Recent Data Shows Positive Attitudes for Decentralized Trials in Rare Disease Patients


87% of people surveyed said they had no concerns about a decentralized approach to clinical trials.

Decentralized clinical trials are already a hot area of interest for CROs and sponsors, but how do patients feel about them, particularly those living with rare diseases? That’s what Raremark, a patient experience network in rare disease, aimed to find out with their recent survey.

Raremark surveyed people around the world living with the following conditions: hemophilia, idiopathic pulmonary fibrosis (IPF), myasthenia gravis (MG), and sickle cell disease. Participants were asked about their attitudes and opinions around clinical trials and whether topical factors like COVID-19 had an impact.

People living with rare diseases are interested in decentralized trials

When asked where they would like to receive trial medication and monitoring appointments, participants’ responses indicated a preference for decentralized trials. 57% of participants said they would like to receive the trial medication through a mix of clinic, home, and nearby hospital visits, and 45% would like the same mix for how they are monitored during trials. Few survey participants said they wanted all of their appointments to take place solely at the clinic or solely at home.

Data from Xperiome

Data from Xperiome

Overall, 87% of survey participants expressed no concerns at the prospect of a decentralized approach to trials.

For those concerned about having all their trial appointments remotely, reservations included: possible adverse reactions to the drug, and not having specialists there to help them; while others said they struggle to comprehend all the trial literature on their own.

Looking at features of decentralized trials patients are most attracted to, Raremark found that people with rare diseases liked the prospect of traveling less or traveling for shorter distances when taking part in trials. While 60% of those asked said they liked the idea of having more of the study take place at home, and less time traveling, 50% also liked the idea of using technology, both to communicate with research staff and to have their health monitored.

Data from Xperiome

Could these results be reflective of the unique nature of rare diseases?

Survey participants generally showed a preference for traveling less and for shorter distances to join trials is partly indicative of the nature of rare diseases. Those few who are diagnosed with a rare condition are typically geographically dispersed and limited in their ability to make multiple long journeys; while sponsors often struggle with determining where best to place trial sites. Raremark has seen many cases where patients committed to trial participation fit the inclusion criteria but don’t have a trial site near them, and travel across multiple states for trial appointments just wouldn’t be practical, or bearable.

Rare diseases can have a huge toll on people’s lives, and in some cases, they have to adapt their whole lives around the demands of their health condition.

In a previous survey, Raremark’s sickle cell community members reported visiting their primary healthcare provider 13 times in a 12-month period. Given this, the idea of making multiple visits to clinical trial sites to take part in a study on top of their regular visits to the hospital can be too much for some. It’s not surprising then to see rare disease patients preferring trial designs where travel is minimized.

One of the greatest features of decentralized trials is the use of technology to improve the patient experience.

Conclusion: ease the burden and bring the trial to the patient!

Sponsors don’t need to completely overhaul their trial designs to make them entirely siteless. As the survey data shows, patients still want some face-to-face appointments, but they just want fewer of them—and closer to where they live.

Sponsors need to look at the trial design, consider more systematically the overall patient burden of participation, and find areas that can be made virtual. For example, this can be easily done for some monitoring and assessment appointments through partnering with a technology vendor that can help patients monitor their health via wearables or a smartphone app.

Additionally, sponsors can reduce site visits by engaging home health nurses to conduct those visits in-home, and by engaging local clinics and hospitals so some appointments can be conducted at the patient’s regular doctor’s office. 

Added benefits for drug launches?

Adopting decentralized trial methods in rare disease trials not only improves patient experience it can also bring sponsors multiple benefits. Removing barriers around travel and site visits helps to increase trial retention and keep drug development timelines on track. Seeing patients in their own homes, in a real-world setting, can also help researchers understand how the treatment they are testing is impacting the patient’s day-to-day life.

In addition, people who have been highly engaged in a trial may also advocate for the treatment once it’s approved and ready for launch and they may be more open to helping researchers with follow-up studies.

Want to learn more about Raremark’s findings?

You can see the full report on patients’ clinical trial willingness in rare disease here.

Jeremy Edwards is the CEO of Raremark

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