Rigor in Monitoring Clinical Trials Is Ethical

Piantadosi, Steven

doi:10.1200/jco.2007.15.5226

Cited by 5 publications

(5 citation statements)

References 4 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The key strength of this study design is its robust ability to reduce potential bias that may lead to inaccurate results. Proponents of RCTs tend to praise both the statistical and ethical virtues of this approach (Piantadosi, 2008), while critics have concerns that relate primarily to what they perceive to be ethical flaws (Wells, 2008). Although some may disagree, the fundamental ethical requirement for the conduct of RCTs is generally considered to be that current evidence supports a condition of equipoise: i.e., there is no clear evidence which exists with respect to the superiority of the two treatment options within an RCT (Freedman, 1987; Gifford, 2007; Miller & Brody, 2003).…”

Section: The Randomized Clinical Trialmentioning

confidence: 99%

Both Sides of the Coin: Randomization from the Perspectives of Physician-Investigators and Patient-Subjects

2010

View full text Add to dashboard Cite

Randomization is the "gold standard" design for clinical research trials, and is accepted as the best way to reduce bias. Although some controversy remains over this matter, we believe equipoise is the fundamental ethical requirement for conducting a randomized clinical trial. Despite much attention to the ethics of randomization, the moral psychology of this study design has not been explored. This paper analyzes the ethical tensions that arise from conducting these studies, and examines the moral psychology of this design from the perspectives of physician-investigators and patient-subjects. We conclude with a discussion of the practical implications of this analysis.

show abstract

Section: The Randomized Clinical Trialmentioning

confidence: 99%

Both Sides of the Coin: Randomization from the Perspectives of Physician-Investigators and Patient-Subjects

2010

View full text Add to dashboard Cite

show abstract

“…3,16 Wells 16 argued that in laboratory research and clinical education, open discussion of preliminary ideas and concepts are commonplace and that availability of preliminary results facilitates meeting federal regulations governing the open exchange of research information and the ethical parameters to inform study investigators and participants. 3,16 Wells 16 argued that in laboratory research and clinical education, open discussion of preliminary ideas and concepts are commonplace and that availability of preliminary results facilitates meeting federal regulations governing the open exchange of research information and the ethical parameters to inform study investigators and participants.…”

Section: ‫ء‬mentioning

confidence: 99%

“…[1][2][3] Presentations of preliminary results may also not acknowledge the statistical implications associated with re-peated analyses. Because abstracts have an abbreviated format and undergo a different peer-review process than articles, assessment of their methodologic quality may be limited.…”

Section: Introductionmentioning

confidence: 99%

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

Booth

Maître

Ding

et al. 2009

JCO

View full text Add to dashboard Cite

show abstract

“…Termination based on a planned interim analysis is quite different to informal reporting of preliminary analyses at major cancer meetings. 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).…”

Section: Reporting Of Trials and Avoidance Of Biasmentioning

confidence: 99%

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

Booth

2010

Clinical Cancer Research

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

Rigor in Monitoring Clinical Trials Is Ethical

Cited by 5 publications

References 4 publications

Both Sides of the Coin: Randomization from the Perspectives of Physician-Investigators and Patient-Subjects

Both Sides of the Coin: Randomization from the Perspectives of Physician-Investigators and Patient-Subjects

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

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

Contact Info

Product

Resources

About