| Children (particularly those 0-28 days old i.e. neonates) | | | | indication the new medicine is then eligible for a 6 |
| respond to drugs in a very different way to adults. | | | | month extension of its patent protection. This patent |
| Typically, doses in children are calculated by weight | | | | protection is further extended for Orphan-designated |
| and occasionally by body surface area but the dose | | | | medicines by another 18 months giving a total of 12 |
| should never exceed the maximum adult dose. This, | | | | years of exclusivity (as opposed to 10 years). |
| however, is only a guide as for some drugs children | | | | Existing product marketing authorisations |
| require a higher dose per kilogram than adults | | | | From 26 January 2009, the requirements above will |
| because of their higher metabolic rates. In addition, | | | | also apply to marketing authorisation variations. |
| working out dosage by weight should not be applied | | | | Paediatric-use Marketing Authorisation (PUMA) |
| to an overweight child as it could result in a much | | | | This is a new marketing authorisation which applies to |
| higher dose being administered than is actually | | | | off-patent medicines which have been developed |
| necessary, safe or desirable. In this instance, the | | | | specifically for paediatric use. Again, product |
| dose has to be calculated using the ideal weight and | | | | development must comply with the agreed PIP and |
| not actual weight. To say that a child is half the size | | | | provided there is compliance, a pharmaceutical |
| of an adult and therefore should receive half the | | | | company will benefit from 10 years of data |
| adult dose is too simplistic and could well be | | | | protection. |
| dangerous. | | | | Paediatric Investigation Plans (PIPs) |
| The British National Formulary (BNF) for Children | | | | Otherwise known as a "drug development |
| provides healthcare professionals with up-to-date | | | | plan" this is a development plan which ensures |
| information on the use of medicines for treating | | | | the collection of relevant data from studies in children |
| children both within the realms of the medicine's | | | | (when it is safe to do so). It must include details of |
| licence (Marketing Authorisation) and for | | | | these studies and the ways in which the medicine |
| "off-label" use i.e. the use of licensed | | | | has been adapted to make it suitable for children. For |
| medicines (in adults) for unlicensed uses. This | | | | example, children will more readily swallow a syrup |
| resource is invaluable for the effective and safe | | | | than a tablet. In some instances studies in children will |
| treatment of children. | | | | be deferred until the studies in adults have been |
| The use of unlicensed and "off-label" | | | | completed. This ensures that studies in children are |
| medicines in children has caused concern, however, | | | | carried out only when it is safe, and ethical, to do so. |
| throughout the EU for a number of years now as at | | | | Of course, there are some illnesses which do not |
| least 50% of medicines used to treat children have | | | | affect children which means a PIP will not be required |
| never been tested in children, only adults. This lack of | | | | and hence 'waived'. |
| supporting data for the treatment of children was | | | | For many companies the Paediatric Regulation is a |
| felt to have contributed to an unacceptable number | | | | completely new challenge and, up until recently, they |
| of adverse events. As a result of this, and the overall | | | | will not have incorporated it into their business |
| need to improve on the information available, the | | | | strategy. With ever decreasing headcount, it is |
| European Paediatric Initiative came into being with a | | | | becoming more and more difficult to keep |
| new EU Paediatric Regulation1 entered into force on | | | | up-to-speed with all the latest legislation and the |
| 26 January 2007. | | | | implications of enforcement. Regulatory Consultancies |
| This new regulation has several implications with the | | | | such as GRS can help companies by offering the |
| overall purpose being to provide "better | | | | services of senior regulatory professionals who |
| medicines for children"2 and these are as | | | | already have working knowledge and experience of |
| follows: | | | | the Paediatric Regulation. With the pharmaceutical |
| New product marketing authorisations | | | | industry, the Health Authorities and regulatory |
| From July 2008 any new products which were not | | | | professionals working together we can have a |
| authorised within the EU before 26 January 2007 | | | | positive effect on the development, availability and |
| have to include results of studies carried out in | | | | safety of medicines for the treatment of children and |
| children. These studies must comply with an agreed | | | | improve the availability of information on the use of |
| Paediatric Investigation Plan (PIP) unless, of course, a | | | | these medicines. This new legislation may be |
| deferral or waiver has been agreed with the EMEA. A | | | | challenging and time consuming however it will provide |
| waiver may be granted, for example, when a new | | | | a safer future for our children. |
| medicine is intended to treat a condition which only | | | | 1 Regulation (EC) No 1901/2006 of the European |
| occurs in adults (for example Parkinson's disease). As | | | | Parliament and of the Council on medicinal products |
| an incentive for pharmaceutical companies, once | | | | for paediatric use, amended by Regulation (EC) No |
| authorisation has been granted for a paediatric | | | | 1902/2006. |