Keywords

Summary



Keywords

1. Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Procedure for designating a medicinal product as an orphan medicinal product

(European Parliament and Council Regulation No 141/2000, Art. 3(1); Commission Regulation No 847/2000, Art. 3(2))

2. Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Procedure for designating a medicinal product as an orphan medicinal product

(European Parliament and Council Regulation No 141/2000, Art. 4)

3. Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Procedure for designating a medicinal product as an orphan medicinal product

(European Parliament and Council Regulation No 141/2000, Art. 5)

Summary

1. Pursuant to the first subparagraph of Article 3(1)(a) and Article 3(1)(b) of Regulation No 141/2000 on orphan medicinal products, the sponsor of a medicinal product for which designation as an orphan product is sought is required to establish that the product concerned is intended for the diagnosis, prevention or treatment of a rare condition and that there does not yet exist a satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised. However, the sponsor of a potential medicinal product intended to treat a rare condition for which such a satisfactory method of diagnosis, prevention or treatment already exists must not only establish, under the first subparagraph of Article 3(1)(a), that the medicinal product in question is actually intended for the diagnosis, prevention or treatment of the rare condition, but also, under Article 3(1)(b), that the potential medicinal product will be of significant benefit to patients affected by that condition.

Establishing significant benefit therefore takes place in the context of a comparison with an existing authorised medicinal product or method. The clinically relevant advantage and the major contribution to patient care, which, according to the definition of significant benefit mentioned in Article 3(2) of Regulation No 847/2000 laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts ‘similar medicinal product’ and ‘clinical superiority’, enable the potential orphan medicinal product to be described as being of significant benefit, can be established only by comparison with treatments that have already been authorised.

As there may be little or no clinical experience with the potential medicinal product for which designation as an orphan medicinal product is sought, the justification for significant benefit is likely to be made on assumptions of benefit by the sponsor, which must be supported by available data and/or evidence supplied by the latter. Only the European Union provisions laying down criteria for the designation of orphan medicinal products are relevant, so the fact that a medicinal product meets the criteria for designation as an orphan medicinal product in other countries is irrelevant in that regard.

(see paras 41-43, 49, 57)

2. The procedure for designating orphan medicinal products provided for by Regulation No 141/2000 on orphan medicinal products is an administrative procedure involving complex scientific assessments in respect of which the Commission has broad discretion. Hence, it is all the more necessary to observe the guarantees conferred by the Community legal order in administrative procedures, including the duty to examine carefully and impartially all the relevant aspects of the individual case. Such an obligation cannot be validly complied with if the opinion of the Committee for Orphan Medicinal Products which the Commission takes as its basis has been given by experts who are partial. The requirement of impartiality to which the Community institutions are subject also extends to experts consulted in that regard. In particular, where an expert is requested to give an opinion on the effects of a potential medicinal product, it is necessary for that expert to perform his task impartially. However, the obligation of impartiality cannot be construed as meaning that there is a legal impediment to an expert being consulted in a procedure for the designation of a medicinal product as an orphan medicinal product solely because he has already given an opinion on the same product in another, national, procedure carried out in a Member State of the Union.

(see paras 77, 88, 93)

3. A feature of the procedure introduced by Article 5 of Regulation No 141/2000 on orphan medicinal products is the essential part assigned to an objective and in-depth scientific assessment by the Committee for Orphan Medicinal Products of the effect of the potential medicinal products considered. Since the Commission is not in a position to carry out scientific assessments of the efficacy and/or harmfulness of a medicinal product during the orphan medicinal product designation procedure, the aim of the mandatory consultation of the Committee is to provide the Commission with the evidence of scientific assessment which is essential for it to be able to determine, in full knowledge of the facts, the appropriate measures to ensure a high level of public health protection. Thus, although the Committee’s opinion does not bind the Commission, it is none the less extremely important. In that regard, it is apparent from Article 5(8) of Regulation No 141/2000 that a case in which a decision is not in accordance with the Committee’s opinion was envisaged as constituting an exceptional situation.

In that context, as part of its judicial review of the legality of a Commission decision refusing to designate a medicinal product as an orphan medicinal product, the Community judicature must determine whether the relevant procedural rules have been complied with, whether the facts established by the Commission are correct and whether there has been a manifest error of appraisal of those facts or a misuse of powers.

(see paras 111-112)