The EU’s regulatory framework for orphan drugs is set out in Regulation (EC) No.141/2000 (the “Orphan Drug Regulation”, or ODR) and implementing Regulation (EC) No. 847/2000, and
has been in force for a while now with relatively few changes. However, in recent years, a number of issues which challenge the current system have emerged, and the European Commission has attempted to address some of these by amending its (non-legally binding) guidance. After consultation, a new “Commission Notice on the application of Article 3, 5 and 7 of [the ODR]” was published on 18 November 2016. This updates and replaces the 2003 Commission Communication on orphan drugs “in view of the experience acquired over the last 13 years”, introducing a number of changes:
- Clarification of the definition of “significant benefit”.
- Simplifying the procedure for reassessing orphan criteria when two authorisation procedures are pending in parallel for two orphan products.
- Introducing reassessment of the orphan criteria for a new subset of the condition when a sponsor extends the use of its product after marketing authorisation.
- Clarifications on processing the transfer of orphan designations between sponsors.
- Encouraging the development of orphans for communicable diseases e.g. Ebola by relaxing the Commission’s strict interpretation of the prevalence criterion.