The availability and age‐appropriateness of medicines authorized for children in the Netherlands

Abstract

WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT• Children are entitled to safe, efficacious, child and parent friendly (i.e. age-appropriate) medicines. However, off-label and unlicensed paediatric prescription rates in hospital range from 36% (surgical and medical wards) to 93% (neonatal wards), which indicates a general lack of age-appropriate medicines for children. In 2007, the European Union issued the Paediatric Regulation aiming at better medicines for children by, for example, increasing the number of authorized paediatric medicines. WHAT THIS STUDY ADDS• This study reviews the availability and age-appropriateness of medicines for children in the Netherlands, herewith providing a baseline for an estimation of the effect of the European Paediatric Regulation in the near future. This study also provides a tool for the evaluation of the child authorization status of medicines on the basis of the information in the Summary of Product Characteristics (SPC) and for the assessment of key aspects of the age-appropriateness of paediatric formulations. AIMTo study the number of medicines and active chemical entities that are authorized and commercially available for children in the Netherlands and to evaluate the age-appropriateness of the available paediatric medicines. METHODSThe availability of paediatric medicines and active chemical entities was studied with the help of a Dutch medicines database and the Summary of Product Characteristics. Medicines were categorized with respect to their route of administration, type of oral dosage form and therapeutic category. The age-appropriateness was assessed on three aspects: dose capability, suitability of the dosage form and inclusion of potentially harmful excipients. RESULTSThree thousand five hundred and forty-two paediatric medicines containing 703 different active chemical entities were identified. This equalled half of all the medicines and chemical entities available for human use. The percentage of paediatric medicines increased with age and varied for the route of administration from 22% (dermal) to 81% (inhalation) and for the therapeutic category from 11% (uro-genital, sex hormones) to 89% (anti-parasites). The appropriateness of the paediatric medicines with respect to their authorization status, dose capability and dosage form increased with age from 27-88%. Fifty-two percent of all oral paediatric liquid formulations contained a potentially harmful excipient. CONCLUSIONThis study confirms the limited availability of paediatric medicines for a broad range of therapeutic areas and shows that paediatric medicines may not be age-appropriate, even if authorized. While confirming the need for a legislative incentive, the results also provide baseline information for an estimation of the effect of the European Paediatric Regulation in the near future.

Prescribing for off-label use and unauthorized medicines in three paediatric wards in Finland, the status before and after the European Union Paediatric Regulation

“…In paediatric inpatients, the off‐label use of medicines and use of unauthorized medicines are common . The availability of authorized and commercially available medicines for children of all approved medicines varies between 48% and 54% according to the recent studies conducted in the Europe, Oceania and the United States of America (USA) . The availability decreases according to age; neonates have the least appropriate medicines with respect to information available .…”

“…The availability of authorized and commercially available medicines for children of all approved medicines varies between 48% and 54% according to the recent studies conducted in the Europe, Oceania and the United States of America (USA) . The availability decreases according to age; neonates have the least appropriate medicines with respect to information available . In addition to active ingredients, preservatives and other excipients constitute problems in paediatric pharmacotherapy.…”

Acceptability of different oral formulations in infants and preschool children

“…Oral liquid medicines are more commonly available for use in infants and preschool children than oral solid (flexible) medicines, such as powders or orodispersible tablets 8. Small-sized tablets, also referred to as minitablets, have been identified as a new type of oral solid dosage form in which to administer medicines to young children.…”

The EMA quality guideline on the pharmaceutical development of medicines for paediatric use

“…The availability of licensed medicines and active substances for children was lagging behind those for adults, unlicensed and off-label prescription rates were high, there was a lack of suitable formulations for children especially the very young and, despite a positive benefit to risk profile, the suitability of some licensed paediatric medicines was questioned in literature (Cohen et al, 2009;European Medicines Agency, 2007;Van Riet-Nales et al, 2010). All this resulted in Regulation 1901Regulation /2006 or the Paediatric Regulation.…”