Clinical trials for neurodegenerative diseases (NDDs) face challenges due to patient variability in symptoms and progression, requiring therapies that effectively cross the blood-brain barrier and target specific areas. Biomarkers, such as fluid, digital, and imaging-based, help create enrichment strategies to stratify patients, enable early diagnosis, and predict treatment responses. Despite the utility, successfully employing these methods requires careful planning and a deep understanding of the field.
This white paper explores strategies and considerations for successfully implementing precision medicine and biomarker-based assessments into NDD trials. Within, we discuss:
- The multifaceted role biomarkers serve across the NDD clinical spectrum
- Participant enrichment and stratification
- Biomarkers as key primary and secondary endpoints
- Clinical development strategies with biomarkers and precision medicine
