Assessing predictive probability of success for future clinical trials

Data-driven decision-making is crucial in drug development, with the predictive probability of success (PoS) being a key quantitative tool. PoS estimates the likelihood of success of a future trial based on the same or surrogate endpoint(s) of interest, utilizing information from interim analyses, or completed historical studies. While it has been extensively studied and broadly applied in clinical practice, there is a growing need of a unified approach for PoS that can effectively incorporate information from surrogate endpoints and multiple historical studies. This paper investigates and assesses a unified Bayesian approach for PoS. We first review PoS based on historical data on the same endpoint and then extend it to include information from a surrogate endpoint with a closed-form solution. Additionally, we utilize a Bayesian meta-analytic approach to incorporate data from multiple historical studies. We illustrate the unified approach with examples from oncology and immunology trials and provide an R package "PPoS" for practical implementation. By integrating the assessment of PoS with information from surrogate endpoints and historical studies, we aim to enhance the decision-making process in drug development.

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Publication Details

Journal
Journal of Biopharmaceutical Statistics
Published
2025-06-16
DOI
https://doi.org/10.1080/10543406.2025.2510262
Primary Topic
Health Systems, Economic Evaluations, Quality of Life
Type
article
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article

Assessing predictive probability of success for future clinical trials

Archie Sachdeva, Ram C. Tiwari, Ming Zhou
Journal of Biopharmaceutical Statistics
Health Systems, Economic Evaluations, Quality of Life
article

Assessing predictive probability of success for future clinical trials

Archie Sachdeva, Ram C. Tiwari, Ming Zhou
article en

Abstract

Data-driven decision-making is crucial in drug development, with the predictive probability of success (PoS) being a key quantitative tool. PoS estimates the likelihood of success of a future trial based on the same or surrogate endpoint(s) of interest, utilizing information from interim analyses, or completed historical studies. While it has been extensively studied and broadly applied in clinical practice, there is a growing need of a unified approach for PoS that can effectively incorporate information from surrogate endpoints and multiple historical studies. This paper investigates and assesses a unified Bayesian approach for PoS. We first review PoS based on historical data on the same endpoint and then extend it to include information from a surrogate endpoint with a closed-form solution. Additionally, we utilize a Bayesian meta-analytic approach to incorporate data from multiple historical studies. We illustrate the unified approach with examples from oncology and immunology trials and provide an R package "PPoS" for practical implementation. By integrating the assessment of PoS with information from surrogate endpoints and historical studies, we aim to enhance the decision-making process in drug development.

Journal of Biopharmaceutical Statistics
Bristol-Myers Squibb (Germany) (DE)
Openalex Percentile: Top 80%
Health Systems, Economic Evaluations, Quality of Life
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Assessing predictive probability of success for future clinical trials — Archie Sachdeva, Ram C. Tiwari, et al. · Journal of Biopharmaceutical Statistics (2025) | TGRS Research Map | TGRS