People with a rare disease are often highly motivated to take part in clinical research. The nature of rare diseases – the fact that individual diseases have small populations and are not as well researched as common diseases – usually leave patients and caregivers with a lot of knowledge gaps and unanswered questions; so any new information is welcomed.

This sentiment shared by a Raremark member is felt across the rare disease community: “Dealing with a rare disease that no one seems to know anything about is tough.”

Of our sickle cell members surveyed, 82% told us they were only somewhat satisfied or less than satisfied with the information currently available on their condition. They also shared concerns about their pain not being taken seriously and not always being believed by healthcare providers.

In another survey, we asked sickle cell patients about their clinical trial experiences. The top reasons they gave for taking part were: gaining insights into their own health, advancing medical science, and benefiting from the extra medical attention they would receive.

Despite these strong motivations people have for taking part in trials, finding enough who are affected by the rare disease in question, in the right place at the right time, can be a major challenge. So, at the very least, it’s vital to ensure every individual who does express an interest in taking part feels valued and informed.

We speak to people affected by rare diseases day in and day out about clinical trials they want to take part in; pre-screening candidates for our clients, as well as supporting those we refer throughout the screening and enrolment phase. Based on our experience of working with study centers, we know these approaches work best in keeping patients interested and engaged in all kinds of clinical and real-world research.

Things to get right, every time

Always respond promptly

• A prompt response is vital for keeping people engaged – within two days of a referral is ideal.
• Ensure prompt payment of travel expenses and honoraria. There's often a concierge service engaged to sort this on the study site's behalf, but the site coordinator should be familiar with what's available and how the patient can access it.

Respect the time patients are willing to give

• Keep the patient's first appointment, if humanly possible. We have had hard-to-reach patients lose heart and turn their back on research after screening appointments were canceled more than once by study sites.
• Agree a time for the first phone call via email. Patients often have busy, complicated lives themselves – and sometimes have similar working hours to the study center. They may not be instantly available to answer an unexpected call – or, more typically, they are unwilling to answer a call from a number they don't recognize.

Trial partners can help with patient records

• If new patients prove hard to get hold of, or you want to see medical records before bringing them in for screening and you’re too busy to do the chasing, trial partners can help. Tell the trial sponsor you need more support, and be precise and selective about which records you really need to see.

Reassure patients concerned about existing treatments

• Appreciate that many patients will be concerned about the effects of coming off an existing treatment, which is often a requirement of joining a trial for an investigational therapy. They may appreciate a conversation about how to mitigate the effects, and some reassurance about all the tests and procedures involved.

Underline expectations as soon as you can

• Underline what’s expected of trial participants in the first conversation.
• People with a rare disease are often highly motivated to take part in clinical research, but may overlook the most obvious requirements – particularly the number and frequency of visits to the study site and the logistics involved in getting there.

Advise patients on data sharing sensitivity

• Before enrollment, explain what they can tell their friends and family about the trial. Many people living with a rare disease are used to posting frequently on social media, and they may need guidance on what details they shouldn’t post, so the privacy of their personal health information and the integrity of the trial is preserved.

Keep the communication going where relevant

• While they are on the trial, do share with patients any patient-friendly materials or blogs about living well with their condition.
• And create opportunities for communicating with them between study visits.

Where possible, take the trial to the patient, not the patient to the trial

• If study procedures can be done just as well by a study nurse in a visit to the patient’s home, or a video call can replace a site visit, then why not?

Xperiome accelerates treatment development in rare disease by connecting patients to research. Contact us to see how we can support you