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.
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?
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
Clinical trials support and advice
Scientific advice support
Medical writing
PIPs
Erik Hedner
Arex Advisor AB
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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.