All Italian physicians, nurses, and pharmacists have recently received, free of charge, the Guida all’uso dei farmaci per i bambini (Guide to the use of drugs for children).¹ Considering the number of health professionals involved (about 600 000), the methodology followed, the completeness of the contents, the size of the book produced, and its free availability, this initiative, set up under the auspices (technical and economic) of the Italian Ministry of Health, is unique at the national and international levels. This formulary can be considered part of the cultural trial begun in the mid 1990s by the Royal College of Paediatrics and Child Health and the Neonatal and Paediatric Pharmacists Group, which led to the creation of Medicines for children² in 1999, a tool characterised by an evidence based approach and designed to assist those who prescribe, dispense, or administer medicines to children. While work was in progress on the first Italian Guide and the second UK edition of Medicines for children, formal exchanges took place between the two groups.

The main objective of a formulary is not simply to list drugs and their therapeutic profiles, but to function as an essential tool for a rational use of medicines. A formulary must therefore be a source of up to date, evidence based information about both medicines and therapeutic approaches for the most frequent clinical problems, in and out of hospital. These traits have characterised the formularies developed since the 1970s, but only recently has such an undertaking focused on children’s needs.

The need for additional sources of paediatric drug information, such as formularies, arises from an inadequate drug evaluation and registration process. The insufficient data leads to inevitable off-label or unlicensed drug use, which may entail a certain degree of risk for patients.³ This unsatisfactory standing of children with regard to optimal drug therapy is well recognised, especially in the USA and Europe, but the situation in the latter has been better defined through numerous studies.⁴ Off-label/unlicensed prescription rates are in the range 23–62% in European paediatric hospital wards, 55–80% in neonatal intensive care units, and 11–49% in the community setting.⁴ Wide differences in therapeutic approaches were found between and within settings and countries, suggesting the need for “harmonisation” in clinical practice.

Paediatric studies are more difficult than adult studies because of ethical, practical, and economic considerations. Attempts to improve the situation in Europe were initiated in 1997 when the Committee for Proprietary Medicinal Products (CPMP), part of the European Agency for the Evaluation of Medicinal Products (EMEA), published the “Note for guidance on clinical investigation of medicinal products in children”. Later efforts involved the European Commission’s Better medicines for children 2000 document proposing new regulatory actions to address the lack of suitably adapted paediatric medicinal products, the creation of a Paediatric Expert Group by the CPMP in 2001 to encourage the development of paediatric medicinal products, and the decision by the European Union, under its Fifth Framework Programme in 2002, to support the development of a European register of clinical trials in children (DEC-net project; www.dec-net.org; contract QLG4-CT-2002-01054). The role of the European register is to handle essential data on completed and ongoing research as a useful resource for planning new studies, promoting communication and collaboration among researchers, facilitating patient access and recruitment into trials, preventing trial duplication and inappropriate funding, and identifying therapeutic needs of children that remain neglected.⁵ The Sixth Framework Programme is currently running and “Medicines for children” is one of the specific topics. In 2004, the European Commission published a draft consultation document, Regulation of the European Parliament and Council on medicinal products for paediatric use, which is currently undergoing final revisions.

The inevitable use of deduction as a means to obtain what is “probably” the best therapy for a child may gradually disappear, but the continued production and availability of evidence based information for health professionals and lay people has to be guaranteed. Children have the same rights as adults to receive safe and effective medicines. In such a context, guiding clinicians to ensure that children benefit from the medications they are given is a priority, and a collaborative, shared paediatric formulary would be useful in accomplishing this. A continuation of the efforts made in the UK and Italy, with the joint participation of different countries in an endeavour to set up a European formulary led up by an international committee, would be a valuable achievement and should be supported. The project would be a challenge for the Community and would result in a consensus on paediatric drug therapies (a qualitative and quantitative synthesis of the evidence),⁶ guaranteeing all children the same approved, acknowledged therapeutic approaches to which they have a right.