Xperiome: The Newest in Rare Disease Research, Therapy, and Patient Advocacy

When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting the needs of the rare disease community without engaging within that community? According to Charcot-Marie-Tooth News, this is how the Xperiome platform was born. Developed by Raremark, an online patient community for those with rare diseases, Xperiome is designed to include more patient insights into research, thus spurring the search for new therapeutic options.


While Raremark is marketed more towards patients with rare diseases, Xperiome is marketed towards pharmaceutical companies. On its website, Xperiome explains how it helps the pharmaceutical industry create more targeted, innovative, and effective drug candidates:

Design robust trial protocols fit for the new normal, including decentralized and hybrid models, with meaningful patient input. Engage, recruit and retain hard-to-reach patients in clinical studies globally, in line with the highest ethical standards. Leverage the Xperiome platform to expedite your route to regulatory success.

By leveraging the Xperiome platform, the company suggests that pharmaceutical organizations will have increased help in reaching regulatory milestones. For data analysis, the platform uses machine learning and behavioral science to understand the patient experience. Scientists and researchers are able to connect with over 11,000 patients and family members who have rare diseases such as hemophilia, cystic fibrosis (CF), or myasthenia gravis (MG).

In addition to regulatory assistance, Xperiome provides resources for clinical development, marketing, commercialization, and receiving patient feedback. For example, the company explains that pharmaceutical companies can “expand their market” by:

[capturing] data on patient satisfaction with new medicines, longitudinally and across international markets. Evaluate the effectiveness of new treatments – globally and in new indications – in the real-world setting, [and] succeed in an increasingly challenging reimbursement landscape with the Xperiome platform’s patient-experience data.

By offering patients  studies, surveys to explain their experiences, and other opportunities for engagement, Xperiome allows patients to advocate for their own needs. If you are a patient with a rare disease whose condition is not listed on this platform, or a researcher looking to study an unlisted rare disease, please contact hello@raremark.com.

Interested in some of the current studies? Check out what is running and what has been completed here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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