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Rare and orphan diseases

Unique challenges and complexities of rare and orphan disease clinical trials explored.

Read our whitepaper for insights on understanding the Bayesian and Frequentist approaches.

Leveraging historical data for use in rare disease trials

Rare disease clinical trials have several challenges that make them more difficult to conduct than clinical trials for more common diseases. Small patient populations, pediatric populations and the desire by patients and their caregivers to receive active therapy while on a clinical trial make it much more likely that these trials will need to incorporate historical data into the design.

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Patient centricity in orphan drug development

Patient centricity in orphan drug development

ICON analysed data on the use of patient-reported outcomes measures (PROMs) in orphan drug labeling claims and performed an extensive literature review on the use of PROMs in all phases of clinical research, observational/registry studies, and instrument development and validation. The findings and recommendations can be found in this whitepaper.

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Receive more insights on rare and orphan diseases from ICON

Please visit ICON's Preference Centre and select 'rare diseases' under 'Therapeutic Areas of Interest' to receive new insights on rare and orphans diseases.

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Rare and orphan diseases videos

Rare and orphan diseases services

ICON is an experienced partner who can assist sponsors in navigating the many challenges of orphan drug development. ICON can help sponsors optimise the whole continuum of the clinical trial lifecycle from patient enrolment to payer reimbursement with its powerful solutions and proven strategies. ICON’s highly experienced clinical and therapeutic teams have conducted numerous rare disease trials across a wide range of therapeutic areas.

Read more about ICON's services in rare diseases