CASE STUDY
Redefining Pain Episodes in Sickle Cell Disease
A Patient-Informed Measurement Strategy Case Study
Sickle cell disease (SCD) trials often rely on healthcare utilization and single-score pain scales to define treatment effect. But many pain episodes are managed at home and shaped by personal baseline, context, and lived experience.
This case study explores how a patient-informed measurement strategy reframed how sickle cell pain episodes can be defined, modeled, and measured across home and clinical settings.
See how grounding endpoint design in disease biology and lived experience can improve signal clarity and strengthen decision-making in sickle cell trials.
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What You'll Learn
A patient-defined model identifying four configurations of sickle cell pain episodes
How baseline pain tolerance and escalation thresholds influence care-seeking
Implications for designing more sensitive, interpretable endpoints in sickle cell and other pain trials
Implications for designing clearer, more sensitive endpoints in pain programs
Why disease-specific diaries and passive data collection may improve feasibility and engagement
