On 23 October 2006, the Council of the European Union approved all the compromise amendments to the proposal for a Regulation on Medicinal Products for Paediatric Use which were agreed at its second Parliamentary reading. The Regulation was officially published on 27 December 2006 and will enter into force on 26 January 2007.
The Regulation is part of the European Commission's (EC) objective of improving the health of children in Europe by increasing the number of medicines that are developed and authorized for their treatment. It has been estimated that the paediatric population of Europe is around 100 million, and that over half of the medicines used to treat this population have not been authorized for use in children. It is felt that market forces have not been an adequate driver to encourage the pharmaceutical industry to incur the extra cost required to adapt adult medicines for use in the various paediatric sub-populations (for example, newborn babies, toddlers, children and adolescents). As a result, prescribing doctors have no alternative but to use the adult product 'off-label,' which has an associated risk of inefficacy or adverse reactions. Although the objective is to increase the development of medicines for children, this must be achieved without subjecting children to unnecessary clinical trials and without unnecessarily delaying the introduction of medicinal products for use in adults.
Key measures in the Regulation
• An expert paediatric committee will be set up with responsibility for agreeing and assessing paediatric investigation plans to collect data on the use of medicinal products in the paediatric population.
• All applicants for marketing authorizations for new medicinal products and line-extension products (such as new pharmaceutical forms or new routes of administration) will have to submit data on the use of the product in children in compliance with a paediatric investigation plan agreed with the paediatric committee.
• A system of waivers of the above requirement will exist for medicinal products unlikely to benefit children and deferrals will be available where it would not yet be safe to conduct clinical trials in children or to ensure there is no delay in the authorization of the product in adults.
• Marketing authorization applicants who comply fully with the paediatric study requirements and whose product is subsequently authorized in all member states, will be rewarded with the right to claim a six-month patent or supplementary protection certificate (SPC) extension (providing that the product is covered by a patent or SPC). This extension will be granted simply for conducting the relevant paediatric studies, even if the product is not actually authorized for such use. For successful products, this extension will result in increased profits for the applicant, hopefully outweighing the extra cost associated with obtaining the paediatric data. However, where the application for a new paediatric indication results in a one year extension of the period of marketing exclusivity under Regulation 726/2004 the SPC extension will not be available.
• All holders of marketing authorizations will be obliged to disclose the results of paediatric studies already conducted prior to the proposed regulation coming into force. Although these studies will be taken into account in order to update product information, their submission will not qualify the applicant for incentives such as SPC extension. The aim of this obligation is to improve the dissemination of information about paediatric uses of medicinal products.
• For orphan medicinal products, the above SPC extension will not be available. The reason is that many such products are, in any event, not covered by patents or SPCs. It was also considered that an unfair double reward would result for patent or SPC-protected orphan products that were granted a six-month SPC extension, as well as the ten-year period of market exclusivity already available as a result of their orphan status. Instead, for orphan medicinal products that comply with the paediatric study requirements (irrespective of whether a paediatric authorization is actually granted), the period of market exclusivity will be extended from ten to 12 years.
• Products already marketed for other indications and which are granted a paediatric marketing authorization as a result of completing a paediatric investigation plan must be put on the market within two years of paediatric approval. A mechanism is also proposed to ensure that paediatric products will remain on the market if the marketing authorization holder discontinues marketing them. These measures should ensure that access to paediatric medicinal products is increased and maintained.
• A new type of marketing authorization, the paediatric use marketing authorization (PUMA), will be available for medicinal products that are already off-patent (and off-SPC). PUMAs will be available only where such products developed exclusively for use in the paediatric population. The necessary clinical data may be derived from published literature or from new studies. The reward for obtaining a PUMA will be ten year period of data and marketing exclusivity and it is hoped that this will stimulate research into paediatric use of many off-patent adult medicinal products.
A number of implementation tasks need to carried out in 2007 to put the Regulation fully into effect and they can be found at the EMEA website: http://www.emea.eu.int/pdfs/human/peg/EC_EMEA_Paediatric%20Regulation.pdf
In addition, the new Regulation introduces some amendments to the SPC Regulation,* the Directive on the Community code relating to medicinal products for human use, the regulation laying down procedures for the authorization and supervision of medicinal products† and the EU clinical trials Directive.‡
The Regulation will significantly affect the regulatory burden of pharmaceutical companies, but the rewards are anticipated to provide a sufficient benefit to make compliance cost-effective.
References
* Regulation (EEC) No 1768/92 (OJ L 182, 2.7.1992, p. 1), as last amended by the Act of Accession 2003.
† Regulation (EC) No 726/2004 (OJ L 136, 30.4.2004, p. 1).
‡ Directive 2001/20/EC (OJ L 121, 1.5.2001, p. 34).
About the Authors
Partners Gerry Kamstra and Paule Drouault-Gardrat co-head Bird & Bird's International Life Sciences Group. Gerry is a UK specialist in IP, commercial and regulatory issues affecting the life sciences sector. Paule is a French specialist in regulatory, IP and product liability issues in this field.
Published Jan 19, 2007 in Pharmaceutical Executive Europe
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