Community building empowers rare-disease patients: Xperiome

By Jenni Spinner

- Last updated on GMT

(Kyryl Gorlov/iStock via Getty Images Plus)
(Kyryl Gorlov/iStock via Getty Images Plus)

Related tags Rare disease Orphan drug Patient centricity patient engagement Patient recruitment

An executive from the rare disease technology solutions company discusses how forcing connections with patients can boost research into such conditions.

Jeremy Edwards, CEO of Xperiome, spoke with Outsourcing-Pharma about the unique challenges rare disease patients and researchers face, and how connecting the two groups can benefit both in many ways.

OSP: What gaps and unfulfilled needs in treatments and the healthcare system rare-disease patients find themselves in that your company seeks to address?

JE: The nature of rare diseases – the fact that individual diseases have small populations and are not as well researched as common diseases - means that rare disease patients and their caregivers face many challenges. They can struggle for years to get a proper diagnosis. Once they finally get a diagnosis, information can be challenging to find, leaving patients and caregivers with knowledge gaps and unanswered questions.

We’re working with patients, parents, and caregivers to build a knowledge bank of their lived experiences. We’re empowering them to raise their voices, pool their wisdom to support others, and help the industry understand what it’s like to live with a rare disease.  Where are the gaps, and what areas they need support?

As part of that journey, we’re providing an opportunity for patients to take part in research. We’re connecting researchers to the right data, the right insights, and the right people for their studies. By bringing patients closer together with those studying rare diseases, we help pave the path to better treatments and even cures.

OSP:  Why is community building (in every sense of the phrase) helpful to patients, researchers/drug developers, caregivers, and other stakeholders?

Jeremy Edwards, CEO, Xperiome

JE: At Xperiome, many of our team members have personal experiences of having a rare disease or having a family member with a rare disease. We know firsthand that sometimes patients and caregivers want to know the day-day details of living with a rare condition. It helps to learn from others about dealing with similar situations, such as communicating with physicians who may not be familiar with your disease or managing symptoms and pain in college or at work.

That’s where the value of crowdsourcing wisdom comes in. We have found that the biggest driver of change in rare diseases is families. Patients and caregivers often know more about their disease than their doctors as they are continually searching for information, seeking out others with the same diagnosis, and sharing knowledge.

Working with patients and families in this way, and getting their input throughout the drug development process, provides researchers with unique insights into the experiences of living with the condition. These insights inform pharma, helping demonstrate the value of the treatments they’re developing and unlock a deeper understanding of rare diseases.

Together, this crowdsourced wisdom and powerful insights lead to better treatments and, ultimately, improve patient lives.

OSP: Then, how does Xperiome work to connect patients to research options?

JE: At Xperiome, we are working to transform orphan drug research by helping pharma get to know rare disease patients.

We do this through Raremark, a knowledge bank for the lived experience of rare disease. Raremark supports every stage of a patient’s journey, enabling members to share their real-world experiences and learning from the crowdsourced wisdom of others. Importantly, we have now opened up Raremark to anyone affected by any rare disease, breaking down barriers between conditions enabling more members to find what they need when they need it.

Raremark is unique from other health communities. We recognized that experiences are not isolated to specific conditions but rather shared across multiple indications.  The connection across indications creates a supportive environment for the rare patient not seen anywhere else. 

We educate members about the importance of research and how their involvement will help make rare disease treatments available faster. Based on their profile and preferences, we can then present them with appropriate research opportunities. 

OSP: What do you enjoy most and find most fulfilling about your work at Xperiome?

JE: We work in such a unique space, and there are so many different ways to make things better for people with rare diseases. There are opportunities to help patients, parents, and caregivers find information and share their experiences and develop potential therapies for these underserved conditions.

Within rare diseases, the number of people living with any particular condition is small, but our impact is considerable. Being connected closely to that process is incredibly rewarding.

OSP: Can you share any information or insights about life with a rare disease that most health researchers/caregivers might not realize?

JE: There is a wealth of insights that can be unlocked by understanding the world of people living with rare diseases. We continue to learn more just by breaking down barriers between rare conditions. One example is the frustration of not being taken seriously in the emergency room.

Something else we’ve learned is that researchers need to think broader about treatments. They need to consider how drug administration, their side effects, and the potential disruption they could cause in a patient and caregiver's life.  People get tired of infusing intravenously or visiting the clinic every other week – or throwing up after taking their medicine. It can interrupt their quality of life and even affect their relationships.

Medically these treatments address the complications associated with a rare disease, but if they don’t consider the patient's experience of taking medicine regularly and the disruption and distress this may cause, will the treatment be effective?

OSP: Finally, do you have any new technologies, services, or other announcements about Xperiome you can share with our readers?

JE: We’re in a fascinating phase of development at the moment. You may have heard that we recently rebranded to better serve our two significant but very different audiences: patients & caregivers, and our pharma clients. Raremark has expanded from our core nine rare indications to all 7,000+ known rare conditions, allowing us to help more people affected by rare diseases and unlock more profound insights into the rare patient experience.

I’m honored to be part of such a fantastic team working to change the face of rare disease. The vision that runs through every aspect and employee of our business is the same: we want to shape a world where all rare diseases are understood and treated.

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