Evaluation of New Treatments in Radiation Oncology

Soares, Heloisa P.; Kumar, Amit; Daniels, Stephanie; Swann, S.; Cantor, Alan; Hozo, Iztok; Clark, Mike; Serdarević, Fadila; Gwede, Clement K.; Trotti, Andy; Djulbegoviĉ, Benjamin

doi:10.1001/jama.293.8.970

Cited by 78 publications

(25 citation statements)

References 36 publications

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“…1,4 Our findings were similar, showing an overall publication rate of 97%, which did not statistically significantly differ according to whether trial results were positive or negative. Earlier observations about the potential for publication bias in clinical trials, and the subsequent requirement that all NIH-funded clinical trials be registered through clinicaltrials.gov, has likely helped to reduce publication bias in NCI-sponsored cooperative group trials.…”

Section: Discussionsupporting

confidence: 72%

“…1–4 But the comparative scientific impact of positive versus negative trials using citation data has not been investigated. In this paper, we utilize the phase III trial database of SWOG, a major national cooperative group, in combination with its trial publication database and citation data from Google Scholar, to compare the scientific impact of positive and negative cancer clinical trials.…”

Section: Introductionmentioning

confidence: 99%

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The Scientific Impact of Positive and Negative Phase 3 Cancer Clinical Trials

et al. 2016

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Importance Positive Phase III cancer clinical trials are widely hailed, while negative trials are often interpreted as scientific failures. We hypothesized that these interpretations would be reflected in the scientific literature. Objective To compare the scientific impact of positive versus negative phase III cancer clinical treatment trials. Design We examined the phase III trial history of SWOG over a 30-year period (1985–2014). Scientific impact was assessed according to multiple publication and citation outcomes. Citation data were obtained using Google Scholar™. Citation counts were compared using generalized estimating equations for Poisson regression. Setting SWOG, a national cancer clinical trials consortium. Participants Any trial that was formally evaluated for the randomized treatment comparison was included for analysis of publication and citation outcomes. Exposures Trials were categorized as positive if they achieved a statistically significant result in favor of the new treatment for the protocol-specified primary endpoint, and negative otherwise. Main Outcomes The main outcomes were the impact factors for the journals publishing the primary trial results, and the number of citations for the primary trial manuscripts and all secondary manuscripts associated with the trials. Results Ninety-four studies enrolling n=46,424 patients were analyzed. Twenty-eight percent of trials were positive (26/94). The primary publications from positive trials were published in journals with higher average impact factors (28.4 vs. 17.5, p=.007) and were cited twice as often as negative trials (average per year, 43 vs. 21, p=.03). However, the number of citations from all primary and secondary manuscripts did not differ between positive and negative trials (average per year, 55 vs. 45, p=.53). Conclusions and Relevance The scientific impact of the primary manuscripts from positive phase III randomized cancer clinical trials was twice as great as for negative trials. But when all of the manuscripts associated with the trials were considered, the scientific impact between positive and negative trials was similar. Positive trials indicate clinical advances, but negative trials also have a sizeable scientific impact by generating important scientific observations and new hypotheses and by showing what new treatments should not be used.

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Section: Discussionsupporting

confidence: 72%

Section: Introductionmentioning

confidence: 99%

The Scientific Impact of Positive and Negative Phase 3 Cancer Clinical Trials

et al. 2016

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“…For example, half or more of the drugs tested in large, late phase III trials show higher effectiveness against older comparators (H 0 :H 1 = <1; Soares et al, 2005). Conversely, the vast majority of tested hypotheses in large-scale exploratory research reflect null effects, e.g., in the search of genetic variants associated with various diseases in the candidate gene era where investigators were asking hypotheses one or a few at a time (the same way that investigators continue to test hypotheses in most other biomedical and social science fields) yielded thousands of putative discovered associations, but only 1.2% of them were subsequently validated to be non-null when large-scale consortia with accurate measurements and rigorous analyses plans assessed them (Chanock et al, 2007;Ioannidis et al, 2011).…”

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confidence: 99%

When null hypothesis significance testing is unsuitable for research: a reassessment

Szűcs

Ioannidis

2016

Preprint

107

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Null hypothesis significance testing (NHST) has several shortcomings that are likely contributing factors behind the widely debated replication crisis of (cognitive) neuroscience, psychology, and biomedical science in general. We review these shortcomings and suggest that, after sustained negative experience, NHST should no longer be the default, dominant statistical practice of all biomedical and psychological research. If theoretical predictions are weak we should not rely on all or nothing hypothesis tests. Different inferential methods may be most suitable for different types of research questions. Whenever researchers use NHST they should justify its use, and publish pre-study power calculations and effect sizes, including negative findings. Hypothesis-testing studies should be pre-registered and optimally raw data published. The current statistics lite educational approach for students that has sustained the widespread, spurious use of NHST should be phased out.

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“…experimentellen) Interventionen im Bereich der Onkologie (anhand von öf-fentlich geförderten Phase-III-Studien, Nicht-Unterlegenheitsstudien wurden ausgeschlossen) belegen sehr eindrucksvoll, dass echte «Durchbrüche» in der Therapie wohl eher die seltene Ausnahme sind, die kleinen Verbesserungen somit die Regel [13][14][15]. In der größten Serie von 624 RCT mit 216 451 Patienten wurde in gerade mal 2% der Studien eine Verbesserung der Überlebenszeit im experimentellen Arm in der Größenordnung eines Hazard Ratios für Tod von 0,5 oder weniger beobachtet [13].…”

Section: Bedingte Erstattungsfähigkeitunclassified

Bedeutung von klinischen Studien für die Entscheidungsfindung in den Gremien der Selbstverwaltung

Lange

2010

Onkologie

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Klinische Studien sind die zentrale Basis für die Entscheidungsfindung in den Gremien der Selbstverwaltung für Fragen der Erstattungsfähigkeit medizinischer Leistungen. Für viele dieser zur Entscheidung anstehenden Fragen fehlen jedoch Studien ausreichender Ergebnissicherheit. Wenngleich die Selbstverwaltung über einige Instrumente verfügt, die Durchführung klinischer Studien zu initiieren (z.B. Aussetzungsbeschluss des Gemeinsamen Bundesausschusses) bzw. diese zu fördern (§ 35c SGB V), sind die Möglichkeiten dennoch begrenzt, diesem Defizit selbst abzuhelfen. Eine weitergehende und stabile Förderung und Finanzierung des Aufbaus von Strukturen zur Durchführung klinischer Studien, die unabhängig von Partikularinteressen (z.B. der Pharmaindustrie) sind, ist weiterhin dringend erforderlich.

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Evaluation of New Treatments in Radiation Oncology

Cited by 78 publications

References 36 publications

The Scientific Impact of Positive and Negative Phase 3 Cancer Clinical Trials

The Scientific Impact of Positive and Negative Phase 3 Cancer Clinical Trials

When null hypothesis significance testing is unsuitable for research: a reassessment

Bedeutung von klinischen Studien für die Entscheidungsfindung in den Gremien der Selbstverwaltung

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