EFCNI has repeatedly expressed concern about the lack of evidence in the treatment of newborn babies and has called for more research in this field. EFCNI is firmly convinced that more clinical trials are needed to develop new, safe and efficient treatments for newborns that are based on hard evidence. However, it is just as important to conduct the trials following high ethical and clinical standards.
In a recent article, published in the scientific journal ACTA PAEDIATRICA, Nicole Thiele et al., on behalf of the EFCNI Parents Advisory Board, explains randomised controlled trials (RCTs) in general and gives recommendations about the conduct of cluster randomised controlled trials in babies in particular.
Randomised controlled trials (RCTs) are the gold standard in clinical research because they reduce bias in trials. In a RCT, the participants (patients) are randomly divided into two groups, one group receiving a treatment and the other one (so-called control group) either receiving no treatment at all, or another treatment, e.g. standard treatment. Depending on the specific goal of a study, the results should be able to provide information, for example, if a new medication is more efficient than an older one, or if a medication is better than no treatment at all.
The article puts a focus on the following two types of RCTs:
In individually randomised controlled trials, parents, as their baby’s legal representatives, are individually given the choice if they want their baby to participate or not. To make an informed decision, parents need to get enough and comprehensive information on the study, and they can freely choose to participate or not. If they do not want to participate, their baby will receive standard treatment according to the relevant guidelines. If the parents decide to participate in the study, their baby is randomly assigned to one of the groups, which means that there is usually a 50% chance (depending on the trial design) to be part of the group receiving the new treatment or be part of the standard group receiving standard treatment.
In a cluster randomised controlled trial, whole organisations participate, e.g. whole hospitals, hospital departments, or doctor’s practices. They usually test certain interventions, e.g. lifestyle or educational interventions, or organisational procedures. The parents are informed about this trial and can choose to participate or not, but if they do not consent, they need to go to a different hospital. In cluster randomised controlled trials it might appear more difficult to make an informed decision.
Therefore, in this article, the authors and EFCNI are giving a long list of recommendations on cluster randomized controlled trials, based on the parental perspective:
• Where possible, individually randomised controlled trials are to be preferred over cluster trials, so there should be a consensus among healthcare professionals that in the particular
case a cluster trial is more adequate.
• The highest scientific and ethical standards need to be observed (defined by the Declaration of Helsinki).
• Parents and parents organisations need to be fully involved in the research project right from the beginning.
• Parents receive comprehensive and comprehensible information on benefits and risks of the trial.
• The parents’ written consent is mandatory.
• The use of study data needs to follow strict rules, and no data are collected if parents refused to participate in the study.
Parental views on the principles for cluster randomised trials involving neonates and infants
Nicole Thiele, Johanna M. Walz, Verena Lindacher, Silke Mader, Gorm Greisen, on behalf of the EFCNI Parents Advisory Board
Acta Paediatr. 2018 Nov 26. doi: 10.1111/apa.14664