Off label medicines and the right to the most appropriate care available. The case of antipsychotics for psychiatric disorders in the developmental age

Drug use in the paediatric population is still characterized by frequent off label prescriptions, in particular for certain classes such as psychotropic drugs. The licensing status varies widely between countries both for marketed drugs, and for age and therapeutic indications for which they are registered. Off label antipsychotic use is common in Italy, where licensing status and evidence of safety and efficacy take longer to become aligned than in other countries. In this regard, a systematic evaluation of the available evidence for the 28 antipsychotic drugs marketed in Italy (out of 67 in the therapeutic class) was carried out. Clinical trials were found for 11 of them, 9 of which were randomised controlled trials. The use of 3 of these antipsychotics (clozapine, olanzapine and quetiapine) is considered not off label based on the Law 648/96. For 4 other drugs (aripiprazole, lithium, pimozide, risperidone) at least 2 high quality RCTs are available and these drugs have been included in systematic reviews and in national and international guidelines. For these reasons, and taking into account the licensing status in the UK and USA, the 4 antipsychotics cited above could be included in the list of drugs reimbursed by the National Health Service, even when prescribed off label (Law 648/96). This measure may represent a formal recognition of therapies of documented efficacy in childhood that address unmet needs such as neuropsychiatric disorders.