Access to investigational medicinal products for minors in Europe. Ethical and regulatory issues in negotiating children’s access to investigational medicines

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 (IMPs), particularly where 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 IMPs is surrounded by various ethical issues, that will be explored in this article. In this paper, we explore the ethical concerns in two potential tracks of seeking access to IMPs for minors: on individual basis, or collectively, as a patient organization. In our discourse, we identify several unique ethical and regulatory concerns related to the direct negotiation of access to IMPs for minor patients, 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. We conclude our paper with a concise reflection on the way forward. The quest for access to investigational drugs is particularly relevant to pediatric practice, where a significant share of the drugs prescribed has never been tested in children or labeled for use in the pediatric population.status: publishe