With 350 million people affected worldwide, rare diseases represent a major unmet medical need.

However, the timely recruitment of eligible participants is a challenge for any rare disease clinical trial. Although many patients with rare diseases are desperate for a treatment, strong engagement is critical, as patients and their families have been perpetually desperate and get burned out over time.

It is important to effectively recruit participants through a partnership with patient organisations, patient contact registries, and clinician education to increase disease recognition and decrease time to diagnosis. 

ICON uses a variety of strategies to find patients such as partnering with rare disease patient groups and employing a digital engagement strategy, which allows recruitment for clinical trials at specific points in time when patients are searching for information online – such as at the point of diagnosis. Additionally, ICON works with ongoing registries to help identify patients for clinical trials and to locate historical control data.

Consistent trial participation is contingent upon the patient’s ability to get to and from site appointments. For success in a rare disease trial, the sponsor and other stakeholders must ensure there are no obstacles blocking patient compliance. It is imperative that contingencies to assist patients with transportation and accommodation costs be included in a company’s trial plan.

ICON provides trial participants with services to support every aspect of their lifestyle in relation to the trial including travel, lodging, and periodic home health nurse visits, and ensures that those arrangements are made so that patients don’t have to. It is important to give patients the resources they need to continue in the study.

ICON’s experienced team of clinical and therapeutic experts have conducted trials in over 60 disease indications across a range of therapeutic areas. To speak to one of our experts, please contact us


This blog is an edited version of “An overview on rare disease research” which appeared in the Journal for Clinical Studies on 18th June 2020. To view the full article, please visit https://www.jforcs.com/.

Rare and orphan diseases insights

ICON's Rare and Orphan Diseases team provides analysis including whitepapers, blogs and contributions to media and industry conversations relating to all aspects of rare and orphan diseases in clinical trials.

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