**

Why You Should Listen

** **If you work in clinical trials or healthcare analytics, this episode will help you: **

• Understand why patient perspectives are now central—not optional
• Learn the difference between PROs and COAs, and when each matters
• See how questionnaires are scientifically developed and validated
• Improve how you select, analyze, and interpret endpoints
• Avoid common pitfalls when using patient-reported data

Episode Highlights

• 00:00 – Introduction and background to the episode
• 01:31 – Rachael’s journey into patient-centered outcomes
• 03:24 – Why PROs have become central to drug development
• 05:18 – What COAs are and how they extend beyond PROs
• 05:46 – The four types of COAs explained
• 08:58 – Generic vs disease-specific questionnaires
• 13:16 – How PRO instruments are developed and validated
• 15:06 – What “meaningful change” really means
• 17:02 – Using Phase II data to inform Phase III endpoints
• 18:24 – FDA expectations and guidance on PROs
• 21:47 – Using the estimand framework for patient-centered endpoints
• 23:15 – What “psychometrically sound” actually means
• 31:16 – How I would choose the right PRO or COA for a trial
• 34:01 – Avoiding missing data and reducing patient burden
• 37:18 – Key takeaways on interpreting patient-reported data

About the Guest

Rachael Lawrance Senior Director & Functional Lead Statistics, Patient-Centered Outcomes (PCO), Adelphi Values

Rachael is a highly experienced statistician specializing in patient-centered outcomes. She works across the full lifecycle of PRO development—from qualitative research and questionnaire design to statistical validation and interpretation.

With a background in pharma and consultancy, she focuses on bridging the gap between rigorous statistical methodology and meaningful patient insights.

🔗 Links & References

• Adelphi Values
• The Effective Statistician
• PSI Conference 2026
• FDA Patient-Focused Drug Development (PFDD) Guidance Series
• PRO Consortium (PRO-C) resources