Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children

Abstract

This is an exciting time for research and novel drug development in cystic fibrosis. However, rarely has the adage, "Children are not just little adults" been more relevant. This article is divided into two main sections. In the first, we explore why it is important to involve children in research. We discuss the potential benefits of understanding a disease and its treatment in children, and we highlight that children have the same legal and ethical right to evidence-based therapy as adults. Additionally, we discuss why extrapolation from adults may be inappropriate, for example, medication pharmacokinetics may be different in children, and there may be unpredictable adverse effects. In the second part, we discuss how to involve children and their families in research. We outline the importance and the complexities of selecting appropriate outcome measures, and we discuss the role co-design may have in improving the involvement of children. We highlight the importance of appropriate staffing and resourcing, and we outline some of the common challenges and possible solutions, including practical tips on obtaining consent/assent in children and adolescents. We conclude that it is unethical to simply rely on extrapolation from adult studies because research in young children is challenging and that research should be seen as a normal part of the paediatric therapeutic journey.

Keywords: children; clinical trials; cystic fibrosis; ethics; patient-centred trials.

Figure 1

Multiple breath washout performed on a healthy subject. Top red line shows volume, whilst in black is the flow. The nitrogen (N₂) trace shown in by the burgundy line, is used as the tracer gas, and it is “washed out” with 100% oxygen. During inspiration of 100% oxygen the N₂ goes to 0%, whereas during expiration the breath is captured. N₂ decreases in a step-like manner throughout the washout. Only 80 s of 100% oxygen breathing is required to reduce N₂ down to the target concentration (dashed grey line = 2.5% of the starting N₂ concentration hence LCI_2.5). Subject has a normal LCI_2.5 of 6.50.