Bayesian Efficacy Monitoring Via Posterior Probability

J. Jack Lee, Ying-Wei Kuo, Diane Liu and Nan Chen

Department of Biostatistics, MD Anderson Cancer Center, Houston, TX 77030

PID:900; v1.1.4.0 ; Last Updated: 10/19/2020

Calculate Stopping Boundaries

Trial Name (Optional)

Efficacy Parameters:

Probability of Response (\(\theta\))

Early Stopping for Futility

Criterion for futility stopping:

Stop for futility if

\(\textit{Prob}\) (\(\theta \le \theta_{fut}\)) \(> P_{fut}\)

Reference response rate for futility monitoring (\(\theta_{fut}\))

Probability confidence threshold for futility stopping (\(\textit{P}_{fut}\))

Early Stopping for Efficiacy

Criterion for declaring efficacy early:

Stop for efficacy if

\(\textit{Prob}\) (\(\theta > \theta_{eff}\)) \(\ge P_{eff}\)

Reference response rate for efficacy monitoring (\(\theta_{eff}\))

Probability confidence threshold for claiming efficacy early (\(\textit{P}_{eff}\))

Criterion for declaring efficacy:

Declare efficacy if

\(\textit{Prob}\) (\(\theta > \theta_{eff.final}\)) \(\ge P_{eff.final}\)

Reference response rate for final decision
(\(\theta_{eff.final}\))

Probability confidence threshold for claiming efficacy(\(\textit{P}_{eff.final}\))

Prior distribution for \(\theta\): \(\textit{Beta}\)(\(a_0, b_0\))

\(a_0\):

\(b_0\):

Sample size and cohort size:

Input Cohorts Manually

Maximum number of patients in the trial

Minimum number of patients treated before early stopping rule applies

Cohort size

Cohorts for interim analysis: (separate by a comma)

Upload Input file (.csv format): [Sample input file]

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Save Input into a csv file

Filename

Download PDF Report
Download EXCEL Report

Operating Characteristics

Trial Name (Optional)

Scenarios:

(separate by a comma)

Response Rates (in ascending order):

Graph Parameters:

Auto Generated Graph Parameters

(separate by a comma)

Color:

Density:

Angle:

Line Type:

Filename

Save Input into a csv file

Download PDF Report Download Excel Report

Trial Monitoring

Trial Name (Optional)

Number of Patients

Seq#

Patient ID

Resp. Outcome

1

2

Save Input into a csv file

Filename

Download PDF Report Download Excel Report

Bayesian Efficacy Monitoring Via Posterior Probability

J. Jack Lee, Ying-Wei Kuo, Diane Liu and Nan Chen

Department of Biostatistics, MD Anderson Cancer Center, Houston, TX 77030

PID:900; v1.1.4.0 ; Last Updated: 10/19/2020

Calculate Stopping Boundaries

Efficacy Parameters:

Probability of Response (\(\theta\))

Criterion for futility stopping:

Stop for futility if

\(\textit{Prob}\) (\(\theta \le \theta_{fut}\)) \(> P_{fut}\)

Reference response rate for futility monitoring (\(\theta_{fut}\))

Probability confidence threshold for futility stopping (\(\textit{P}_{fut}\))

Criterion for declaring efficacy early:

Stop for efficacy if

\(\textit{Prob}\) (\(\theta > \theta_{eff}\)) \(\ge P_{eff}\)

Reference response rate for efficacy monitoring (\(\theta_{eff}\))

Probability confidence threshold for claiming efficacy early (\(\textit{P}_{eff}\))

Criterion for declaring efficacy:

Declare efficacy if

\(\textit{Prob}\) (\(\theta > \theta_{eff.final}\)) \(\ge P_{eff.final}\)

Reference response rate for final decision (\(\theta_{eff.final}\))

Probability confidence threshold for claiming efficacy(\(\textit{P}_{eff.final}\))

Prior distribution for \(\theta\): \(\textit{Beta}\)(\(a_0, b_0\))

\(a_0\):

\(b_0\):

Sample size and cohort size:

Maximum number of patients in the trial

Minimum number of patients treated before early stopping rule applies

Cohort size

Cohorts for interim analysis: (separate by a comma)

Upload Input file (.csv format): [Sample input file]

Go to Operating Characteristics ⇒

Operating Characteristics

Scenarios:

(separate by a comma)

Response Rates (in ascending order):

Graph Parameters:

(separate by a comma)

Color:

Density:

Angle:

Line Type:

Trial Monitoring

Number of Patients

Seq#

Patient ID

Resp. Outcome

1

2

Support Document

Reference response rate for final decision
(\(\theta_{eff.final}\))