The European Paediatric Regulation (EUPR) calls for the fostering of high quality ethical research and medicinal products to be used in children. The EUPR provides the background, goals, and requirements for paediatric clinical trials. Paediatric clinical trials in children are mandatory to generate data on new drugs as well as on drugs used off-label or for unlicensed indications. The Family P...

Micro bubbles contrast agents registered in Europe are licensed only for cardiac, liver, breast and vascular applications (Sono-Vue®). Levovist® is also registered for vesi coureteric studies, but is practically no longer marketed, and is expected ultimately to be no longer available. Thus, lots of daily non-hepatic applications continue to be off-label and patients should be informed and must ...

For too long children have received medicines not sufficiently studied for their needs and, in fact, being considered as small replicas of adults, it was deemed sufficient to adjust the dosage of a drug approved for adults. Together with the limited availability of appropriate drug formulations, especially for neonates and toddlers, this approach has caused increased iatrogenic risk and/or subo...

The use of contrast-enhanced ultrasound (CEUS) in adults is well established in many different areas, with a number of current applications deemed "off-label", but the use supported by clinical experience and evidence. Paediatric CEUS is also an "off-label" application until recently with approval specifically for assessment of focal liver lesions. Nevertheless there is mounting evidence of the...

Rigorous scientific investigation is the foundation for many of the remarkable advances in medical care that have occurred over the course of the last century. Patients with cardiovascular diseases, in particular, have received considerable benefit from such research, especially that which pertains to drugs and medical devices. Infants and children, however, have not benefited proportionately f...

We present a review and analysis of the ethical considerations in off-label ketamine use for severe, treatment-resistant depression. The analysis of ethical considerations is contextualised in an overview of the evidence for ketamine use in depression, and a review of the drug's safety profile. We find that, based on current evidence, ketamine use for severe, treatment-resistant depression does...

Bone morphogenetic proteins are osteoinductive factors which have gained popularity in orthopaedic surgery and especially in spine surgery. The use of recombinant human bone morphogenetic protein-2 has been officially approved by the United States Food and Drug Administration only for single level anterior lumbar interbody fusion, nevertheless it is widely used by many surgeons with off-label i...

BACKGROUND Reported off-label/unlicensed prescribing rates in children range from 11% to 80%. Research into pharmacokinetic profiles of children's medicines is essential in the creation of more knowledge on the safety and efficacy of medicines in children. This study investigated how often pharmacokinetic data are collected in clinical trials of medicines in children by analysing registered rec...

Prescribing medications off-label is commonplace in dermatology. Recent policy changes on the regulatory abilities of the US FDA and legal precedents regarding this topic have led to intense debate on free speech about off-label drug use by physicians and drug manufacturers. Here, we summarize and discuss the risks and benefits of off-label promotion and how this relates to quality patient care...

Dear Editor: Sterile ophthalmic injections with 2 antibiotics (ceftazidime and vancomycin) and 1 glucocorticoid (dexamethasone) are used at University Hospital of Geneva in some patients with infectious complications after intraocular surgeries. This is an off-label indication. They are administered as intravitre-ous injections and need to be prepared just before utilization , because the subst...