Rare Diseases

GCR is committed to advancing the development of medicines for adults and children with rare diseases and we have developed solutions to reach, recruit and retain small, globally dispersed patient populations.

By leveraging our medical, operational, regulatory and real-world expertise, we design and operationalise studies with the customised approach that each rare disease requires.

We successfully execute trials with organisations of all sizes. In the past five years, we have conducted more rare disease studies than any other contract research organisation (CRO).

rare diseases

Rare Disease Framework

We deploy a four-part framework to support and conduct rare disease studies that ensures the application of best practices in study development. The key elements of this framework include:

  • Innovative and executable development plans– Deploying innovative, patient-centric development plans to navigate challenges and support clients in the development of clinical programs
  • Rigorous feasibility and study planning – Identifying the right sites and finding and enrolling patients crucial to a study’s success
  • Study execution – Providing unmatched patient focus to retain and consistently engage patients and sites
  • Proactive post-study planning – Leveraging key partnerships to plan for future development

Patient Advocacy Groups

GCR’s rare disease team cares deeply about the rare disease patients in our clinical trials and strives to better understand their experiences.

By collaborating with patient advocacy groups, we are able to incorporate patients’ voices into our study designs and gain valuable insights about the patient journey as we strive to enhance the clinical trial experience for patients and their caregivers.