The purpose of licensing is to ensure that medicines are examined for efficacy, safety and quality. Only when a company holds a product licence (or marketing authorisation) may they advertise and sell their product in the UK. It is not the intention of the Medicines Act legislation to restrict what doctors prescribe. Clinicians are allowed to use or recommend medicines which do not have a licence (unlicensed), or to use medicines for other purposes than those in the licence specification (off label).
'Unlicensed' medicines include chemicals used as medicines, modification of licensed medicines (extemporaneous dispensing), imported medicines licensed in other countries and special manufacture or assembly. 'Off label' medicine, (an American term), describes the use of licensed medicines in a dose, age group, or by a route not in the product licence specification. Some of the medicines we give to children are 'unlicensed', many are 'off label'.
This creates a dilemma for all concerned, the Health Trusts, pharmaceutical companies and the licensing authority, for on the one hand children may be denied an effective medicine because it is not licensed, or on the other, given a medicine which has not been subject to rigorous scrutiny.
Giving an unlicensed or 'off label' medicine is not illegal, but it is a risk management issue which needs to be addressed. The data sheet (summary of product characteristics) is considered to be the most authoritative source of information on drug doses and indications. Although there is no case law, it is generally considered that if a practitioner prescribes or dispenses a medicine within the terms of the product licence, s/he will not be found at fault (though the licensing authority might be sued for negligence!).
Authorities in the US, Canada and Australia have all begun to review this problem. In the UK a joint working party of the BPA (with a pharmacist member) and the ABPI have prepared a report on 'Licensing Medicines for Children', which has been seen and approved by the CSM. It makes recommendations on age ranges, clinical trials, CPMP licensing guidelines, interpretation of clinical studies, surveillance of unlicensed and 'off label' usage and the provision of suitable information. The latter includes statements on 'orphan drugs', expanded data sheets and the preparation of a national formulary for children.
Because so many medicines given to children have not been evaluated fully it is hoped that the formulary will provide an opportunity for all those concerned in giving medicines to children; doctors, pharmacists, nurses; in the home, school or hospital, to share information to promote the safe and effective care of children.
In answer to the question posed - yes there is a problem, but there are solutions. Children need not be 'therapeutic orphans'.
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