Stopping guidelines for an effectiveness trial: what should the protocol specify?

Jon E Tyson; Claudia Pedroza; Dennis Wallace; Carl D'Angio; Edward F Bell; Abhik Das

doi:10.1186/s13063-016-1367-4

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Stopping guidelines for an effectiveness trial: what should the protocol specify?

Journal article

Open access

Peer reviewed

Stopping guidelines for an effectiveness trial: what should the protocol specify?

Jon E Tyson, Claudia Pedroza, Dennis Wallace, Carl D'Angio, Edward F Bell and Abhik Das

Trials, Vol.17(1), pp.240-240

05/10/2016

DOI: 10.1186/s13063-016-1367-4

PMCID: PMC4862046

PMID: 27165260

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Abstract

Despite long-standing problems in decisions to stop clinical trials, stopping guidelines are often vague or unspecified in the trial protocol. Clear, well-conceived guidelines are especially important to assist the data monitoring committees for effectiveness trials. To specify better stopping guidelines in the protocol for such trials, the clinical investigators and trial statistician should carefully consider the following kinds of questions: 1. How should the relative importance of the treatment benefits and hazards be assessed? 2. For decisions to stop a trial for benefit: (a) What would be the minimum clinically important difference for the study population? (b) How should the probability that the benefit exceeds that difference be assessed? (c) When should the interim analyses include data from other trials? (d) Would the evidence meet state-of-the-art standards for treatment recommendations and practice guidelines? 3. Should less evidence be required to stop the trial for harm than for benefit? 4. When should conventional stopping guidelines for futility be used for comparative effectiveness trials? Both clinical and statistical expertise are required to address such challenging questions for effectiveness trials. Their joint consideration by clinical investigators and statisticians is needed to define better stopping guidelines before starting the trial.

Data Interpretation, Statistical

Clinical Trials as Topic - statistics & numerical data

Risk Assessment

Practice Guidelines as Topic - standards

Humans

Early Termination of Clinical Trials - statistics & numerical data

Patient Safety - standards

Treatment Failure

Medical Futility

Clinical Trials Data Monitoring Committees - standards

Research Design - standards

Early Termination of Clinical Trials - standards

Clinical Trials as Topic - standards

Research Design - statistics & numerical data

Clinical Trials Data Monitoring Committees - statistics & numerical data

Details

Title: Subtitle: Stopping guidelines for an effectiveness trial: what should the protocol specify?
Creators: Jon E Tyson - Center for Clinical Research and Evidence-Based Medicine, Department of Pediatrics, University of Texas Health Science Center at Houston, 6431 Fannin St, MSB 2.106, Houston, TX, 77030, USA. jon.e.tyson@uth.tmc.edu
Claudia Pedroza - Center for Clinical Research and Evidence-Based Medicine, Department of Pediatrics, University of Texas Health Science Center at Houston, 6431 Fannin St, MSB 2.106, Houston, TX, 77030, USA
Dennis Wallace - RTI International, Durham, NC, 27709, USA
Carl D'Angio - University of Rochester School of Medicine and Dentistry, Rochester, NY, 14627, USA
Edward F Bell - Department of Pediatrics, University of Iowa, Iowa City, IA, 52242, USA
Abhik Das - Social, Statistical and Environmental Sciences Unit, RTI International, Durham, NC, 27709, USA
Resource Type: Journal article
Publication Details: Trials, Vol.17(1), pp.240-240
DOI: 10.1186/s13063-016-1367-4
PMID: 27165260
PMCID: PMC4862046
NLM abbreviation: Trials
ISSN: 1745-6215
eISSN: 1745-6215
Grant note: UG1 HD053109 / NICHD NIH HHS U10 HD036790 / NICHD NIH HHS UG1 HD027853 / NICHD NIH HHS UG1 HD068263 / NICHD NIH HHS
Language: English
Date published: 05/10/2016
Academic Unit: Stead Family Department of Pediatrics; Neonatology
Record Identifier: 9984093356402771

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