Regulatory affairs

Orphan product support

Orphan drugs are a special category of medicines intended for use against very rare diseases. To qualify for this designation, the medicine must meet specific criteria.

Our experts help you make the right decisions for your orphan product, from development to commercialisation.

Questions to ask

By definition, a rare disease affects fewer than 5 in 10 000 people. The EU orphan designation program is there to encourage companies to work with rare diseases.

Still, out of 2840 medicines that meet the criteria only about 240 have reached the market. 

There are several scientific and clinical questions, as well as strategical matters, to consider. For example:

Is there a robust scientific rationale behind the drug?

Are you fully informed about the regional requirements for orphan drug designation in your target market?

Is there a comprehensive development plan in place?

What does the timeline for submission and approval look like?

Why use our support with orphan product support?

Our experts have extensive experience in regulatory affairs for orphan drugs, from development to commercialisation. We support sponsors throughout the product lifecycle, with services such as:
– Clinical regulatory affairs for orphan drugs
– Orphan Drug Designation (ODD) support
– Regulatory strategy for orphan drugs
– Regulatory affairs for submission
– Regulatory operations for orphan drugs

More clinical regulatory affairs services

Clinical trials support and advice
Scientific advice support
Medical writing

Staffan Thunell

Founding partner

BSc Economics and BA

Staffan has a long background in entrepreneurship within the life science industry. He has 20+ years experience from posit­ions as Founder, Chair­man, CEO and CFO within medical affairs consulting and small pharma. Previously Staffan worked in executive positions in big pharma and specialty pharma companies.