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