Case T‑733/17
(Publication by extracts)
GMP-Orphan (GMPO)
v
European Commission
Judgment of the General Court (Seventh Chamber), 16 May 2019
(Medicinal products for human use – Article 3(1)(b) of Regulation (EC) No 141/2000 – Definition of ‘significant benefit’ – Availability of orphan medicinal products – Article 5(12)(b) of Regulation No 141/2000 – Commission decision to remove a medicinal product from the Register of Orphan Medicinal Products – Error of assessment – Error of law – Legitimate expectations)
1.      Approximation of laws – Uniform legislation – Medicinal product for human use – Orphan medicinal products – Procedure for designating a medicinal product as an orphan medicinal product – Significant benefit – Criteria for assessment – Burden of proof – Major contribution to patient care – Availability of orphan medicinal products – Benefit of marketing authorisation at EU level as compared to a medicinal product that is authorised in only one Member State – Possibility of a significant benefit which must be substantiated by concrete evidence
(European Parliament and Council Regulation No 141/2000, Art.3(1)(b); Commission Regulation No 847/2000, Art. 3(2))
(see paragraphs 30-32, 37-43, 45, 46, 53, 60, 76)
2.      Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Review procedure for designating a medicinal product as an orphan medicinal product – Opinion of the Committee for Orphan Medicinal Products – Discretion of the Commission – Judicial review – Limits – Taking into account of all the considerations set out in the opinion
(European Parliament and Council Regulation No 141/2000, Art. 5(12)(b))
(see paragraphs 33, 34)
3.      Approximation of laws – Medicinal products for human use – Directive 2001/83 – Importation into a Member State of a product constituting a medicinal product – Need to obtain marketing authorisation – Exception in cases of special needs – Scope
(European Parliament and Council Directive 2001/83, Art. 5(1))
(see paragraphs 47-51)
4.      Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Procedure for designating a medicinal product as an orphan medicinal product – Commission decision refusing the designation of a medicinal product as an orphan medicinal product – Judicial review – Limits
(European Parliament and Council Regulation No 141/2000, Art. 5)
(see paragraphs 56, 57)
5.      Approximation of laws – Uniform legislation – Medicinal products for human use – Orphan medicinal products – Review procedure for designating a medicinal product as an orphan medicinal product – Significant benefit – Criteria for assessment – Determination by the Committee for Orphan Medicinal Products – Higher standard of proof and information required than at the time of the initial designation
(European Parliament and Council Regulation No 141/2000, Arts 3(1)(b) and 5(12)(b))
(see paragraphs 88-90)
6.      EU law – Principles – Protection of legitimate expectations – Conditions – Specific assurances given by the authorities
(see paragraphs 91-95)
Résumé
In the judgment in GMPO v Commission (T‑733/17), delivered on 16 May 2019, the Court dismissed in its entirety the action for partial annulment of the Commission’s decision to withdraw a medicinal product based on the active substance trientine, the sponsor of which is GMP-Orphan (GMPO), the applicant, from the European Union’s Register of Orphan Medicinal Products on the grounds that it did not satisfy the criteria laid down in Regulation No 141/2000, (1) namely to provide a ‘significant benefit’ to patients with a rare disease as compared to another similar medicinal product which has already been authorised. The orphan medicinal product status would have allowed the applicant to benefit from a period of market exclusivity of 10 years from the date on which the marketing authorisation was obtained for it.
The applicant’s main argument was that its medicinal product, for which a marketing authorisation procedure was in progress under the centralised procedure introduced by Regulation No 726/2004, (2) would have provided, by means of that authorisation, ipso jure, a ‘significant benefit’ for patients within the meaning of Regulation No 141/2000 as compared with another similar medicinal product already authorised but only on the market of one Member State.
The Court held that no provision either in Regulation No 141/2000 or Regulation No 847/2000 (3) provides that marketing authorisation at EU level for an orphan medicinal product constitutes per se a significant benefit in comparison with an existing medicinal product, which is as effective and already authorised, albeit in only one Member State. Moreover, according to the Court, a sponsor cannot rely for those purposes on presumptions or assertions of a general nature, such as, for example, an alleged lack of availability of treatments which already exist and which are already authorised in the Member States. However, a sponsor must, on the basis of concrete and substantiated evidence and information, demonstrate that the new medicinal product provides a benefit to patients and that it contributes to their care. In that regard, the Court specified that the expected advantage of that new medicinal product over the existing medicinal product must exceed a certain quantitative or qualitative threshold in order that it may be considered ‘significant’ or ‘major’ within the meaning of the relevant legal framework.
In the present case, the Committee for Orphan Medicinal Products of the European Medicines Agency had considered in its opinion, on the basis of which the Commission adopted the contested decision, that the applicant had not submitted sufficient supporting information to establish that there was a problem with the availability of the existing medicinal product. In that regard, while noting that the opinion of that committee does not undertake complex technical or scientific assessments, but is essentially based on findings of fact concerning the availability within the European Union of the reference medicinal product, the Court carried out a full judicial review of the legality of that opinion. It observed, first, that the investigation carried out by the Committee in order to verify the assumptions relied on by the applicant before the Committee has a high probative value and, secondly, that the evidence adduced by the applicant before that committee was properly assessed. Thus, the Court concluded that the contested decision, which endorses the opinion of the Committee for Orphan Medicinal Products, is not vitiated by an error of assessment.
1      Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ 2000 L 18, p. 1).
2      Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (OJ 2004 L 136, p. 1).
3      Commission Regulation (EC) No 847/2000 of 27 April 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’ (OJ 2000 L 103, p. 5).