Key Considerations when Conducting Rare Disease Paediatric Trials

When conducting rare disease paediatric trials, understanding, and integrating the multitude of varied regulations and ethical considerations into the design of the study continues to be essential. However, the experience and protection of children within trials, as well as the quality of data gathered to facilitate approval is dependent upon the operational delivery of the study itself. Within this whitepaper we would like to share with you some of the learning, considerations, and insights that we have gained through many years of experience delivering paediatric trials in rare disease, with the hope that this sparks healthy debate and further roll-out of optimised trials.

What’s Inside:


  • Discussion around the most efficient way to create a successful protocol.
  • Recruitment and retention considerations to take into account when enrolling patients.
  • Informed consent processes.
  • Example trial communication activities.
  • Child-first approach to trial delivery.

If you have any questions while reading this whitepaper or would like additional information on orphan reach’s expertise, feel free to request a consultation.

    Do you have a clinical trial currently at risk? YesNo

    Do you have an upcoming clinical trial? YesNo