Article Text

other Versions

PDF
Access to investigational medicinal products for minors in Europe: ethical and regulatory issues in negotiating children's access to investigational medicines
  1. Wim Pinxten,
  2. Herman Nys,
  3. Kris Dierickx
  1. Katholieke Universiteit Leuven, Centre for Biomedical Ethics and Law, Leuven, Belgium
  1. Correspondence to Wim Pinxten, Centre for Biomedical Ethics and Law, KU Leuven, Kapucijnenvoer 35 Box 7001, BE-3000, Leuven, Belgium; wim.pinxten{at}med.kuleuven.be

Abstract

Patients who search for a better treatment, an increased quality of life, or even a chance to preserve life itself may claim to have an interest in accessing investigational medicinal products (IMP), particularly when no validated treatment for their disease or condition exists. For many, awaiting the uncertain and time-consuming process of converting an IMP into an approved drug may not appear a realistic option, as prognoses may be grim and a dramatic outcome may seem hard to avert. Gaining access to an IMP, however, often proves to be a difficult enterprise with a highly uncertain outcome. In addition, the process of seeking access to IMP is surrounded by various ethical issues that will be explored in this article. This paper explores the ethical concerns in two potential tracks of seeking access to IMP for minors: on an individual basis, or collectively, as a patient organisation. In this discourse, several unique ethical and regulatory concerns related to the direct negotiation of access to IMP for minor patients are identified, with a focus on product safety, the recruitment of research subjects, the unnoticed entry of market mechanisms in the recruitment of research subjects, and the sidelining of third parties in the recruitment process. The paper concludes with a concise reflection on the way forward. The quest for access to investigational drugs is particularly relevant to paediatric practice, in which a significant share of the drugs prescribed has never been tested in children or labelled for use in the paediatric population.

  • Access
  • investigational medicinal products
  • Massart
  • minors
  • newborns and minors
  • patient organisations

Statistics from Altmetric.com

Footnotes

  • Competing interests None.

  • Provenance and peer review Not commissioned; externally peer reviewed.

Request permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.