Medicines Control Agency (Hansard, 20 July 2001)

§ Ms Blears

[holding answer 17 July 2001]: In the United Kingdom, there is a legal exemption to allow supply of unlicensed medicines under certain conditions. This allows a doctor or dentist to prescribe an unlicensed medicine or a licensed medicine outside its licensed indications ('off-label' prescribing) for the special needs of his individual patient. For instance a doctor may prescribe a medicine for a child that is licensed only for adults. The Department has not undertaken research into such prescribing, or monitored the level of its use. However, reports in the literature of results of hospital-based surveys indicate that such prescribing is extensive in children. For example, in a neonatal intensive care unit around 90 per cent, of neonates received off-label or unlicensed medicines¹; in an intensive care unit around 70 per cent. of children received a medicine outside a licensed indication and 31 per cent. of prescriptions were for unlicensed or off-label use²; and in a study of children in medical and surgical wards 25 per cent. of the products prescribed during the admission were for unlicensed indications³. In primary care the incidence of off-label or unlicensed prescribing to the paediatric population is thought to be around 10 per cent.⁴. Copies of the referenced papers will be placed in the Library. No similar evidence exists for adults.

We recognise the critical importance of this issue and agree that children should have access to medicines that have been fully evaluated to the same high standards of safety, quality and efficacy as those available for the adult population. The problem is not confined to the UK, but affects the whole of Europe and the United States of America. The UK is leading the call for action at a European level. Medicines regulation in the UK derives largely from European legislation and an international approach to the problem is therefore needed.

We have consequently raised the profile of this issue at a European level, including taking the lead in developing a European guideline, adopted in 1997, to encourage companies to undertake appropriate clinical trials on the use of medicines in the treatment of children. This formed the basis for an international guideline, which came into operation in January in the European Union, and also applies in the USA and Japan. In addition, the Council of the European Union has asked the European Commission to bring forward measures to make sure that medicines for children are fully adapted to their specific needs. We are presently waiting for their proposals. However, the Commission has stated its intention to comply with this request. The UK will play an active role in the development of this initiative to ensure that effective solutions are found.

In addition the Committee on Proprietary Medicinal Products (CPMP), the scientific advisory committee of the European Medicines Evaluation Agency (EMEA), recognising the importance of this topic has announced its intention to set up a paediatric expert group to advise the EMEA and its scientific committees on all questions 726W relating to the development and use of medicinal products in children. The UK has nominated representatives to this paediatric expert group.

In the meantime, the UK Government have taken important steps at a national level within the existing regulatory framework. The Committee on Safety of Medicines (CSM), an independent expert committee that advises the licensing authority, asks companies for paediatric development plans where appropriate and has established a paediatric sub-group to provide expert advice on the licensing and safety of medicines for children. Product information for parents and carers is being simplified and the profile of monitoring the safety of medicines used in the treatment of children has been increased. Since 1998, the MCA has jointly funded a pilot scheme in Trent, the Paediatric Regional Monitoring Centre (PRMC), to stimulate reporting of adverse drug reactions (ADRs) to medicines used in the treatment of children. The scheme aimed to stimulate the reporting of suspected ADRs in children. The progress of the PRMC was reviewed after two years and it was concluded that it has been only of limited success. The MCA and CSM are now proposing to pursue a wider strategy to enhance existing data collection mechanisms and the lessons learned from the PRMC will be fully utilised in taking forward this strategy. The Government are committed to finding a long-term solution so that parents and carers can be reassured about the safety of medicines given to children.

I also refer my hon. Friend to the reply I gave to my hon. Friend the Member for Edmonton (Mr. Love) on 8 May 2001, Official Report, columns 147–48W.

¹ Conroy S. McIntyre J. Choonara I. Unlicensed and off label drug use in neonates. Arch. Dis. Childhood 1999: 80: F142–145

² Turner S. Gill A. Nunn AJ. Hewitt B. Choonara I. Use of 'off label' and unlicensed drugs in paediatric intensive care unit. The Lancet 1996: 347: 549–550

³ Turner S. Longworth A. Nunn AJ. Choonara I. Unlicensed and off label drug use in paediatric wards: prospective study: BMJ 1998: 316: 343–346

⁴ McIntyre J. Conroy S. Avery A. Corns H. Choonara I. Unlicensed and off label drug use in general practice. Arch Dis Child 2000: 83: 498–501