Why relationships are key in rare disease patient engagement

Real-world data is a vital part of rare disease drug development, but to get a true picture of patients’ unmet needs pharma should take a broader view of their lived experiences, says Xperiome’s Jeremy Edwards.

It is difficult to get data on rare diseases and the people who live with them by their very nature. But these patients face unique challenges in their everyday lives that pharma needs to account for when developing treatments, and understanding these challenges requires strong real-world data.

Jeremy Edwards, CEO of Xperiome, believes that bringing patients from across different rare disease areas together will allow pharma to get a better picture of how they can truly address unmet needs.

He says he would like to see the industry engaging rare disease patients at every stage of drug development.

“Companies need to learn to think beyond individual studies and develop an understanding of the day-to-day experiences of those living with the disease. That will enable companies to gain deeper insights into aspects of treatment they might not have otherwise considered.

“Plus, there are many ways rare disease patients can contribute to development beyond the constraints of clinical studies by engaging in long-lasting relationships with pharma.”

This philosophy was behind Xperiome’s recent decision to split into two different functions – Raremark, a community where patients and carers can share their experiences of living with rare diseases, and Xperiome, which leverages those insights for pharma companies looking to develop drugs for these conditions..

Edwards says the idea is to create a single ecosystem for rare disease patient insights, with pharma able to investigate patients’ “real, lived experiences”.

“It’s always a challenge to find patients and gather data for rare diseases – and most valuable insights don’t necessarily come from single touchpoints.

“Developing a true understanding of the patient is all about the relationship you build with them, but that’s been difficult for pharma in the past.”

Such “lived experience” insights might include why someone would or would not want to participate in a clinical trial, why they would want to change their medication, or what it’s like to be a parent of a child with a rare disease and work full time.

“There are so many pieces to the puzzle that have to be properly assembled, but typically pharma only collects data based on specific study objectives and goals,” says Edwards. “There’s a missed opportunity there to widen the spectrum and see the complete picture of real-world experiences, not just a clinical outcome.

“From there companies can even look beyond the initial problem they were trying to solve – and they might realise there’s so much more they can do just by developing that relationship.”

Edwards says a common thread he sees from these insights is that rare disease patients are “always looking for answers”.

“There are so many pieces to the puzzle of patient experience, but typically pharma only collects data based on specific study objectives and goals”

“Many times what we’ve found is that patients just want to be heard. They want options and they want information, which is often hard to find. They are also highly motivated to support other people who have been recently diagnosed.

“Pharma is starting to realise that tapping into that and building a relationship based on those concepts of value makes it much more likely for these patients to want to participate in clinical research and other activities.”

In other words, Edwards says, pharma needs to move from “asking” to “listening and giving back”.

“A two-way dialogue is key.”

Since Xperiome interacts with people from many disease areas, the company has also seen the value in finding commonalities in experiences across different conditions.

“We realise that patients aren’t defined by their indication, and in many instances they may have different diseases but shared lived experiences,” Edwards says.

“For example, in many cases parents of children with different rare diseases are going to face a lot of similar struggles. Being able to share those insights can be incredibly powerful.”

Xperiome uses machine learning and behavioural science to identify and analyse these commonalities and other aspects of the patient experience – for example by categorising responses to patient surveys based on the COM-B (Capability, Opportunity, Motivation – Behaviour) model.

“Models like that can tell companies what they need to focus on in order to further drive patient engagement and build that relationship based on what people with rare diseases really want,” says Edwards

He adds that, ultimately, taking this view of rare disease patient engagement will not only improve pharma’s relationship with the people it serves, but also result in market benefit.

“I think pharma genuinely wants to have that patient-centric view, but with standard approaches to data collection they’re unable to achieve a two-way dialogue. We want to help the industry build a true relationship with advocacy groups and patients and allow them to learn from one another.”

About the interviewee

Jeremy Edwards is CEO of Xperiome. For nearly 25 years, Jeremy has focused on aiding the biopharmaceutical and health sciences industry in the development of key compounds and new therapies. His background includes leadership positions across the clinical development continuum; from full-service CROs, to highly specialised imaging modalities, to niche patient-focused service providers.

About the author

George Underwood is the editor for pharmaphorum’s Deep Dive digital magazine. He has been reporting on the pharma industry since 2014 and has worked at a number of leading publications in the UK.