Medicines for children

Summary

On 29 September 2004, the Commission adopted a proposal for a regulation on medicinal products intended for paediatric use. A Commission background document explains that the aim of the paediatrics proposal is to "improve the health of the children of Europe by stimulating the research, development and authorisation of medicines to treat children". 

About 100 million babies, toddlers, children and adolescents make up the ‘paediatric population’ of the EU-25, accounting for over 20 per cent of the EU’s total population. Yet the European Commission argues that more than half of the medicines used to treat children in the EU have neither been specifically tested, nor authorised for use in children. 

In a resolution adopted in December 2000, the Council called on the Commission to make proposals to ensure that new and currently used medicinal products fully cater for the particular needs of children. Since then, the Commission has conducted two rounds of public consultation, the results of which have been fed into the draft legislation. 

The Commission looked at legislation in the US, which has succeeded in stimulating the development of medicinal products for paediatric use thanks to a combination of measures of incentives and obligations. 

The new regulation will have links with existing EU legislation, such as the ‘Clinical Trials Directive’ (2001/20/EC), which establishes requirements to protect children who take part in clinical trials. Some elements of the ‘Orphan Regulation’ ((EC) 141/2000), which has successfully stimulated research and authorisations of medicines to treat rare diseases, have served as a basis for provisions in the paediatrics proposal. 

Issues

The Commission has concluded that market forces alone do not sufficiently stimulate the development of medicines that take into account the specific needs of children. Therefore, it has opted for legislative means, comprising obligations, rewards and incentives to achieve this objective. 

Based on the proposal, the European Medicines Agency (EMEA) would establish a new committee, to be called the 'Paediatric Committee', which would be in charge of determining the cases in which there is a potential therapeutic benefit for children and request specific studies. It would then assess and agree the 'investigation plans' upon which studies conducted in children would be based. The committee would be in a position to grant a delay in situations where studies in children would take longer than studies in adults (The EMEA's Committee on Human Medicinal Products and national Competent Authorities is to remain responsible for the assessment of safety, quality and efficacy required for marketing authorisation).

The legislation intends to introduce incentives for conducting research into the paediatric use of medicines and for new medicinal products, patent-protected authorised medicines and off-patent medicinal products. The Commission proposal is for a six month extension of the exclusive patent given by the the supplementary protection certificate (SPC). However, at the Health Council of 3 June 2005, ministers could not reach agreement on this: some felt automatic extension would be disproportionate.

Based on the proposal, information on the results of studies in children and information on the status of the paediatric studies (waivers and delays) would be included in product information. Medicines authorised for children would include a superscript of the letter 'P', after the brand name, for easier recognition. 

The proposal also calls on the member states to develop their own national incentives for research and development. The Commission recommends that EU countries consider the training of doctors and healthcare professionals who are instrumental in conducting clinical trials in children. It advocates investment in infrastructure, such as clinical trials centres.

Positions

After the final approval of the regulation on 1 June 2006:

The European Federation of Pharmaceutical Industries and Associations (EFPIA)'s Director General, Brian Ager: "This new paediatrics regulation will allow Europe to re-claim a central role in innovative drug development worldwide, and catch up with other world regions where the necessary framework for paediatric research has been implemented to meet the needs of the patient population."

MEP Hiltrud Breyer (Greens/EFA), while welcoming the final result, thinks that pharma industry is "disproportionately rewarded for doing what it should have done all along".

MEP Françoise Grossetête, Parliament's rapporteur on the dossier said that the paediatric drugs regulation is an example of the value added the EU brings into people's everyday life. "A member state on its own cannot deliver such a policy which will benefit all children," she said. 

The International Primary Care Respiratory Group (IPCRG) welcomes the adoption of the paediatrics regulation highlighting the need for more research and appropriate authorisation of asthma medication for children. "Needs of children with asthma regarding medicinal treatment differ significantly from those of adult asthma patients. This is true not only in terms of choice of medication and dosage, but also the mode of intake." 

Earlier comments:

A press release issued by the European Generic medicines Association (EGA) complains about insufficient incentives in the off-patent sector. Setbacks for the development of medicines for children include the absence of an EU paediatric research fund and weaker protection for patent-expired medicines than in the Orphan Regulation. The EGA questioned how "reasonable" the proposed extension of patent protection is from the point of view of healthcare budgets.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) welcomed the proposal but takes the view that an incentive of six-months extension to patent protection is an absolute minimum.

Professor Jose Ramet of the Confederation of European Specialists in Paediatrics (CESP) has welcomed the proposal as a first step, saying "Europe desperately needs more medicines which are proven to be safe for children." However, he stressed the need for more action, especially on incentives, to close the gap between research capabilities in the EU and those in the US.

The European Organisation for Rare Diseases (EURORDIS)  supports the proposal and, in particular, its proposed extension of the current ten-year orphan market exclusivity to twelve years (if the requirements for data on treating children are fully met). However, EURORDIS has put forward a position paper with proposed amendments in ten different areas. EURORDIS warns that the incentives may not be enough for the sponsors, the regulatory procedure may be too complex (particularly for SMEs) and that the sponsors' obligations may prove too non-committal to guarantee the expected paediatric studies.

The European Union Geriatric Medicine Society (EUGMS) points out that the majority of medicines and treatments given to older people have not been studied in clinical trials on older populations. They say that this is the reality despite the fact that the percentage of older people in Europe is expected to rise from 3.9 to 9.4 per cent between 1993 and 2020 with the number of older people over the age of 80 set to double every ten years.

EurActiv welcomes position statements from other organisations which could be added to this dossier.

Timeline

• The Council adopted the regulation on 23 October 2006. It wll enter into force 30 days after its publication in the Official Journal.
• The European Parliament approved the final agreement on a regulation on paediatric medicines on 1 June 2006 after an informal inter-institutional (Commission, Parliament, Council) compromise on the final text was reached on 15 May 2006. 
• A joint Commission/EMEA document (September 2006) set out priorities for implementation of the regulation.
• Further provisions may be put forward by the Commission, as guidelines. 

External Links

EU official documents

• Eur-Lex:Vorschlag für eine Verordnung über Kinderarzneimittel und zur Änderung der Verordnung (EWG) Nr. 1768/92, der Richtlinie 2001/83/EG und der Verordnung (EG) Nr. 726/2004COM(2004) 599 final; 2004/0217 (COD)
• GD Unternehmen:Medicines for Children
• Europäische Agentur für die Beurteilung von Arzneimitteln (EMEA):Medicines for Children - The European Paediatric Initiative
• Europäisches Parlament / OEIL:Medicinal products for paediatric use: implementation of good clinical practice in the conduct of clinical trials (amend. regul. 1768/92/EEC, direct. 2001/83/EC, regul. 726/2004/EC)
• PreLex:Paediatric medicines proposal

Political Groups

• Peter Liese, Mitglied des Europäischen Parlaments:Background paper "Better medicines for children"
• Peter Liese, Mitglied des Europäischen Parlaments:European Cooperation vital for Fight against Child Cancer [DE]

International Organisations

• WHO-Büro für Europa:Child and adolescent health and development

Governments

• UK Medicines and Healthcare products Regulatory Agency:Medicines for children
• US Food and Drug Administration:Office of Paediatric Therapeutics

Business & Industry

• EFPIA:Industry Leaders Urge MEPs to Keep Paediatrics Regulation Simple and Effective(5. Juli 2005)
• EFPIA:EFPIA views on the European Commission's Proposal for a Regulation on Medicinal Products for Paediatric Use(April 2005)
• EFPIA:EFPIA Calls on EU Policy-makers to Set in Place a Strong and Attractive Paediatric Framework for the Benefit of Children(27. April 2005)
• EFPIA:EFPIA welcomes the Commission Proposal to Encourage Research and Development of Medicines for the Benefit of Children(29 September 2004)
• European Generic Medicines Association:EU Paediatric Medicines Proposal: EGA Urges Measures to Maximise Benefits to Children(29 September 2004)
• EFPIA:EFPIA Welcomes European Commission's New Impetus to Move Forward Europe's Competitiveness Agenda in the Pharmaceutical Sector(2. Juni 2005)

NGOs and Think-Tanks

• Confederation of European Specialists in Paediatrics (CESP):Europe’s paediatricians call for stronger research incentives to deliver safe medicines for children(9. Juni 2005)
• European Platform for Patients' Organisations, Science and Industy and other organisations:Priority Medicines for Children Conference(17-18 November 2004)
• EU Geriatric Medicine Society:Website
• European Organisation for Rare Diseases (EURORDIS):Final stages of European regulation on medicines for children(Februar 2005)
• RAND Europe (für die Europäische Kommission):Extended Impact Assessment of a Draft EC Regulation on Medicinal Products for Paediatric Use