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Our perspectives on news and trends in rare disease

Patients can be powerful agents of change in health. Learn how we turn patient experience into insights that help pharma get to know rare disease patients and their conditions faster.

Articles and publications

Our latest insights and news on rare disease, and how we’re helping drive change through research.

White Paper

January 2022

Patient-driven insights into the impact of COVID-19 on the rare disease community

The COVID-19 pandemic had a profound impact on people around the world, but arguably none more-so than the rare disease community. Xperiome recently partnered with Magnolia Innovation, a healthcare research consultancy, to explore how COVID-19 impacted the rare disease community including changes in patient and caregiver behaviors and the emotional toll of the pandemic.

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Media Article

June 2021

Rare Disease Patients Overlooked During COVID

Xperiome CEO Jeremy Edwards shares the findings from our latest rare disease survey with Applied Clinical Trials

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Media Article

May 2021

Why relationships are key in rare disease patient engagement

Xperiome CEO, Jeremy Edwards, speaks to Pharmaphorum about how addressing the unmet needs of rare disease patients means thinking beyond individual studies

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Case studies

Discover how we’re making our mark in research and accelerating the development and reach of new treatments.

Case Study

Patient preferences for injectable drug-delivery options

A pharmaceutical company commissioned Xperiome to survey our sickle cell community to understand how people living with the condition handle their pain crises and hospital visits, and how they would feel about different ways to take or receive a new injectable medication. Specifically, the client wanted to know if any factors influence preference for auto-injector devices over intravenous (IV) infusions.

Case Study

Longitudinal, real-world data to support US FDA submission

A specialist pharmaceutical company commissioned Xperiome to recruit a group of people living with pouchitis, a rare gastrointestinal condition, and to track how it affects their lives over a six-month period. The objective was to generate real-world data to support a future regulatory submission to the US FDA, for approval of the company’s investigational drug.

Case Study

Phase I study to assess safety and tolerability of investigative medication in pediatric patients with Sanfilippo syndrome (MPS IIIA)

A specialty healthcare company developing treatments for rare diseases sought Xperiome’s support for an early-stage pediatric trial program testing an investigational medicine in Sanfilippo syndrome. Xperiome was commissioned to run a nine-month online campaign to raise the profile of the program and to find and refer up to four of the nine patients needed for the Phase I study in the United States; to a study site that initially had no patients of their own.

Case Study

Phase II study to evaluate safety and efficacy of investigative medication in adults with generalized MG

A clinical-stage biopharmaceutical company developing treatments for autoimmune diseases brought in Xperiome to boost recruitment to a Phase II study of an experimental drug for generalized myasthenia gravis (MG); a super-hot area for clinical research experiencing high competition for eligible patients. By reaching out to Xperiome’s online community of MG patients, which is the largest in the world in this condition, we were able to boost enrollment by 25% within 90 days - saving the sponsors at least three months off their recruitment timeline.

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Get in touch

Interested in hearing more? Feel free to reach out to us, and we’ll make sure to get back to you as soon as possible.