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Patient Finding for Rare Disease Studies


Rare diseases are defined as those affecting fewer than 200,000 people. Trials in rare diseases present unique challenges for recruitment due to the very limited patient population. However, a number of strategies can improve enrolment for these trials. Methods used to attract and retain rare disease patients for trials, are more often than not being adopted for non-rare disease trials as trial sponsors struggle to meet recruitment goals.

A strategy often adopted is partnering with patient advocacy and support groups which can leverage their connections to the rare disease community for finding willing participants. These groups often maintain patient databases and run social media networks which are ideal for spreading trial awareness.


Expanding the geographic scope of recruitment reaches more of the highly dispersed rare disease patient population. Multi-national trial sites, remote participation through DCT design/ Telehealth, and travel support allows for a broader participant pool. Incentives and enhanced compensation can also assist patients with logistical difficulties attending trials far from home.


Something rarely used; employing mass media campaigns will allow you to reach untargeted/ unknown rare disease patients for potential volunteers. Local newspaper advertisements, social media promotion, TV advertisements, Podcast and radio mentions raise general disease and trial awareness. The downside being that untargeted, mass media is often very expensive and may result in reaching little to no rare disease patients.


Identifying specialist medical centres with significant rare disease patient populations can boost enrolment. The physicians running these specialist centres will have in-depth patient knowledge, and be able to accurately diagnose patients for trial eligibility.


In conclusion, the main thing is being creative and persistent in how you reach this highly dispersed community. With some out-of-the-box thinking, we can overcome the enrolment challenges that come with rare disease trials.



To learn more on this topic, Mari Maurer will be presenting at this year's COG West Coast on 'Applying the Principals of Patient Finding to Non-Rare Disease Studies'. Speaker referral code: GG-MM3. Complimentary attendance is reserved for pharmaceutical, biotech, health and academic representatives.



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