scholarly journals Predictors of Citation Rates in High Impact Glioblastoma Trials

2021 ◽  
Author(s):  
Ammer M Jamjoom ◽  
Abdulhakim B Jamjoom
Keyword(s):  
Clinical Trials ◽  
Pearson Correlation ◽  
Secondary Analysis ◽  
Treatment Protocol ◽  
High Impact ◽  
Phase Iii ◽  
Treatment Modalities ◽  
Primary Analysis ◽  
Study Population ◽  
Trial Phase

Abstract Background Clinical trials are at the top of research study designs and tend to attract high citation numbers. Glioblastoma multiforme (GBM) is a multidisciplinary disease that continues to be the subject of peak research interest. The literature relating to predictors of citation rates in clinical trials in general remains limited. We aim to identify the factors that influence citation numbers in high impact GBM trials. Methods The 100 most cited published GBM trials were identified and reviewed. The primary analysis was correlating articles citation numbers with various trial and publication-related predictors using Pearson correlation coefficient. The secondary analysis was comparing the mean citation numbers for the different subgroups using mean difference test. Results The median (range) citation numbers for the selected 100 trials were 349 (135- 16384). The primary analysis showed significant correlation between articles citation numbers and study population (P=0.024), trial phase (P=0.0427) and journal’s IF (P<0.0001). The secondary analysis demonstrated significantly higher mean citation numbers in trials with the following features: study population ≥ 115 (P=0.0208), phase III (P=0.0372), treatment protocol that included radiotherapy (RT) (P=0.0189) and temozolomide (TMZ) (P=0.0343), journal’s IF ≥ 14.9 (P=0.02) and general medical journals (P=0.28). Conclusions The most significant predictors of citation rates in high impact GBM trials were study population, trial phase, and journal IF. The treatment protocol was a positive predictor when it included the currently widely accepted treatment modalities (RT and TZM). Randomization, age of publication as well as the numbers of arms, authors, centres, countries, and references were not significant predictors. Increasing awareness of the factors that could affect citations may be useful to researchers undertaking clinical trials.

Tandem Autologous Stem Cell Transplants (auto-auto) with or without Maintenance Therapy Versus Single Autologous Transplant Followed by HLA-Matched Sibling Non- Myeloablative Allogeneic Stem Cell Transplant (auto-allo) for Patients (pts) with High Risk (HR) Multiple Myeloma (MM): Results From the Blood and Marrow Transplant Clinical Trials Network (BMT-CTN) 0102 Trial

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 526-526 ◽  
Author(s):  
Edward A Stadtmauer ◽  
Amrita Krishnan ◽  
Marcelo C Pasquini ◽  
Marian Ewell ◽  
Edwin P Alyea ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplant ◽  
Chronic Gvhd ◽  
Secondary Analysis ◽  
Progression Free Survival ◽  
Phase Iii ◽  
Cell Transplant ◽  
Primary Analysis ◽  
Hematopoietic Stem

Abstract Abstract 526 The prognosis of patients with high-risk myeloma (HR MM) continues to be dismal, despite the early incorporation of novel agents. Early phase trials of allogeneic hematopoietic stem cell transplant (alloHCT) suggest the possibility of an immunologic graft-versus-myeloma effect that might favorably affect survival. Less toxic reduced-intensity HCT preparative regimens now allow more widespread use of alloHCT in the MM population. BMT CTN 0102 is a phase III multicenter clinical trial that biologically assigned patients to either melphalan 200mg/m2 (MEL 200) auto-auto without (obs) or with 1 year of thalidomide and dexamethosone (ThalDex), or an auto-allo approach using MEL 200 followed by alloHCT using 2 Gy total body irradiation. Graft-versus-host disease (GVHD) prophylaxis was cyclosporine and mycophenolate mofetil. Patients were stratified by biological prognostic factors that were considered to be high risk at the time of the trial design: chromosome 13 deletions by metaphase karyotype and beta-2 microglobulin ≥4 mg/dl. The primary endpoint was 3-year progression free survival (PFS). Between December 2003 and March 2007, 710 patients from 43 US centers were enrolled, and 85 fulfilled the criteria of HR MM. Among them, 48 were assigned to auto-auto (24 Thal-Dex and 24 obs) and 37 to auto-allo. Groups differed in age (median 57 y and 51y, p=0.02) but were otherwise balanced. Compliance with second transplant was 65% for auto-auto and 78% for auto-allo. Compliance with ThalDex was poor, so the two auto-auto arms were pooled for the primary analysis. Three-year PFS was 33% (95% Confidence Interval (CI), 22–50%) and 40% (95% CI, 27–60%, p=0.74) and 3-year OS was 67% (95% CI, 54–82%) and 59% (95% CI, 49–78%, p=0.46) for auto-auto and auto-allo, respectively. Corresponding probabilities for 3-year progression/relapse was 53% and 33% (p=0.09), and 3 year treatment-related mortality was 8% and 20% (p=0.3). Among auto-allo patients, probabilities of grade 3–4 acute and chronic GVHD were 9% and 48%, respectively. Among the 59 (31 auto-auto, 28 auto-allo) patients who received second transplant, 3 year PFS was 35% and 46% (p=0.6). Disease response at day 56 after second transplant was 57% for very good partial response (VGPR) or better and 37% for complete response (CR) and near CR (nCR) in the auto-auto group; and 48% (VGPR or better) and 41% (CR+nCR) in the auto-allo group. In conclusion, this planned secondary analysis of a cohort of HR MM patients demonstrated equivalent 3-year PFS and OS for auto-auto and auto-allo in both intention-to-treat and as-treated analyses. However, trends in late PFS and time to progression/relapse suggest further follow-up is needed before final conclusions regarding the utility of auto-allo in this HR cohort can be made. Finally, this study shows the feasibility of an alloHCT approach for HR MM patients and may serve as a platform for future studies seeking to enhance graft-versus-myeloma effects. Disclosures: Stadtmauer: Celgene: Speakers Bureau. Krishnan:Celgene: Speakers Bureau. Qazilbash:Celgene: Speakers Bureau. Vesole:Celgene: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Giralt:Celgene: Honoraria, Speakers Bureau; Millenium: Honoraria, Speakers Bureau.

INTEGRATE: A randomized phase II double-blind placebo-controlled study of regorafenib in refractory advanced oesophagogastric cancer (AOGC)—A study by the Australasian Gastrointestinal Trials Group (AGITG), first results.

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 9-9 ◽  
Author(s):  
Nick Pavlakis ◽  
Katrin Marie Sjoquist ◽  
Eric Tsobanis ◽  
Andrew Martin ◽  
Yoon-Koo Kang ◽  
...  
Keyword(s):  
Phase Ii ◽  
Kinase Inhibitor ◽  
Secondary Analysis ◽  
Progression Free Survival ◽  
Best Supportive Care ◽  
Phase Iii ◽  
Prognostic Indicators ◽  
Controlled Study ◽  
Primary Analysis ◽  
Double Blind

9 Background: Advanced Oesophago-Gastric Carcinoma (AOGC) has limited options following failure of first or second line chemotherapy (CT). Regorafenib (REG) is an oral multi-kinase inhibitor of kinases involved in angiogenesis, tumor microenvironment, and oncogenesis. This study examined whether REG has sufficient activity and safety for further evaluation. Methods: International (Australia & New Zealand (ANZ), Korea, Canada (NCIC CTG)) randomised phase II trial with 2:1 randomisation and stratification by: (1) Lines of prior CT for advanced disease (1 vs. 2) and (2) Region. Eligible patients received best supportive care plus 160mg REG or matching PBO orally on days 1-21 each 28-day cycle until disease progression or prohibitive adverse events. Primary endpoint was progression free survival (PFS) in the REG arm, assuming median 8 weeks (wks) in PBO arm, aiming for 13.2 wks with REG to be of interest. Results: From Nov 2012 to Feb 2014, 152 patients were enrolled, 147 evaluable [pre-specified primary analysis population]: (REG n=97: PBO n=50); well matched for key baseline prognostic indicators; male:female (118:29); primary location: OG Junction (56), stomach (85); lines of prior therapy: 1 (63), 2 (84); ECOG 0 (62): 1 (85). Time on treatment: Median: 7.9 (REG) v 4 (PBO) wks. In the evaluable population median PFS was 11.1 wks (95% CI: 7.7 - 12.3) (REG) and 3.9 wks (95% CI: 3.7 - 4.0) (PBO), log-rank p <0.0001; HR 0.41 (95% CI: 0.28 to 0.59). PFS results were maintained for secondary analysis including all randomized patients (n = 152). REG was well tolerated, with the spectrum of toxicity in keeping with previous reports. Conclusions: PFS was clearly significantly longer with REG than PBO, though PBO PFS was less than anticipated. The pre-specified exploratory comparisons provide compelling evidence that REG has sufficient activity with acceptable tolerability in refractory AOGC to warrant phase III evaluation. Mature OS results will be presented at the meeting. Clinical trial information: 12612000239864.

Application of ASCO Value Framework to Treatment Advances in Hepatocellular Carcinoma

2021 ◽  
pp. OP.20.00558 ◽  
Author(s):  
Emerson Y. Chen ◽  
Madeline Cook ◽  
Christopher Deig ◽  
Asad Arastu ◽  
Vinay Prasad ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Secondary Analysis ◽  
Health Benefit ◽  
Progression Free Survival ◽  
Phase Iii ◽  
Primary Analysis ◽  
Free Survival ◽  
Drug Registration ◽  
Phase Iii Trials

BACKGROUND: Determination of the comparative efficacy of one therapy over another for hepatocellular carcinoma (HCC) can be challenging. Application of a recognized value framework to published studies could objectively compare the potential benefit across available therapies. MATERIALS AND METHODS: An umbrella review of phase III trials for HCC therapies was performed. ASCO Value Framework Net Health Benefit Score version 2 (ASCO-NHB v2) scores, the primary analysis, and European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 scores, the secondary analysis, were computed using selected drug registration trials. Both scores were compared between drugs that were Food and Drug Administration (FDA)-approved by 2020 and those that were not. RESULTS: Of the 22 studies identified, nine were FDA-approved and 13 were not. Across 22 trials, the median overall survival (OS) was 9.2 months (range, 1.9-16.4 months), with a median gain of 0.35 month (range, 2.3-3.3 months). HCC therapies that were FDA-approved showed longer OS (median 10.7 v 7.9 months, P < .01) and higher ASCO NHB scores (+18.4 v −5.7 scores, P < .01). The median gain in OS was 2.2 months in the approved treatments compared with −0.3 months in the unapproved group, with no difference in progression-free survival between the two groups. CONCLUSION: The nine FDA-approved therapies for HCC have higher mean NHB score than those that were not FDA-approved. The application of ASCO-NHB v2 and other proposed value frameworks could examine data of future therapies for HCC through a patient-oriented approach.

Bayesian survival analysis in clinical trials: What methods are used in practice?

2016 ◽  
Vol 14 (1) ◽  
pp. 78-87 ◽  
Author(s):  
Caroline Brard ◽  
Gwénaël Le Teuff ◽  
Marie-Cécile Le Deley ◽  
Lisa V Hampson
Keyword(s):  
Clinical Trials ◽  
Survival Analysis ◽  
Bayesian Methods ◽  
Regression Models ◽  
Final Analysis ◽  
Bayesian Regression ◽  
Phase Iii ◽  
Informative Priors ◽  
Primary Analysis ◽  
Survival Analyses

Background Bayesian statistics are an appealing alternative to the traditional frequentist approach to designing, analysing, and reporting of clinical trials, especially in rare diseases. Time-to-event endpoints are widely used in many medical fields. There are additional complexities to designing Bayesian survival trials which arise from the need to specify a model for the survival distribution. The objective of this article was to critically review the use and reporting of Bayesian methods in survival trials. Methods A systematic review of clinical trials using Bayesian survival analyses was performed through PubMed and Web of Science databases. This was complemented by a full text search of the online repositories of pre-selected journals. Cost-effectiveness, dose-finding studies, meta-analyses, and methodological papers using clinical trials were excluded. Results In total, 28 articles met the inclusion criteria, 25 were original reports of clinical trials and 3 were re-analyses of a clinical trial. Most trials were in oncology (n = 25), were randomised controlled (n = 21) phase III trials (n = 13), and half considered a rare disease (n = 13). Bayesian approaches were used for monitoring in 14 trials and for the final analysis only in 14 trials. In the latter case, Bayesian survival analyses were used for the primary analysis in four cases, for the secondary analysis in seven cases, and for the trial re-analysis in three cases. Overall, 12 articles reported fitting Bayesian regression models (semi-parametric, n = 3; parametric, n = 9). Prior distributions were often incompletely reported: 20 articles did not define the prior distribution used for the parameter of interest. Over half of the trials used only non-informative priors for monitoring and the final analysis (n = 12) when it was specified. Indeed, no articles fitting Bayesian regression models placed informative priors on the parameter of interest. The prior for the treatment effect was based on historical data in only four trials. Decision rules were pre-defined in eight cases when trials used Bayesian monitoring, and in only one case when trials adopted a Bayesian approach to the final analysis. Conclusion Few trials implemented a Bayesian survival analysis and few incorporated external data into priors. There is scope to improve the quality of reporting of Bayesian methods in survival trials. Extension of the Consolidated Standards of Reporting Trials statement for reporting Bayesian clinical trials is recommended.

scholarly journals Gender and sex disparity in cancer trials

ESMO Open ◽  
2020 ◽  
Vol 5 (Suppl 4) ◽  
pp. e000773 ◽  
Author(s):  
Eudocia Lee ◽  
Patrick Wen
Keyword(s):  
Clinical Trials ◽  
Elderly Women ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Phase Iii Clinical Trials ◽  
The Usa ◽  
Study Population ◽  
Cancer Trials ◽  
Early Phase Clinical Trials ◽  
Sex Disparity

The study population within phase III clinical trials leading to approval of new cancer agents should ideally more closely mirror the population who will ultimately receive these agents. Although the number of females participating in clinical trials has increased over the past several decades, females are still under-represented in preclinical studies, in early phase clinical trials and even in some later phase cancer clinical trials. In the USA, this is particularly true for women from minority populations and elderly women. In this review, we review gender and sex disparities in cancer trials, the reasons for these disparities, the barriers to clinical trial enrolment and ways to improve diversity in cancer clinical trials.

scholarly journals Betrixaban: A Novel Oral Anticoagulant With a New Niche

2018 ◽  
Vol 34 (3) ◽  
pp. 123-133 ◽  
Author(s):  
Grazia Murphy ◽  
Yasmin Grace ◽  
Sadaf Chaudry ◽  
Rita Chamoun
Keyword(s):  
Clinical Trial ◽  
At Risk ◽  
Clinical Trials ◽  
Oral Anticoagulant ◽  
Phase Iii ◽  
Medical Illness ◽  
Safety And Efficacy ◽  
Vte Prophylaxis ◽  
Significant Difference ◽  
Trial Phase

Objective: To evaluate the efficacy, safety, and clinical implication of betrixaban for prophylaxis of venous thromboembolism (VTE) in patients with acute medical illness. Data Sources: A search for clinical trials was performed from January 2006 to January 2017 in English language using Clinicaltrials.gov and PubMed/MEDLINE. The following search terms were used: betrixaban, pharmacokinetics, pharmacology, and drug safety. Study Selection: The following limits were used to access the clinical trials: controlled clinical trial, randomized clinical trial, clinical trial, clinical trial phase II, and clinical trial phase III. The search was narrowed to include only humans. Data Extraction: The search criteria resulted in 6 clinical trials assessing the safety and efficacy of betrixaban. Additionally, references from publications assessing the safety and efficacy of betrixaban in atrial fibrillation, treatment and prevention of VTE, and extended duration VTE prophylaxis were assessed. Data Synthesis: Prior to 2017, no anticoagulant therapy had been approved for extended VTE prophylaxis in acutely ill medical patients. Betrixaban is the first direct oral anticoagulant approved for VTE prophylaxis in adult, acutely ill patients at risk for thromboembolisms. Based on the APEX trial, betrixaban 80 mg administered daily for 35 to 42 days was compared to enoxaparin administered daily for 6 to 14 days. In 7441 patients, fewer VTEs were seen in the betrixaban compared to enoxaparin with no significant difference in adverse reactions. Conclusion: Based on clinical trials, betrixaban appears to be safe and effective in preventing VTE in acutely ill patients who are at risk of developing VTE.

scholarly journals Chinese Clinical Studies for Pharmacological Treatments of Coronavirus Disease 2019 (COVID-19)

2020 ◽  
Author(s):  
Ru Han ◽  
Yitong Wang ◽  
Monique Dabbous ◽  
Shuyao Liang ◽  
Tingting Qiu ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Studies ◽  
Observational Studies ◽  
Reference Group ◽  
Descriptive Analysis ◽  
Secondary Analysis ◽  
Trial Registry ◽  
Primary Analysis ◽  
Clinical Trial Registry

Objectives: This study aims to identify, report, and analyze registered and published clinical trials and observational studies for the pharmacological treatment of COVID-19 conducted in China. Methods: A strategic search was conducted via the Chinese Clinical Trial Registry to identify and extract clinical trials and observational studies registered and conducted in China for the pharmacological treatment of COVID-2019 between January 1st, 2020 and March 21st, 2020. This was further supplemented by searches conducted via the China National Knowledge Infrastructure (CNKI) database, the MEDLINE database, the World Health Organization (WHO) database, and MedRxiv and BioRxiv electronic platforms for preprint articles, published up until April 8th, 2020. Studies available in Chinese and English were included in the searches and extracted. A primary descriptive analysis was performed for registered clinical trials and observational studies identified in the Chinese Clinical Trial Registry based on the extraction of the following clinical study information: trial ID, planned date of enrollment, recruitment status, study design, population, sample size, intervention/exposure group, control /reference group, dosage, and primary outcomes. A secondary descriptive analysis was performed for published clinical trials and observational studies identified from the supplementary databases based on the extraction of the following published clinical study information: study design, population, intervention/exposure group, control /reference group and main results as appropriate. Results: A total of 221 clinical trials and observational studies were included from all databases searched. From the Chinese Clinical Trial Registry, 195 registered clinical studies including 170 clinical trials and 25 observational studies were identified and included for primary analysis. From the supplementary databases, 26 published clinical studies including 8 clinical trials and 18 observational studies were included for secondary analysis. Of these 26 published clinical studies, 18 studies, including 3 clinical trials and 15 observational studies were identified from CNKI, 2 studies including 1 clinical trial and 1 observational study from MEDLINE, 2 including 1 clinical trials and 1 observational studies from the WHO database, and 4 including 3 clinical trials and 1 observational studies from MedRxiv and BioRxiv platforms. In the primary analysis, among the 170 clinical trials included from the Chinese Clinical Trial Registry, 101 investigated western medicines (WMs), while 15 investigated Traditional Chinese Medicines (TCMs), and 54 investigated a combination of TCMs and WMs. Among the 25 included observational studies from the Chinese Clinical Trial Registry, 2 investigated WMs, 2 investigated TCMs, and 21 investigated a combination of TCMs and WMs. The total number of exposed patients in all 195 clinical studies from the Chinese Clinical Trial Registry amounted to 24,500. In the secondary analysis, treatment with Lopinavir-ritonavir and treatment with Hydroxychloroquine was not associated with a difference from standard of care in the rate of RT-PCR negativity; treatment with a combination of Lopinavir-ritonavir, interferon &alpha;, and Lian-Hua-Qing-Wen capsule was found to significantly improve the effective rate of treatment compared with Interferon &alpha; combined with Lian-Hua-Qing-Wen capsule. Conclusions: China is generating a massive source of evidence that is critical for defeating the COVID-19 pandemic. Not only the clinical experience, but also the scientific evidence should be shared with the global scientific community.

Hydrogen Peroxide 40% for the Treatment of Seborrheic Keratoses

2020 ◽  
Vol 55 (2) ◽  
pp. 216-221
Author(s):  
Colton H. Funkhouser ◽  
Kathleen M. Coerdt ◽  
Wasim Haidari ◽  
Michael A. Cardis
Keyword(s):  
Hydrogen Peroxide ◽  
Clinical Trials ◽  
Phase Ii ◽  
Treatment Options ◽  
Phase Iii ◽  
Treatment Modalities ◽  
Cochrane Library ◽  
Seborrheic Keratosis ◽  
Safe Alternative ◽  
Phase Iii Clinical Trials

Objective: Hydrogen peroxide 40% (HP40) was approved by the US Food and Drug Administration for topical treatment of seborrheic keratosis (SK) in December 2017. This article will review phase II and III clinical trials to assess the drug’s efficacy, safety, and clinical application. Data Sources: A systematic literature review was performed using the terms “Eskata AND seborrheic keratosis,” and “hydrogen peroxide AND seborrheic keratosis” in the OVID MEDLINE, PubMed, Cochrane Library, EMBASE, and Web of Science databases. ClinicalTrials.gov was searched to identify ongoing or nonpublished studies. Study Selection and Data Abstraction: Articles written in English between January 2000 and mid-June 2020 discussing phase II and phase III clinical trials were evaluated. Data Synthesis: In 2 phase III clinical trials, 4% and 8% of patients treated with HP40 had a Physician Lesion Assessment score of zero for all 4 SKs, respectively, compared with 0% in both vehicle groups at the primary end point of day 106 ( P < 0.01; P < 0.0001). Relevance to Patient Care and Clinical Practice: HP40, although less effective, has a better safety profile than other treatment options. It should be especially considered for treatment of facial SKs, where it is most efficacious and where other treatment modalities, such as cryotherapy, are more challenging. Conclusions: HP40 is a new, safe alternative treatment for SKs, although it is expensive and only modestly effective, both of which somewhat limit its overall utility. HP40 is a promising topical alternative, particularly for cosmetically sensitive locations, such as the face.

Sown the seeds: An analysis of published trials in clinical oncology.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 6068-6068
Author(s):  
Natalie M. Spradlin ◽  
David H. Johnson ◽  
Leora Horn
Keyword(s):  
Clinical Trials ◽  
Phase I ◽  
Clin Oncol ◽  
Phase Ii ◽  
High Impact ◽  
Phase Iii ◽  
Major Market ◽  
Therapeutic Class ◽  
Disease Site ◽  
Pubmed Search

6068 Background: Competition in the pharmaceutical marketplace has resulted in “seeding trials” which appear to serve little scientific purpose other than to gain a financial hold in the marketplace. Hallmarks of “seeding” clinical trials [Kessler, et al N Eng J Med 1994] include trial design that does not support stated research goals, industry sponsorship without novel findings or poor scientific rationale, and drugs introduced into an already crowded therapeutic class, i.e. “me too” drugs. We evaluated clinical trials published in high impact oncology journals to ascertain the prevalence of trials bearing the characteristics of a seeding trial. Methods: We conducted a PubMed search using “clinical trial”, “oncology”, published 2/2005 – 2/2010, in Ann Oncol, J Clin Oncol, Lancet, Lancet Oncol, or the N Engl J Med. All phase I/II, II and III studies were included. Phase I, hematology, radiation oncology, and pediatric studies were excluded. Data collected included disease site, phase, funding source, journal, and whether the trial fit criteria for “seeding” as previously defined. Results: 1781 articles have been evaluated to date. 528 met criteria for analysis. 130 were considered seed studies. Seed studies per journal were Ann Oncol 26 of 128, J Clin Oncol 88 of 340, Lancet 8 of 22, Lancet Oncol 5 of 16, and N Engl J Med 3 of 22. Studies published per disease site and percent seed studies were melanoma 19 and 31.6%, breast 106 and 30.2%, GI 126 and 20.6%, lung 101 and 29.7%, GU 65 and 24.6%, gynecology 40 and 30%, head and neck 24 and 12.5%, CNS 24 and 12.5%, sarcoma 11 and 18.2%, and advanced cancer 12 with zero seeds. Overall phase totals were phase I/II 16, phase II 272, and phase III 240. Seed studies per phase were phase II 28.7%, phase III 21.7% and zero for phase I/II. 210 studies were solely industry sponsored, and of these, 37.1% were seed studies. Funding was not listed for 73 studies. Conclusions: The integrity of clinical trials is challenged in the race to claim major market share. Our analysis of clinical trials published in high impact oncology journals found seeding trials account for 24.6% of publications. 37.1% of industry sponsored trials are seed studies. Seed studies are more common among the major disease sites or sites for which targeted therapies exist.

Application of the ASCO framework for assessing value in metastatic colorectal cancer clinical trials.

2017 ◽  
Vol 35 (4_suppl) ◽  
pp. 687-687
Author(s):  
Doreen Anuli Ezeife ◽  
Sunil Parimi ◽  
Ellen R. Cusano ◽  
Matthew K Smith ◽  
Tony H. Truong ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Clinical Trials ◽  
Metastatic Colorectal Cancer ◽  
Pearson Correlation ◽  
Statistical Significance ◽  
Linear Regression Analysis ◽  
Health Benefit ◽  
Phase Iii ◽  
Multivariate Linear Regression Analysis ◽  
Nccn Guidelines

687 Background: Phase III trials (P3T) in metastatic colorectal cancer (mCRC) have collectively led to incredible advancements in patient outcomes over the past thirty years. It is unknown how many of these trials have led to clinically relevant improvements based on clinical benefit, toxicity and cost. Our objective was to characterize the evolution of mCRC P3T with the ASCO Value Framework. Methods: P3T of systemic therapy for mCRC published between 1980 and 2015 were identified. Data regarding the trial sample size, journal year of publication, statistical significance, journal impact factor, and citation by the 2015 mCRC National Comprehensive Cancer Network (NCCN) guidelines were extracted. For each trial, the Net Health Benefit (NHB) score was calculated using the June 2015 (original) and May 2016 (revised) ASCO Value Advanced Disease Frameworks. Data were analyzed to identify associations using the Chi-square, Kruskal-Wallis, and t-tests. Results: There were 108 mCRC P3T eligible for calculation of the original and revised NHB score. Median NHB score was 4.1, range -30 to 43.5, using the revised framework. Only 13% of trials received at least 10 bonus points for palliation, quality of life, or tail of the curve. The revised framework demonstrated moderate correlation with the original framework (Pearson correlation coefficient = 0.63, p<0.0001). NHB scores were normally distributed with the revised framework, unlike the original framework. Trials with significant results had significantly higher revised NHB scores (median NHB score 23.1 vs 2.5, p < 0.0001). Clinical trials cited in NCCN had higher revised NHB scores than those not cited (median NHB score 7.6 vs 0, p = 0.03). In multivariate linear regression analysis, the only significant predictor of a high revised NHB score was a statistically significant improvement in the primary outcome. Conclusions: Median revised NHB score for mCRC P3T was 4.1. Higher NHB scores were associated with significant studies and citation by NCCN guidelines, a surrogate for practice-changing trials. The revised ASCO Value Framework can be a useful tool to assess the value of new mCRC treatments.

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