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's Rare Disease Experience in the Last Five Years
420Rare disease studies
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.
Whitepaper: Rare & Orphan Diseases Reimbursement Strategies Roundtable - Part 1: Identifying Evidence
Download our whitepaper to read our experts' perspectives on various challenges in identifying the proper evidence necessary to support reimbursement across geographies globally, including the role of patient-reported outcomes (PROs) and patient advocacy organisations.