Assistance with orphan drug development

A general understanding of an orphan disease is that it describes diseases neglected by the public – orphaned from the medicinal community. Frequently the term “orphan disease” is used synonymously with rare disease. Rarity is in fact a key feature of these conditions but the definition according to the European legislation is stricter.

To stimulate drug development for such diseases with small markets particular legislation has been introduced in the US, EU as well as several other countries that offer benefits for companies working in this field. The initial mandatory step is the formal orphan designation that qualifies the product for incentives such as free scientific advice at the EMA. Upon licensure market exclusivity is granted for orphan products between 7-10 years in the US and EU respectively.

Granzer Regulatory Consulting & Services offers full support during all stages of orphan drug development – from the initial orphan designation to final marketing authorisation. Our experience covers more than 70 orphan drugs in the EU and/or US.