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 ten years, there have been:

  • 252

    Rare disease studies
  • 36,000

    Patients involved
  • 9,000

    Sites globally

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.  

The 100,000 Genomes Project

ICON is proud to have been selected by Genomics England as its data management partner for the 100,000 Genomes Project, the largest coordinated genome study in human history. The project seeks to profile 100,000 genomes—50,000 each for rare disease and cancer—from 75,000 patients, with medical histories that total millions of data points.

By using ICON’s expertise to manage these immense datasets, the 100,000 Genomes Project is able to ensure accuracy and high-throughput access for all researchers involved while safeguarding patient data with the tightest cybersecurity measures.