Presentation of Nonfinal Results of Randomized Controlled Trials at Major Oncology Meetings

Booth, Christopher M.; Maître, Aurélie Le; Ding, Keyue; Farn, K T; Fralick, Michael; Phillips, Catherine; Cescon, David W.; Meyer, Ralph M.

doi:10.1200/jco.2008.18.8771

Cited by 30 publications

(17 citation statements)

References 16 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…That is, unlike formal inferential processes, the investigator’s global judgment is content and context dependent[32]. Of interest, recently Booth et al [33] analyzed differences between conclusions of the abstracts of RCTs presented at major oncology meetings with the subsequent final analyses published in full papers. They found that in about 10 percent of the trials, there was a reversal in the researchers’ conclusions about the efficacy of treatment when the abstracts were compared with the final reports.…”

Section: Discussionmentioning

confidence: 99%

Optimism bias leads to inconclusive results—an empirical study

Djulbegoviĉ

Kumar

Magazin

et al. 2011

Journal of Clinical Epidemiology

View full text Add to dashboard Cite

Objective Optimism bias refers to unwarranted belief in the efficacy of new therapies. We assessed the impact of optimism bias on a proportion of trials that did not answer their research question successfully, and explored whether poor accrual or optimism bias is responsible for inconclusive results. Study Design Systematic review Setting Retrospective analysis of a consecutive series phase III randomized controlled trials (RCTs) performed under the aegis of National Cancer Institute Cooperative groups. Results 359 trials (374 comparisons) enrolling 150,232 patients were analyzed. 70% (262/374) of the trials generated conclusive results according to the statistical criteria. Investigators made definitive statements related to the treatment preference in 73% (273/374) of studies. Investigators’ judgments and statistical inferences were concordant in 75% (279/374) of trials. Investigators consistently overestimated their expected treatment effects, but to a significantly larger extent for inconclusive trials. The median ratio of expected over observed hazard ratio or odds ratio was 1.34 (range 0.19 – 15.40) in conclusive trials compared to 1.86 (range 1.09 – 12.00) in inconclusive studies (p<0.0001). Only 17% of the trials had treatment effects that matched original researchers’ expectations. Conclusion Formal statistical inference is sufficient to answer the research question in 75% of RCTs. The answers to the other 25% depend mostly on subjective judgments, which at times are in conflict with statistical inference. Optimism bias significantly contributes to inconclusive results.

show abstract

Section: Discussionmentioning

confidence: 99%

Optimism bias leads to inconclusive results—an empirical study

Djulbegoviĉ

Kumar

Magazin

et al. 2011

Journal of Clinical Epidemiology

View full text Add to dashboard Cite

show abstract

“…Preliminary data should not be made public for epidemiologic and statistical reasons (40,41), which primarily relate to the statistical instability of early results and the potential for nonfinal analyses to misinform clinical practice. In a recent overview of 138 oncology RCTs published between 2000 and 2004, 44% of related conference abstracts used language to imply that the data were nonfinal and 63% of abstracts contained important data discordance when compared with the final publication (42). Furthermore, when compared with published articles, authors' conclusions were substantively discordant in 10% of abstracts.…”

Section: Reporting Of Trials and Avoidance Of Biasmentioning

confidence: 95%

Evaluating Patient-Centered Outcomes in the Randomized Controlled Trial and Beyond: Informing the Future with Lessons from the Past

Booth

2010

Clinical Cancer Research

Self Cite

View full text Add to dashboard Cite

In the era of molecular oncology, patients still define a useful therapy as one that allows them to live longer and helps them to live better. Although patient outcomes have clearly improved as a result of randomized controlled trials (RCT), it is critical that contemporary trials retain the perspective of these fundamental patient-centered outcomes. Trends in study design, results, and interpretation of oncology RCTs from the past provide a useful framework in which to consider how the research community may approach trial design in the future. Although the RCT remains the standard for establishing efficacy, this article also considers how population-based outcome studies can provide insight into effectiveness of new therapies and explores how the results of RCTs translate into benefit in the general population. Clin Cancer Res; 16(24); 5963-71. Ó2010 AACR.Although patient outcomes have improved as a result of clinical trials conducted since the 1970s, in the contemporary era, patients still define a useful therapy as one that improves the quality, or increases the quantity, of survival. Despite considerable change in the treatment and outcome of patients with cancer, it is critical that investigators, clinicians, and policy makers not lose sight of these fundamental patient-centered outcomes. Furthermore, although the randomized controlled trial (RCT) remains the gold standard for establishing efficacy of new therapies, increased effort is required to understand how the results of RCTs influence clinical practice and to assess the benefit of new treatments in the general population. This article explores how the RCT in oncology has evolved over time and proposes issues that require consideration in the design and interpretation of future trials. The article also considers how population-based outcome studies can provide insight into the uptake of new medical therapies and explores how the results of RCTs translate into benefit in the "real world." Evolution of Design and Outcomes of Oncology Randomized Controlled TrialsA recent overview of 321 RCTs of systemic therapy in breast, colorectal, and non-small cell lung cancer (NSCLC), published between 1975 and 2004, provides insight into the methods, results, and outcomes of oncology trials over time (Table 1; ref. 1). RCTs in oncology have become larger and increasingly involve multiple international centers. Although the median sample size of RCTs has increased from 100 to 446 patients per study, the total duration of study accrual for these trials has remained relatively stable since the 1970s. During the study period, encouraging changes occurred in the statistical methodology and endpoints in RCTs. The primary endpoint of oncology RCTs has shifted away from response rate to survival and other time-to-event endpoints. The proportion of trials using intention-to-treat (ITT) analyses has also increased. Despite these improvements, in the most recent decade (1995 to 2004), one third of systemic therapy RCTs published in high-impact and widely read journa...

show abstract

“…However, there is evidence to suggest that there are frequently discrepancies in the results and conclusions between conference abstracts and subsequent corresponding published papers [8,9]. Clinicians are advised not to hurry and incorporate the findings presented at conferences in to clinical decisionmaking but rather to wait for the publication of the complete manuscript in a scientific journal [10].…”

Section: Issemination Of Findings Is a Pivotal Goal Of Anymentioning

confidence: 98%

Knowledge Dissemination Resulting from the Australian Cardiac Rehabilitation Association Annual Scientific Meetings

Fernandez

Rowe

Redfern

et al. 2011

Heart, Lung and Circulation

View full text Add to dashboard Cite

Presentation of Nonfinal Results of Randomized Controlled Trials at Major Oncology Meetings

Abstract: Meeting abstracts often include NFAs and are frequently discordant with subsequent article publication.

Cited by 30 publications

References 16 publications

Optimism bias leads to inconclusive results—an empirical study

Optimism bias leads to inconclusive results—an empirical study

Evaluating Patient-Centered Outcomes in the Randomized Controlled Trial and Beyond: Informing the Future with Lessons from the Past

Knowledge Dissemination Resulting from the Australian Cardiac Rehabilitation Association Annual Scientific Meetings

Contact Info

Product

Resources

About