Framework conditions facilitating paediatric clinical research

Abstract

The use of unlicensed and "off-label" medicines in children is widespread. Between 50-80% of the medicines currently administered to children have neither been tested nor authorized for their use in the paediatric population which represents approximately 25% of the whole European population. On 26 January 2007, entered into force the European Regulation of Paediatric Medicines. It aims at the quality of research into medicines for children but without subjecting the paediatric population to unnecessary clinical trial. This article addresses ethical and legal issues arising from the regulation and makes recommendations for the framework conditions facilitating the development of clinical research with children.

Pediatric clinical trials

“…[7–12] The European Regulation of Pediatric Medicines has three major initiatives for ensuring that children will receive drugs that are safe and efficacious: the adoption of incentives for industry, the implementation of a mandatory Pediatric Investigation Plan (PIP) considering all age ranges and the creation of a Pediatric Committee (PDCO). [13] The pharmaceutical companies are obliged to submit a PIP for new indications, new routes of administration or new formulations of already patented products and for the development of new medicinal products. If information is correctly provided after conducting the required studies in compliance with the PIP, the company is rewarded with a six-month extension of the Supplementary Protection Certificate.…”

“…Under the Pediatric Use Marketing Authorization (PUMA), if studies based on pediatric indications and formulations are carried out in line with the agreed PIP; the applicant can get a PUMA approval with 10-year market exclusivity. [13] In comparison, the US approach for pediatric authorization seems pragmatic and more flexible. It asks companies to complete Pediatric Development Plan (equivalent to PIP in the EU) providing sufficient data base from adult population.…”

Ethical challenges in paediatric clinical trials in multiple sclerosis

“…The inclusion of paediatric patients in MS trials evaluating new therapeutic agents is essential to reduce the use of medications that are currently off-label, and to ensure that children are exposed to safe as well as effective treatments [Knellwolf et al 2011;Henschel et al 2010]. Nevertheless, there is an urgent need to update the methodology used to conduct clinical trials in paediatric patients, as well as to define validated endpoints for these studies.…”

“…Therefore, using an international network may be essential to enhance the work of ethics committees in paediatric research. In addition, the selection of an ethics committee coordinator/facilitator, ethically and economically independent from the sponsors of the study, would guarantee the quality of discussions during the evaluation process [Knellwolf et al 2011].…”

“My Parents Decide If I Can. I Decide if I Want to.” Children's Views on Participation in Medical Research

“…R esearch involving children is increa singly regarded as important, since 50-80% of all drugs prescribed to children have not been tested on children (Knellwolf et al, 2011). Researchers carrying out research involving children face a number of ethical challenges: to judge whether the children to be involved are competent, to respect their views, and to actively involve them in the research (cf.…”