within Xperiome’s network include cystic fibrosis, myasthenia gravis, sickle cell disease, hemophilia, and idiopathic pulmonary fibrosis.

The shared guidance from this community can be leveraged to help pharmaceutical companies find and retain hard-to-reach patients, and to monitor the effectiveness of new therapies in real-world settings. Such real-world knowledge is unavailable elsewhere, Raremark says on its website.

In addition, pharmaceutical companies may use Xperiome’s platform throughout clinical development and commercialization to connect with the right patients at the right time, and to complete datasets. Overall, Raremark sees patient experiences — as gathered through this digital platform — as a key to improving the search for therapies for all rare diseases.

“The missing puzzle piece is often the patient perspective, especially in rare disease, where patients are so much harder to find,” Alex Garner, the company’s chief operations officer, said in a press release.

“The Xperiome platform enables pharmaceutical companies and contract research organizations to connect with these hard-to-reach communities and uncover key insights that make the drug development process smarter,” Garner said.

Raremark currently supports longitudinal studies via Xperiome to collect long-term data. Examples of active and completed studies, in which participants are asked about their experiences with certain medications, are available here. The company welcomes requests at [email protected] to include disorders not currently listed.

To further give voice to people in the rare disease community, Raremark hosts the podcast Raremark Voices, in which patients, family members, and healthcare professionals tell their stories.

The company’s founder, Julie Walters, has been featured on the podcast Talking Precision Medicine, hosted by Genialis. Walters has a degree in molecular genetics and a background in popular journalism. She was named a Female Entrepreneur of the Year at the UK’s 2017 Enterprise Awards.

Earlier this year, Raremark partnered with TriNetX, to help connect rare disease patients with applicable clinical trials.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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