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Exploratory Analysis of the Factors Associated With Success Rates of Confirmatory Randomized Controlled Trials in Cancer Drug Development.

ABSTRACT: This study examined the outcomes of recent confirmatory randomized controlled trials (RCTs) in phase III that were initiated between 2005 and 2017 for oncologic drugs in the United States and identified several factors that were associated with the success of RCTs. Our regression analysis showed that studies with progression-free survival or response rate as primary end point were more likely to succeed than studies with overall survival (odds ratio (OR) = 2.94 and 6.23, respectively). The status of development was also linked with success rates. Studies for non-lead indication tended to have lower success rates than studies for lead indication (OR = 0.68). Studies for first-line therapy were observed to have low success rates compared with studies for post second-line therapies (OR = 0.37). Studies for which strong prior evidence was not listed in their publication tended to be more successful than studies that followed rigorous RCTs or single arm studies for the indication. These results suggest that historical success rates may reflect not only the important features of trials, which can be observed directly from study design and results, but also the background status of trials in clinical development pathways.

SUBMITTER: Wu C

PROVIDER: S-EPMC7877835 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature

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Exploratory Analysis of the Factors Associated With Success Rates of Confirmatory Randomized Controlled Trials in Cancer Drug Development.

Wu Can C Ono Shunsuke S

Clinical and translational science 20200821 1

This study examined the outcomes of recent confirmatory randomized controlled trials (RCTs) in phase III that were initiated between 2005 and 2017 for oncologic drugs in the United States and identified several factors that were associated with the success of RCTs. Our regression analysis showed that studies with progression-free survival or response rate as primary end point were more likely to succeed than studies with overall survival (odds ratio (OR) = 2.94 and 6.23, respectively). The statu ...[more]

PMID: 32702190

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Project description:IntroductionTeaching procedural skills in medicine is time- and resource-intensive, and it is therefore important to determine which educational strategies are most effective. Test-enhanced learning (TEL) has been demonstrated to be effective in improving learner retention; however, there is little research evaluating the testing effect on the acquisition of procedural skills in medicine. The objective of this study was to examine the impact of TEL on learning ultrasound-guided intravenous (USG IV) insertion on a simulated model.MethodsWe conducted a prospective randomized controlled trial of medical students at a single tertiary care academic hospital. Participants were randomized to either the TEL group (TEG) or control group (CG). Each group received an USG peripheral IV teaching session that included a didactic portion and hands-on skills training. The training sessions were identical except that the TEG was informed at the outset that there would be an assessment at the end of the session. The TEG then received a formal assessment of the skill during the last 15 minutes of the session, whereas the CG had continued practice time. Subjects in both groups were evaluated 10-14 days later to compare skill performance using a simulation-based assessment tool consisting of a global rating scale (GRS) and checklist items.ResultsThirty medical students completed the study, 15 in the TEG and 15 in the CG. There were no significant differences between the two groups at baseline based on year of medical training or prior IV or ultrasound experience. The overall procedural success rate was 93.3% (95% CI = 79.0%-100.0%) in the TEG and 80.0% (95% CI = 57.1%-100%) in the CG (p = 0.60). The first-attempt failure rate was 13.3% (95% CI = 0.0%-32.8%) in the TEG and 33.3% (95% CI = 6.3%-60.4%) in the CG (p = 0.39). There were no statistically significant differences between the means of the GRS (TEG = 4.7, CG = 4.2; p = 0.53, r = 0.11) or checklist scores (TEG = 78.6%, CG = 75.3%; p = 0.20, r = 0.24).ConclusionsIn this study of novice learners, both TEL with structured practice and structured practice alone lead to high success rates performing USG IV insertion on a simulated model. While we noted a trend toward higher procedural success rates and lower first-attempt failure rates in the TEG, these did not meet statistical significance. Further studies with larger sample sizes are required to determine whether the beneficial effects of TEL can be transferred to procedural skills teaching.

Project description:BackgroundThe generalisability of randomized controlled trials (RCTs) can be uncertain because the impact of exclusion criteria is rarely quantified. The aim of this study was to systematically review studies examining the percentage of clinical populations with a physical health condition who would be excluded by RCTs of treatments for that condition.MethodsMedline and Embase were searched from inception to Feb 11th 2018. Two reviewers independently completed screening, full-text review, data extraction and risk-of-bias assessment. The primary outcome was the percentage of patients in the clinical population who would have been excluded from each examined trial. Subgroup analyses examined exclusion by population setting, publication date and funding source.ResultsTitles/abstracts (20,754) were screened, and 50 studies were included which reported exclusion rates from 305 trials of treatments in 31 physical conditions. Estimated rates of exclusion from trials varied from 0% to 100%, and the median exclusion rate was 77.1% of patients (interquartile range 55.5% to 89.0% exclusion). Median exclusion rates for trials in common chronic conditions were high, including hypertension 83.0%, type 2 diabetes 81.7%, chronic obstructive pulmonary disease 84.3%, and asthma 96.0%. The most commonly applied exclusion criteria related to age, co-morbidity and co-prescribing, whereas more implicit criteria relating to life expectancy or functional status were not typically examined. There was no evidence that exclusion varied by the nature of the clinical population in which exclusion was evaluated or trial funding source. There was no statistically significant change in exclusion rates in more recent compared with older trials.ConclusionsThe majority of trials of treatments for physical conditions examined excluded the majority of patients with the condition being treated. Almost a quarter of the trials studied excluded over 90% of patients, more than half of trials excluded at least three quarters of patients, and four out of five trials excluded at least half of patients. A limitation is that most studies applied only a subset of eligibility criteria, so exclusion rates are likely under-estimated. Exclusion from trials of older people and people with co-morbidity and co-prescribing is increasingly untenable given population aging and increasing multimorbidity.Trial registrationPROSPERO registration CRD42016042282.

Project description:BackgroundCurrent German and EU package leaflets (PLs) do not distinguish to what extent listed side effects are indeed side effects caused by drug intake or instead symptoms that occur regardless of drug use. We recently showed that most health professionals misinterpret the frequencies of listed side effects as solely caused by the drug. The present study investigated whether (1) these misinterpretations also prevail among laypeople and (2) alternative PLs reduce these misinterpretations.MethodsIn March 2017, 397 out of 400 laypeople approached completed an online survey. They were randomized to one of four PL formats: three alternative PLs (drug facts box with/without reading instruction, narrative format with numbers) and one standard PL. Each PL listed four side effects for a fictitious drug: two were presented as occurring more often, one as equally often, and one as less often with drug intake. The alternative formats (interventions) included information on frequencies with and without drug intake and included a statement on the causal relation. The standard PL (control) only included information on frequency ranges with drug intake. Questions were asked on general occurrence and causality of side effects.ResultsParticipants randomized to the standard PL were unable to answer questions on causality. For side effects occurring more often (equally; less often) with drug intake, only 1.9% to 2.8% (equally: 1.9%; less often: 1.9%) provided correct responses about the causal nature of side effects, compared to 55.0% to 81.9% (equally: 23.8% to 70.5%; less often: 21.0% to 43.2%) of participants who received alternative PLs. It remains unclear whether one alternative format is superior to the others.ConclusionIn conclusion, information on the frequency of side effects in current package leaflets is misleading. Comparative presentation of frequencies for side effects with and without drug intake including statements on the causal relation significantly improves understanding.

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Exploratory Analysis of the Factors Associated With Success Rates of Confirmatory Randomized Controlled Trials in Cancer Drug Development.

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Exploratory Analysis of the Factors Associated With Success Rates of Confirmatory Randomized Controlled Trials in Cancer Drug Development.

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