The Evolution of Evidence-Based Medicine: When the Magic of the Randomized Clinical Trial Meets Real-World Data

Circulation ◽  
2022 ◽  
Vol 145 (2) ◽  
pp. 107-109
Author(s):  
Seng Chan You ◽  
Harlan M. Krumholz
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Real World ◽  
Evidence Based Medicine ◽  
Evidence Based ◽  
Real World Data ◽  
World Data ◽  
Based Medicine

The future of clinical trials

2010 ◽  
pp. 45-47
Author(s):  
Perry Nisen ◽  
Patrick Vallance
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Alternative Medicine ◽  
20Th Century ◽  
Evidence Based Medicine ◽  
Evidence Based ◽  
Scientific Evaluation ◽  
New Treatments ◽  
Based Medicine ◽  
Short Section

Clinical trials are the bedrock of evidence-based medicine. Introduced in the mid 20th century, they heralded a move away from opinion and anecdote to a more scientific evaluation of new treatments. Indeed, it could be argued that it is the clinical trial and the application of scientific method to determine which treatments work that distinguishes ‘medicine’ from ‘alternative medicine’. The aim of this short section is to outline the way in which clinical trials are likely to evolve over the next few years....

Exploring the Real World: Medical Librarians' Involvement in Supporting Evidence-Based Medicine (EBM) Practice

2013 ◽  
Author(s):  
Ping Li ◽  
Lin Wu
Keyword(s):  
Data Analysis ◽  
Survey Data ◽  
Real World ◽  
Evidence Based Medicine ◽  
Online Survey ◽  
Evidence Based ◽  
Supporting Evidence ◽  
The Real ◽  
Based Medicine

This paper reports the results of an online survey that explores medical librarians’ roles and activities in supporting EBM practice. More than 500 medical librarians replied to the survey. Data analysis reveals that librarians have been taking on various EBM-related responsibilities both routine by nature and project-related.Cet article présente les résultats d’un sondage en ligne portant sur les rôles et les activités des bibliothécaires du domaine des sciences de la santé pour soutenir les pratiques de médecine fondée sur les preuves (MFP). Plus de 500 bibliothécaires ont répondu au sondage. L’analyse des données révèlent que ces bibliothécaires ont participé à des activités routinières et à des projets relevant du domaine de la MFP. 

Evidence-Based Medicine for Rare Diseases: Implications for Data Interpretation and Clinical Trial Design

2007 ◽  
Vol 14 (2) ◽  
pp. 160-166 ◽  
Author(s):  
Madhusmita Behera ◽  
Ambuj Kumar ◽  
Heloisa P. Soares ◽  
Lubomir Sokol ◽  
Benjamin Djulbegovic
Keyword(s):  
Clinical Trial ◽  
Rare Diseases ◽  
Evidence Based Medicine ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Data Interpretation ◽  
Evidence Based ◽  
Based Medicine

The rate of tumor growth, g, as a biomarker for overall survival (OS) in prostate cancer (PC) in clinical trials as well as in real-world data from the Veterans Administration Medical Centers (VAMCs).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 5074-5074
Author(s):  
Harshraj Leuva ◽  
Mengxi Zhou ◽  
Julia Wilkerson ◽  
Keith Sigel ◽  
Ta-Chueh Hsu ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Clinical Trial ◽  
Overall Survival ◽  
Clinical Trials ◽  
Tumor Growth ◽  
Real World ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Real World Data ◽  
World Data

5074 Background: Novel assessments of efficacy are needed to improve determination of treatment outcomes in clinical trials and in real-world settings. Methods: Cancer treatments usually lead to concurrent regression and growth of the drug-sensitive and drug-resistant fractions of a tumor, respectively. We have exploited novel methods of analysis that assess these two simultaneous processes and have estimated rates of tumor growth ( g) and regression ( d) in over 30,000 patients (pts) with diverse tumors. Results: In prostate cancer (PC) we have analyzed both clinical trial and real-world data from Veterans. Using clinical trial data from 6819 pts enrolled in 15 treatment arms we have established separately and by combining all the data that g correlates highly (p<0.0001) with overall survival (OS) – slower g associated with better OS. In PC, abiraterone (ABI) and docetaxel (DOC) are superior to placebo, prednisone and mitoxantrone. ABI (median g =0.0017) is superior to DOC ( g=0.0021) in first line (p=0.0013); and ABI in 2nd line ( g=0.0034) is inferior to ABI in 1st line ( g=0.0017; p<0.0001). Finally, using combined clinical trial data as a benchmark we could assess the efficacy of novel therapies in as few as 30-40 patients. Amongst 7457 Veterans, the median g on a taxane ( g=0.0022) was similar to that from clinical trials ( g=0.0012). Although only 258 Veterans received cabazitaxel (CAB), g values for CAB ( g=0.0018) and DOC ( g=0.0023) were indistinguishable (p=0.3) consistent with their identical mechanism of action. Finally, outcomes with DOC in African American (AA) ( g=0.00212) and Caucasian ( g=0.00205) Veterans were indistinguishable (p=0.9) and comparable across all VAMCs. Conclusions: The rate of tumor growth, g, is an excellent biomarker for OS both in clinical trials and in real-world settings. g allows comparisons between trials and for large trial data sets to be used as benchmarks of efficacy. Real-world outcomes in the VAMCs are similar to those in clinical trials. In the egalitarian VAMCs DOC efficacy in PC is comparable in AA and Caucasian Veterans -- indicating inferior outcomes reported in AAs are likely due to differential health care access, not differences in biology.

Tumor response and growth rate of nivolumab treatment in advanced gastric cancer: Real-world data from a large observational/translational study, JACCRO GC-08 (deliver trial).

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 4527-4527
Author(s):  
Ryohei Kawabata ◽  
Yasuhiro Sakamoto ◽  
Eisuke Inoue ◽  
Atsushi Ishiguro ◽  
Yusuke Akamaru ◽  
...  
Keyword(s):  
Gastric Cancer ◽  
Clinical Trial ◽  
Growth Rate ◽  
Tumor Growth ◽  
Real World ◽  
Fold Increase ◽  
Real World Data ◽  
World Data ◽  
Translational Study ◽  
Before And After

4527 Background: Nivolumab (Nivo) demonstrated survival benefit in previously treated gastric cancer (GC) patients (pts), with a response rate (RR) of 11% and a disease control rate (DCR) of 40% (Kang YK, et al. Lancet 2017). There are few real-world data of Nivo and its predictive markers are needed in GC. It has been demonstrated that some tumors grow rapidly after Nivo treatment, but the proportion is uncertain. Methods: DELIVER trial was a prospective, multicenter, observational/translational study which assessed pts with advanced GC treated with Nivo alone and ECOG Performance Status (PS) 0-2 (UMIN000030850). The aims were to evaluate the efficacy and safety of Nivo in real world, and to discover novel host-related immune-biomarkers (gut microbiome, genetic polymorphism, gene expression, and metabolome) using fecal and blood samples which were collected before and after Nivo treatment. The RR, DCR, progression-free survival, overall survival, and tumor growth rate (TGR) were estimated as the efficacy. The response was evaluated by first imaging based on RECIST version 1.1. The TGR was calculated as a percentage increase in tumor volume during 1 month (Champiat et al. Clin Cancer Res 2017). Results: A total of 501 pts was enrolled in this study from Mar 2018 to Aug 2019, and 487 pts were evaluable for analysis (median age 70-y, 71% male, ECOG PS0/1/2 42%/44%/14%, tub/por/sig 45%/41%/5%, 21% HER2-pos, 42% pts with ascites). The DCR was 39.2% (95%CI 34.9-43.7) in evaluable pts. In 282 pts with measurable lesions, the RR was 6.7% (95%CI 4.1-10.3) and DCR was 36.5%. Sub-analysis by patient background indicated that DCR was 41% for PS0, 42% for PS1, and 24% for PS2. In addition, the DCR was lower in pts with ascites compared to those without ascites (28.6% vs. 47.0%, p= 0.005). The TGR decreased after introduction of Nivo in 124 (56.6%) of 219 evaluable pts for TGR; however, 20.5% pts were identified as experiencing hyper-progressive disease (HPD) which was defined as a ≥2-fold increase of the TGR before and after Nivo. When defining HPD as a ≥2-fold increase of tumor growth kinetics ratio and 50% increase of tumor burden, 9.6% pts experienced it. Conclusions: The real-word data of the large observational trial showed a comparable DCR to that of clinical trial in advanced GC treated with Nivo. This trial revealed the tumor behavior and some pts who experienced rapid tumor growth after Nivo treatment in clinical practice; biomarkers for HPD and the definition should be established. Clinical trial information: UMIN000030850 .

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