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.

Over the past 5 years, ICON has conducted:

  • 271

    Rare disease studies
  • 41,203

    Patients involved
  • 13,558

    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.

Reimbursement of Orphan Drugs

With the average orphan drug today costing $137,782 per person/per year, what strategies can ensure reimbursement? ICON experts convened a roundtable to discuss reimbursement and market access issues in rare and orphan disease, specifically how best to identify, obtain and communicate evidence. 

Part 1: Identifying Evidence

Part 2: Obtaining Evidence

Part 3: Communicating Evidence - examines the importance of setting early expectations and strategies for successful evidence communications.