ICON can help you navigate the clinical development challenges in rare and orphan diseases

Rare diseases present unique challenges when conducting clinical trials. One of the biggest challenges in disease areas characterised by small patient populations and a smaller number of specialised sites is patient recruitment and retention.

In addition, reimbursement can also pose challenges to drug developers and smart strategies are required to gain sufficient market access.

ICON Rare Disease experience in the last five years

  • 375

    Studies conducted
  • 62,824

    Patients involved
  • 17,080

    Sites worldwide

Our experienced team of clinical and therapeutic experts have conducted trials in over 60 disease indications across a range of therapeutic areas.

ICON’s experts can advise on study design, targeted patient enrolment, data management and analysis, visualisation of large real-world datasets, complex regulatory issues, and pricing, market access and reimbursement challenges.

Incorporating the patient voice into rare disease research

The healthcare industry and continues to strengthen efforts to incorporate the patient viewpoint into rare disease research. To what extent have sponsors been successful in developing and marketing medications that address the needs that are most important to rare disease patients and their families? Our analysis indicates that rare disease research needs innovative solutions.

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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