35.  The need for an EU-wide regulatory framework to establish common standards for testing medicines in children and authorising products for them was already clear from the Commission Proposal and accompanying Staff Working Paper[22]. So was the need to stimulate the research and production of medicines specifically suitable for children.

36.  Earlier correspondence from the Government to us strongly supported the Proposal in principle.[23] The seven substantive responses we received from our selective consultation on the Proposal all welcomed it. Several endorsed it in strong terms.

37.  While welcoming the Proposal, the Association of the British Pharmaceutical Industry (ABPI)[24] expressed disappointment that it would be some six years after the original Council Resolution in 2000 before legislation based on the Regulation would be in place[25].

38.  Similarly strong support for the Regulation was also evident from the summary produced by the Minister of the 19 substantive responses to the MHRA consultation conducted between May and August 2005[26].

39.  The urgent need for the Regulation was emphasised in evidence from Professor Sir Cyril Chantler, the Chairman of the Great Ormond Street NHS Trust (QQ 2-4, pp1-2). He told us about a study indicating that 36% of children admitted to hospital involved the use of unlicensed or off-label treatments. Unlicensed use in new-born babies, who were particularly vulnerable, could well reach 90%. Another study suggested that 40% of new medicines had the potential to be used in children but had no market authorisation for that use.

40.  Professor Chantler explained that, without testing, it was not possible to know how a child's body might absorb, distribute, metabolise and eliminate the drug (pharmacokinetics). Nor could it be known what effect the medicine might have on the child (pharmacodynamics). Post-operative pain for children was a major problem in hospitals throughout the country. Drugs in wide-spread use to relieve acute pain in children had not been licensed for that purpose.

41.  The size and maturity of children affected the pharmacokinetics: simply scaling an adult dose based on body size dramatically oversimplified drug clearance from the body. Systems such as kidney function developed with age. The immunosuppressive effect of medicines given to prevent the rejection of transplanted organs was very different in children than in adults.

42.  For example, over-dosage had led to some antibiotics causing deafness in children. Serious errors could occur in diluting dosages of medicines suitable for adults without specific test information. He had no doubt that the lack of suitable and properly-licensed medicine exposed children to significant mortality and morbidity.

43.  Pharmaceutical preparations that were not made for children could be difficult to administer. They might need to be reformulated without adequate information on the consequences of reformulation, which could either make them dangerous or ineffective.

44.  The need for clinical trials in children was borne out by experience in the USA and was supported by 90% of European paediatricians in a survey recently reported to the European Parliament. He believed that the Regulation was long overdue (Q 21).

45.  The Minister told us that the proposed Regulation would be the key to addressing long-standing UK concern over the lack of medicines authorised and formulated specifically for paediatric use. The regulatory approach proposed by the Commission included incentives and requirements designed to ensure that new medicines for children, and those already on the market, met the specific needs of children. But it also ensured that children were not subjected to unnecessary clinical trials or that the authorisation of medicines for adults was needlessly delayed (Q 37).

46.  We conclude, in principle, from the evidence we have had that there is an overwhelming and urgent need to take effective action at European level to govern clinical trials in children and the authorisation of medicinal products for paediatric use with the minimum of delay.

23   pp 12-14 and pp 31-32 Back

24   pp 25-26 Back

25   pp 26-27 Back

26   pp 48-56 Back