By nature, pediatric rare disease studies require unique planning. Meaningfully testing subjects who are highly heterogeneous necessitates strategic trial design and targeted assessment selection. Even small changes in behavior or function are critical and need to be detected.
Test selection is a key component of pediatric rare disease clinical trial development. Assessments need to be appropriate for the functioning level of the population and also have adequate sensitivity and specificity to detect change over time in the target population.
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