This page summarises the legal background to the procedure for orphan designation in the European Union (EU). It includes the key milestones in EU legislation adopted since the Orphan Regulation was first adopted in 1999.
Regulation (EC) No 141/2000 (the Orphan Regulation)
On 16 December 1999, the European Parliament adopted Regulation (EC) No 141/2000 (the Orphan Regulation). This was published in the Official Journal of the European Communities on 22 January 2000.
- lays down the EU procedure for designation of orphan medicines;
- defines incentives for the development and placing onto the market of designated orphan medicines;
- establishes the Committee for Orphan Medicinal Products (COMP).
Regulation (EC) No 847/2000
On 27 April 2000, the European Commission adopted Regulation (EC) No 847/2000, which:
- lays down implementing rules;
- sets out definitions essential for the application of the Orphan Regulation.
This Regulation entered into force on 28 April 2000. On this date, sponsors could begin to submit applications for orphan designation to the European Medicines Agency.
Regulation (EC) No 726/2004
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;
- the CHMP can issue guidance regarding compassionate-use programmes.
Regulation (EC) No 507/2006
On 29 March 2006, the European Parliament adopted Regulation (EC) No 507/2006, which provides the legal framework for the granting of a conditional marketing authorisation to medicines that fall within the scope of Regulation (EC) No 726/2004. It establishes that orphan medicines can be granted a conditional marketing authorisation within this legal framework.
Regulation (EC) No 1901/2006
On 12 December 2006, the European Parliament adopted Regulation (EC) No 1901/2006 on medicinal products for paediatric use. It establishes that the usual period of market exclusivity for orphan medicines may be extended to twelve years if study results are submitted in compliance with an agreed paediatric investigation plan at the time of marketing authorisation.
Regulation (EC) No 2049/2005
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.
Commission notice 2016/C 424/03
On 18 November 2016, the European Commission adopted Commission notice on the application of Articles 3, 5 and 7 of Regulation (EC) No 141/2000 on orphan medicinal products (2016/C 424/03), which is intended to facilitate the application of Articles 3 (criteria for designation), 5 (procedure for designation and removal from the register) and 7 (Union marketing authorisation) of Regulation (EC) No 141/2000. The notice replaces the Communication 2003/C 178/02 of 29 June 2003.
European Commission guidelines
The European Commission has also issued guidelines on aspects of the application of the Orphan Regulation:
- Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity;
- Guideline on aspects of the application of Article 8(2) of Regulation (EC) No 141/2000 of the European Parliament and of the Council: Review of the period of market exclusivity of orphan medicinal products.
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