Table 1 Seven characteristics of useful PD endpoints

1. Meaningfully describe the patient’s pharmacological and clinical responses to drug therapy with respect to

(a) Incorporating both harms and benefits

(b) Accounting for patient and families well-being (quality of life)

2. Can be interpreted against data extrapolated from other diseases or age-groups and existing scientific literature

3. Can be used to answer the research question while informing healthcare decision making at the bedside and policy level

4. Is responsive to change and comes with a defined age-specific minimally important difference

5. Is reproducible and, where possible, objective

6. Can be consistently and reliably measured by outcome assessors

7. Has an established age-appropriate validated measure with established reference ranges in the specific age group and disease state

8. Is feasible with respect to

(a) Acceptability in terms of burden on the child or caregivers with minimal compliance/adherence concerns

(b) Timing: where possible combined with routine tests

(c) Cost considerate: license, equipment, and skill set of the outcome assessor