Defining Success in Rare Disease Pediatric Trials

Success means different things to different people.

Pediatric trials for rare diseases are crucial. They play a vital role in developing future treatments and targeting the specific needs of pediatric patients, allowing them to experience a positive childhood with as much normal development as possible.

As highlighted in our previous paper, "Thinking of the Individual," these trials are not without challenges. Each individual is affected differently by the condition, making mainstream trial design, analysis, and real-world data methods less than optimal. Collaboration among all stakeholders is essential to conduct feasible studies and develop treatments that truly matter to those who need them.

What’s Inside?

• In this whitepaper we discuss the complexities of agreeing the definitions and approaches used to measure trial success that are meaningful for all involved. Experts might imply we are missing out the family/patient voice?
• What success looks like to different stakeholders within the clinical trial landscape
• The traditional approach - a look into the impact of PREMs (Patient Reported Experience Measures) and PROMs (Patient Reported Outcome Measures)
• An exploration of early-stage development of new measures
•  Valuable insights related to designing endpoints that matter to patients and families.