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