Rare Disease Day: What to know about Orphan Drug Designations

February 28th is Rare Disease Day worldwide.

Many pharmaceutical companies develop drugs for the rare disease and orphan drug market. It is important to define what exactly this therapeutic area is because the definition differs based on region.

orphan drug designation rare disease day

In Europe, a disease or disorder is considered rare if it affects fewer than 1 in 2000 people. One in 17 people in the European Union has a rare disease.
Meanwhile in the United States, it is defined as rare if it affects fewer than 20,000 people.

There are over 6,000 rare diseases in this classification, and it is estimated that in Europe as many as 30 million people are affected by a rare disease. According to RareDiseaseDay.org, 80% of rare diseases have identified genetic origins, and 50% of rare diseases affect children.

Orphan Drugs is an important area of clinical research. In the EU alone, the EMA estimates that there are more than 1,900 medicines with orphan drug designation. Orphan Drug Designations are an incentive to encourage companies to research and develop medicines for rare diseases. Companies in the EU with this designation get access to scientific advice from the EMA for study protocols and may have access to fee reductions. Once an orphan drug makes it to market approval, they are granted 10 years of market protection.

The FDA also has an Orphan Drug Designation status where companies can qualify for tax credits for clinical testing. According to the FDA and the Orphan Drug Act, an Orphan Drug Modernization Plan was unveiled in 2017 in order to speed up responses to requests for Orphan Drug Designation.

CROS NT supports companies looking to gain Orphan Drug Designation with regulatory consultancy and support in both the EMA and FDA. Our biometrics team also has extensive experience providing study design, methodology and reporting services for orphan drug studies.

See our recent blog on removing complexities from orphan drug designation requests.

Have an upcoming study in your pipeline with Orphan Drug Designation status or looking for support? Contact us via the RFI form on our webpage.