Europe – Procedure for orphan medicinal product designation

Guidance for sponsors submitting an application via IRIS secure online portal

1. Legislative background

The legislation on orphan medicinal products, Regulation (EC) No 141/2000 of the European Parliament and of the Council, was adopted on 16 December 1999 and published in the Official Journal of the European Communities on 22 January 2000 (Ref. L18/1). This Regulation lays down a Community procedure for the designation of medicinal products as orphan medicinal products and provides incentives for the development and placing on the market of designated orphan medicinal products. The Regulation also establishes the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA), which is responsible for examining applications for orphanmedicinal product designation.

On 27 April 2000, the Commission adopted Regulation (EC) No 847/2000 laying down implementing rules and setting out definitions essential for the application of Regulation (EC) No 141/2000 (Ref. L103/5). As of 28 April 2000, the date this Regulation entered into force, sponsors have been able to submit applications for orphan medicinal product designation to the EMA.

On 31 March 2004, the European Parliament adopted Regulation (EC) No 726/2004, which provides the legal framework for the centralised authorisation and supervision of medicines for human and veterinary use and establishes the European Medicines Agency (EMA). It determines that all marketing authorisations for orphan medicines in the EU should follow the centralised authorisation procedure and that the CHMP can issue guidance regarding compassionate-use programmes.

On 15 December 2005, the European Commission adopted Regulation (EC) No 2049/2005 regarding the payment of fees to, and receipt of assistance from, the EMA by micro, small and medium-sized enterprises (SMEs). It determines that scientific advice and scientific services for designated orphan medicines shall be provided by the EMA to SMEs free of charge…