Keywords

Summary



Keywords

1. Approximation of laws — Medicinal products for human use — Directive 2001/83 — Authorisation to market — Orphan medicinal products — Well-established medicinal use — Systematic and documented use of the substance concerned as a hospital preparation excluded from the scope of the directive — Lawfulness — Conditions — Substance meeting special needs and supplied in response to a bona fide unsolicited order — Hospital preparations of cholic acid — Included

(European Parliament and Council Directive 2001/83, as amended by Directive 2004/27, Arts 5(1), and 10a)

2. Approximation of laws — Medicinal products for human use — Directive 2001/83 — Scope — Hospital preparations — Included

(European Parliament and Council Directive 2001/83, Art. 5(1))

3. Approximation of laws — Medicinal products for human use — Directive 2001/83 — Medicinal products covered by Article 5 — No declaration and control obligation

(European Parliament and Council Directive 2001/83, Art. 5(1))

4. Approximation of laws — Medicinal products for human use — Directive 2001/83– Authorisation to market — Orphan medicinal products — Well-established medicinal use — Exceptional circumstances — Simultaneous application — Lawfulness

(European Parliament and Council Regulation No 726/2004, Art. 14(8); European Parliament and Council Directive 2001/83, Art. 22)

Summary

1. The power of a Member State under Article 5(1) of Directive 2001/83 on the Community code relating to medicinal products for human use, in order to fulfil special needs, to exclude from the application of the provisions thereof medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised healthcare professional and for use by one or more individual patients under his direct personal responsibility may be exercised only in case of need, taking account of the specific needs of patients. In that regard, the concept of ‘special needs’, referred to in Article 5(1) of that directive, applies only to individual situations justified by medical considerations and presupposes that the medicinal product is necessary to meet the needs of patients. Also, the requirement that medicinal products be supplied in response to a ‘bona fide unsolicited order’ means that the medicinal product must have been prescribed by the doctor as a result of an actual examination of his patients and on the basis of solely therapeutic considerations.

Cholic acid-based hospital preparations which, under the legislation of a Member State, are medicinal products prepared in accordance with the prescriptions of a pharmacopoeia and in compliance with the rules of good practice laid down in that legislation where there is no appropriate medicinal product prepared by a hospital’s in house pharmacy or by that hospital’s authorised pharmaceutical establishment in accordance with that legislation fulfil the requirements of those concepts of ‘special needs’ and ‘bona fide unsolicited order’ and thus comply with the conditions laid down by Article 5(1) of that directive where those hospital preparations are provided on medical prescription to one or more patients by the hospital’s in-house pharmacy and the Member State concerned has excluded them from the scope of EU legislation on medicinal products for human use since the start of the use of that medicinal product.

In those circumstances, those hospital preparations of cholic acid are, in the first place, intended to fulfil special needs, in that they respond to individual situations justified by medical considerations and are necessary to meet patients’ needs taking account of the fact that there is no medicinal product on the market capable of treating the liver disorders in question, which are likely to lead rapidly to the death of the person concerned any person who is diagnosed with those disorders, and, moreover, are prescribed by a doctor as a result of an actual examination of his patients and on the basis of solely therapeutic considerations.

(see paras 37-39, 41, 43-46)

2. See the text of the decision.

(see para. 48)

3. See the text of the decision.

(see para. 49)

4. A marketing authorisation for a medicinal product for human use may be granted in exceptional circumstances pursuant to Article 14(8) 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 and Article 22 of Directive 2001/83 on the Community code relating to medicinal products for human use only if (i) the applicant can show that, for objective, verifiable reasons, he is unable to provide comprehensive data regarding the efficacy and safety, under normal conditions of use, of the medicinal product and (ii) it is based on one of the grounds set out in Annex I to that directive. Moreover, Part II-1(c) of Annex I to Directive 2001/83 provides for the possibility of a marketing authorisation being granted even where information is missing, as long as the demonstration of an acceptable level of safety and/or efficacy can be supported although some studies are lacking. Finally, nothing in Regulation No 726/2004 or Directive 2001/83 precludes the simultaneous application of the concepts of ‘well-established medicinal use’ and ‘exceptional circumstances’. On the contrary, it should be noted that, in the specific context of orphan medicinal products, Directive 2001/83 expressly refers to the possibility of applying both the provisions relating to exceptional circumstances and those provisions relating to a claim of well-established medicinal use, under the first and second indents of Part III-5 of Annex I.

(see paras 58, 64, 65, 67-69)