Lessons Learned from Drug Development Programs in Autism: Implications for Future Programs

There are no approved drugs for autism core features; the heterogeneous biology, symptom presentation, and clinical outcomes complicate drug trials design, and have hampered therapeutic drug development. We synthesized expert viewpoints from industry and academia on recent diagnostics and biomarker advances as well as pharmacotherapy evidence, aligned to patient-focused drug development outcomes and key concepts. Key learnings include: (i) measure what matters to patients and caregivers; (ii) enrich populations where they can be rationally matched to mechanism; (iii) do not use diagnostic tools as efficacy endpoints; (iv) align biomarker science with a potential qualification pathway associated with relevant behavior and biology; (v) design trials that reduce placebo response, burden, and attrition; (vi) assess co-occurring conditions and treatments explicitly. Observations and recommendations serve as a practical roadmap for sponsors and clinicians to increase trial informativeness and chance of success.