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OHP-042 Impact of the Paediatric Regulation on the environment of paediatric medicines use: paediatric investigation versus off-label drug use
  1. T Roldan Sevilla,
  2. E Villamañán Bueno,
  3. M Ruano Encinar,
  4. P Gómez Salcedo,
  5. C Rueda Pérez,
  6. A Herrero Ambrosio
  1. Hospital Universitario La Paz, Hospital Pharmacy, Madrid, Spain


Background In 2006, the European Parliament approved paediatric standards (Paediatric Regulation 1901/2006) in order to stimulate the development of paediatric clinical trials (PCT) reducing off-label drug use (ODU).

Purpose To evaluate the variation in PCT and ODU in children over the last 6 years since this paediatric regulation came into force. We also analysed the type of PCT and characteristics of drugs involved in PCTs or prescribed in conditions that they are not approved (off label).

Materials and methods Observational retrospective study in a 252-bed children’s hospital which forms part of a tertiary hospital. The main outcome measured was the number of PCT carried out per year and ODU per year (from 2007 to 2012). Furthermore, PCT design, type of drugs used in both conditions (experimental and off-label), and reasons for off-label use according to the summary of product characteristics were assessed as well.

Results We analysed 87 drugs involved in PCT and 449 non-investigational drugs, of which 117 (26%) were considered off-label prescriptions. We observed an increase in PCT carried out per year from 9 in 2007 to 23 in 2011, reaching 19.3% of total clinical trials performed in our centre. Off-label drug use remained stable over the study period. The most common type of PCT design was phase III non-randomised open-label (27.6%). Concerning the drugs involved, antibiotics and antineoplastic-immunosuppressant agents were the most common drugs investigated in PCT, while off-label prescriptions mainly involved hypnotics-sedatives and anticoagulants. Most cases of these off-label prescriptions were related to the lack of studies.

Conclusions Since January 2007 when the paediatric regulations came into force in the European Union, an increase of PCT performed has been detected in our centre. However, this fact has not affected off-label drug use which has not changed mainly due to a lack of research into drugs in children.

No conflict of interest.

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