Medical Research Involving Minors
Last update: December 2016
Contact: Bert Heinrichs
I. Medical aspects
Especially since the second half of the twentieth century, medical research (see module Medical Research) has led to the development of numerous therapeutic, diagnostic and preventive techniques. These advances have also been reflected in paediatrics. Yet the balance of progress in this field, compared with other areas of medicine, has been considerably more modest. In a paper from 1968 the paediatrician Harry Shirkey coined the phrase "therapeutic orphans" (see module Therapeutic Orphans) in order to highlight the extent to which children are neglected - in comparison to adults - when it comes to the development of new medical products and procedures.
It continues to be the case that the treatment of minors with drugs frequently takes place using so-called off-label or off-licence (see module Off-Label and Off-Licence) products i.e. with substances that are either not licensed for paediatric use at all due to a lack of clinical trials or at least do not have the appropriate licence for the specific area of application. Paediatricians then have to decide on the administering of a drug based on empirical values. Most significantly, they also have to determine the dose (see module Dose Finding) without recourse to scientifically secure data.
Even though the off-label or off-licence use of drugs is widespread in paediatrics, this can only be a makeshift solution from the medical standpoint. Children are not "little adults", especially from the perspective of developmental physiology, which is, amongst others, highlighted by the German Academy for Pediatrics and Adolescent Medicine (DAKJ) (see module German Academy for Pediatrics and Adolescent Medicine (DAKJ)) in a statement on medical research involving minors. As numerous examples have demonstrated, it is therefore often not possible to simply adjust the dosage of drugs that are successfully used on adults. Cases like the “grey baby syndrome” (see module Grey Baby Syndrome) or the off-label prescription of antidepressants to minors (see module Off-label prescription of antidepressants to minors) exemplify these controversies. A further problem consists in that the off-label use of drugs can only take place under certain conditions which were set by the Federal Social Security Court (see module Verdicts of the Federal Social Court Concerning the Off-Label Drug Use) during two verdicts in 2002 and 2006 respectively, to the disadvantage of the 'Gesetzliche Krankenversicherung' (GKV) which stands for statutory health insurance.
In this context it cannot even be considered appropriate to use the blanket term "children". On the contrary, it is vital to differentiate between several developmental phases (see module Child Development Phases and Medical Research) up to adulthood, each of which has its own distinct physiological characteristics. Only by conducting targeted clinical trials on minors will it be possible to really remedy the problem of children as "therapeutic orphans".
In certain areas of paediatrics, most notably in paediatric oncology, large numbers of clinical trials are already being carried out today. Indeed, estimates suggest that in the United States, for example, roughly 70% of all child cancer patients are treated as part of clinical trials. This does, however, raise the question of whether crucial distinctions between medical research and medical practice (see module Therapeutic Research and Group Benefit) are being ignored. In Germany, too, clinical tests on minors are being carried out. According to an evaluation amongst VFA-businesses (see module VFA (Association of Research-Based Pharmaceutical Companies)), this is, however, the case stagnantly and only on a small scale.