Methods and Novel Endpoints (Patient-Reported Outcomes & Digital Health Data) in Biomedical Studies

Wed, Aug 5, 8:30 AM - 10:20 AM Room CC-206B Thomas M. Menino Convention & Exhibition Center

Steven GilbertChair

Biopharmaceutical Section

6 Presentations

Cumulative Live Birth After Planned Egg Freezing: A Comparison of Three Predictive Approaches

8:35 AM - 8:50 AM

Katharine Correia (Amherst College)

Co-authors: Leontine Alkema (University of Massachusetts-Amherst), Rachel Weinerman (University Hospitals Fertility Center), Kathryn Coyne (University Hospitals Fertility Center)

discrete-time survival analysis random effects logistic regression frailty Bayesian hierarchical model right-censored data discrete-time hazards

A CV-TMLE Global Test Approach to Multiple-Component Endpoints in Rare Disease Clinical Trials

8:50 AM - 9:05 AM

Tianyue Zhou

Co-authors: Susan Gruber, Mark Van Der Laan (UC Berkeley), Hana Lee (Food and Drug Administration), Wonyul Lee (FDA Center for Drug Evaluation and Research (CDER)), Lei Nie (The US FDA)

CV-TMLE global test composite outcome rare disease trials

Analyzing Daily Digital Health Data in Clinical Trials Using Two-level MMRM & Functional Methods

9:05 AM - 9:20 AM

Liuqing (Jasmine) Yang (AbbVie)

Co-authors: Man Jin (AbbVie), Xin Huang (AbbVie), Dan Webster (AbbVie), Jie Shen (AbbVie)

Daily outcomes Digital health MMRM Functional data analysis

Assessing Responder Analyses for Patient-Reported Outcomes: Maximum Responder Threshold and Effect

9:20 AM - 9:35 AM

Joseph Cappelleri (Pfizer Inc)

measurement scale minimum clinically important difference meaningful within-patient change meaningful score difference interpretation

Endpoint Sensitivity in Chronic Pain Studies

9:35 AM - 9:50 AM

Zhangchen Zhao

Co-authors: Ixavier Higgins (Eli Lilly), Yushi Liu (Eli Lilly), Phebe Kemmer (Eli Lilly and Company)

Early‑phase study design Endpoint selection Latent factor analysis Multivariate copula simulation Bootstrap power assessment Early‑phase clinical trials

Statistical Analysis on Ambulatory Blood Pressure Monitoring (ABPM)

9:50 AM - 10:05 AM

Hongqi Xue (Clario)

ambulatory blood pressure monitoring assessment central tendency analysis categorical outlier analysis concentration-ABPM analysis