Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children
Rebecca Dobra1,2, Siân Bentley1,3, Claire Edmondson1,2
1National Heart and Lung Institute, Imperial College, London SW3 6LY, UK.
Insights
Pediatric research in cystic fibrosis is crucial because children differ from adults in drug absorption and effects. Involving children in studies ensures ethical, evidence-based care and avoids risks from adult data extrapolation.
Area of Science:
- Pediatric Research
- Cystic Fibrosis Therapeutics
- Clinical Trial Design
Background:
- Novel drug development for cystic fibrosis (CF) is advancing rapidly.
- Children with CF are not simply small adults; physiological differences necessitate specific research.
- Extrapolation of adult data to pediatric populations can be inappropriate due to pharmacokinetic and safety concerns.
Purpose of the Study:
- To explore the importance and rationale for involving children in CF research.
- To discuss methodologies and challenges in conducting pediatric CF clinical trials.
- To emphasize the ethical imperative for evidence-based pediatric therapies.
Main Methods:
- Review of ethical and practical considerations in pediatric research.
- Discussion of appropriate outcome measure selection and co-design principles.
- Exploration of staffing, resourcing, and consent/assent procedures for pediatric participants.
Main Results:
- Children have a legal and ethical right to evidence-based treatments.
- Pediatric medication pharmacokinetics and adverse event profiles may differ significantly from adults.
- Co-design and appropriate methodology are vital for successful pediatric research.
Conclusions:
- Relying solely on adult study extrapolation for pediatric CF care is unethical.
- Dedicated pediatric research is essential for understanding and treating CF in children.
- Research should be integrated as a standard component of pediatric healthcare.
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.
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