Confidence Beyond Compliance

Orphan Medicines are intended to treat rare diseases. Both, the EU and US drug legislation have financial and regulatory incentive systems in place to encourage developers engaging in these neglected therapeutic areas. Nevertheless, often development of a new orphan medicine follows alternative development and regulatory pathways which require specific scientific and regulatory expertise.

Our experts provide hands-on regulatory support including development consultancy, support in health authority interactions, labeling, (electronic) dossier compilation & submission, procedure management (pre- and post-approval as well as marketing authorization applications).

  • Strategic advice for defining the orphan condition
  • Orphan designation applications to the FDA and EMA including FDA rare pediatric disease designation (and maintenance of designations)
  • Set up of integrated development plans
  • Development of clinical trial designs
  • Preparation and management of clinical trial applications (e.g. IND / IMPD)
  • Health agency interactions (e.g. EMA protocol assistance, FDA meetings, joint EMA / FDA advice)
  • Pediatric investigational plan / waiver applications to the EMA / PDCO
  • Pediatric study plan / waiver applications to the FDA
  • Strategic consultancy and hands-on support for EU and US expedited program eligibility applications (e.g. PRIME, Accelerated Assessment, Breakthrough Designation, Priority Review)