Keywords

Summary



Keywords

1. Approximation of laws – Uniform legislation – Medicinal product for human use – Orphan medicinal products

(European Parliament and Council Regulations No 141/2000, Art. 5(1), and No 726/2004)

2. Approximation of laws – Uniform legislation – Medicinal product for human use – Orphan medicinal products

(European Parliament and Council Regulation No 141/2000, Art. 3(1)(b))

3. Approximation of laws – Uniform legislation – Medicinal product for human use – Orphan medicinal products

(European Parliament and Council Regulations No 141/2000, Arts 3(1)(b), 5(1) and 8(1) and No 726/2004, Art. 3(2)(b))

Summary

1. Article 5(1) of Regulation No 141/2000 on orphan medicinal products states clearly and unequivocally that applications for designation of a medicinal product as an orphan medicinal product can be submitted at any stage of the development of the medicinal product before the submission of the application for marketing authorisation. That provision does not make a distinction according to whether the application for marketing authorisation is made using the mutual recognition procedure at Member State level or using the centralised European Union procedure, nor has it been amended following the entry into force of Regulation No 726/2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency in order to indicate that the only possible authorisation procedure for orphan medicinal products is the centralised procedure.

Regulation No 726/2004 in no way amended Article 5(1) of Regulation No 141/2000. That confirms that the essential question for the legislature is whether the medicinal product concerned represents a development in comparison with the treatment for which an application for marketing authorisation has already been made or which has already been authorised. It matters little, therefore, whether the application for marketing authorisation of the medicinal product concerned was made, or a fortiori obtained, at Member State level or at European Union level.

(see paras 44, 60)

2. Article 3(1)(b) of Regulation No 141/2000 on orphan medicinal products provides that a medicinal product is to be designated as an orphan medicinal product if its sponsor can establish that no other satisfactory method of diagnosis, prevention or treatment of the condition in question has been authorised in the European Union or that, if such a method exists, the medicinal product for which designation is sought ‘will be’ of significant benefit to those suffering from that condition. In that regard, as EMA rightly points out, the fact that the verb ‘will be’ is in the simple future tense shows that that provision indisputably refers to a future benefit.

That provision thus tends to confirm that the application of the incentives laid down by that regulation is not justified for a medicinal product which has already been authorised in the European Union. The same is true for any other medicinal product intended to treat the same condition, unless that medicinal product will have a significant benefit by comparison with the medicinal product already authorised.

(see para. 46)

3. Article 5(1) of Regulation No 141/2000 on orphan medicinal products does not infringe property rights or impair the freedom to pursue professional activities.

Where an undertaking already holds a number of national marketing authorisations for a medicinal product, without, however, holding a designation as an orphan medicinal product for that medicinal product, and where a competing undertaking which wishes to obtain the designation as orphan medicinal product of a competing medicinal product to treat the same condition as that treated by human fibrinogen must demonstrate, in accordance with Article 3(1)(b) of Regulation No 141/2000, that that medicinal product will be of significant benefit to patients suffering from the condition in question. In that regard, the criteria for a finding of a significant benefit are strict and the development of a medicinal product which is of significant benefit in comparison with the medicinal product already authorised to treat the same condition involves, for the undertaking working on it, investment in research and development of this potentially improved medicinal product. A competing undertaking cannot thus merely develop a similar medicinal product in order to obtain its designation as an orphan medicinal product.

In addition, so long as competing undertakings have not developed a medicinal product which will be of significant benefit in comparison with the medicinal product already authorised and obtained its designation as an orphan medicinal product and marketing authorisation, the holder of the authorised medicinal product retains its right to use the procedure for mutual recognition for that medicinal product for which it can be the holder of a number of marketing authorisations. It is entitled to submit an application for mutual recognition of one of those authorisations in one or more other Member States of the European Union, in accordance with the procedure laid down in Chapter 4 of Directive 2001/83 on the Community code relating to medicinal products for human use.

Furthermore, should a competing undertaking obtain designation as an orphan medicinal product for a medicinal product similar to the medicinal product for which there is a marketing authorisation, but having a significant benefit, it still has to obtain marketing authorisation for that medicinal product. The medicinal product must be evaluated by the Committee for Medicinal Products for Human Use, which is independent of the Committee on Orphan Medicinal Products. Only if, following that committee’s opinion, the Commission issues the marketing authorisation can the competing undertaking benefit from market exclusivity pursuant to Article 8(1) of Regulation No 141/2000. Nevertheless, even if the competing undertaking were to obtain market exclusivity for its medicinal product, that does not cause the loss of existing marketing authorisations for medicinal products to treat the same condition.

Moreover, obtaining designation as an orphan medicinal product in no way constitutes the only possibility of access to the marketing authorisation procedure at European Union level. Article 3(2)(b) of Regulation No 726/2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency provides that any medicinal product not appearing in the annex thereto may be granted a marketing authorisation by the European Union in accordance with the provisions of that regulation, if the applicant shows that the medicinal product constitutes a significant therapeutic, scientific or technical innovation or that the granting of authorisation in accordance with that regulation is in the interests of patients at European Union level.

(see paras 91, 93-96, 98, 102, 104)