How best to keep people with rare diseases engaged in clinical trials
October 2020Lessons from Raremark’s day-to-day interactions with interested patients.
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 leaves patients and caregivers with a lot of knowledge gaps and unanswered questions; so any new information is welcomed.
This sentiment shared by a Raremark community member is felt across the rare disease community: “Dealing with a rare disease that no one seems to know anything about is tough.”
Things to get right, every time
- 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.
- 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.
- 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.
- 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 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.
- 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.
- 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.
- 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?
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