Orphan Drug Designation

350 million people worldwide are living with a rare disease, but only 5% of known rare diseases have an approved treatment.¹

We can help you change that.

Accelerate your orphan drug designation application with our specialty understanding of rare diseases, clinical development and complex regulatory pathways. Position your orphan drug for exclusive clinical trial benefits sooner.

We have a proven record to help you succeed. IQVIA is the largest submitter of FDA and EMA orphan designations, and has helped

Develop or commercialize 85% of all approved orphan drugs.²

We have experience with breakthrough therapy and regenerative medicine advanced therapy designations as well as priority review vouchers. 

Let us help you achieve orphan drug designation fast and efficiently – and make rare disease cures more common.

_{1. http://phrma-docs.phrma.org/sites/default/files/pdf/medicines-in-development-report-rare-diseases.pdf
2.FDA-approved orphan drug in 2015}