Orphan Drugs: Advocating for the Underserved

Bringing Rare Disease Treatments to Light

The Orphan Drug Designation (ODD), a legislative and regulatory framework, provides a number of incentives supporting companies developing drugs for rare diseases. This framework has been adopted by a number of countries and regions, including the EU, US, and Japan. Although there are similarities among these regions, criteria and procedures vary.

We can support you with:

Application for ODD in the EU, US, and Japan

• Strategic advice
• Scientific documentation
• Procedural steps
• Pre-submission meetings (where applicable)
• Follow up as needed

Activities after ODD

• Annual reports
• Scientific advice (protocol assistance)
• Best use of incentives

Have a question concerning Orphan Drugs? contact Dr. Christiane Chène.