Real-world survival of men with metastatic hormone-sensitive prostate cancer (mHSPC) treated with abiraterone acetate (Abi) or docetaxel (Doc) and comparison with clinical trial outcomes.

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 53-53
Author(s):  
Daniel M. Geynisman ◽  
Andres F Correa ◽  
Chethan Ramamurthy ◽  
J Robert Beck ◽  
Elizabeth A. Handorf
Keyword(s):  
Clinical Trial ◽  
Observational Study ◽  
Real World ◽  
Trial Data ◽  
Phase Iii ◽  
Real World Data ◽  
Large Variability ◽  
World Data ◽  
Level Data

53 Background: Multiple phase III trials have proven that Abi and Doc both improve overall survival (OS) in men with mHSPC. No randomized trials have compared the two approaches. Methods: We conducted a retrospective, observational study to compare OS in de novo M1 men, treated with Abi vs. Doc using patient-level data from the Flatiron health EHR-derived de-identified database. We also compared this real-world OS to trial level data using extracted data points along the OS curves from CHAARTED and LATITUDE trials. OS was compared via Kaplan-Meier curves. Analyses were adjusted via propensity score weighting for age, Gleason score, PSA at diagnosis, race, ethnicity, ECOG PS, insurance type and treatment setting. Results: The cohort included 418 Abi pts and 807 Doc pts (Table). Median follow-up was 13.5 mo for Abi and 31.6 mo for Doc. Unadjusted median OS for Abi and Doc were 31.6 mo (95% CI 28.1-NA) and 41.8 mo (95% CI 37.4-46.3) respectively (P=0.09). Twelve mo and 24 mo OS for Abi was 86.3% and 69%; for Doc it was 89.8% and 72.1 %. Median adjusted OS for Abi and Doc were 31.6 mo (95% CI 28.0-undefined) and 38.8 mo (95% CI 33.1-46.3) respectively (P=0.4). Twelve mo and 24 mo adjusted OS for Abi was 86.6% and 69.4%; for Doc it was 87.9% and 69.2%. Based on extracted trial data, in LATITUDE, Abi treated pts had 12 mo and 24 mo OS of 93.5% and 77.0%; in CHAARTED, Doc treated pts had 12 mo and 24 mo OS of 94.3% and 83.6%. Conclusions: Utilizing real-world data, we demonstrate that 12 and 24-months OS are clinically and statistically similar between Abi and Doc in men with mHSPC. Median OS is also similar, although due to limited follow-up, the estimate of median OS for Abi has large variability. In addition, we show that clinical trial pts had superior outcomes to those in a real-world clinic population. Recent meta-analyses of trial data have not found significant differences in OS for Abi vs. Doc; this analysis of real-world data confirms these findings and indicates that they may be generalizable to a broader patient population. Although this observational study is subject to residual confounding and missing data, it provides further evidence to support the use of both Abi and Doc in men with mHSPC. Differentiating costs, side-effects and QOL can thus become more prominent when making decisions about therapy. [Table: see text]

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.

Estimate of Real-World Medical Costs Avoidance When New Oral Anticoagulants Are Used Vs. Standard Therapy for the Treatment of Patients with Venous Thromboembolism in the U.S

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 687-687
Author(s):  
Alpesh N Amin ◽  
Amanda Bruno ◽  
Jeffrey Trocio ◽  
Jay Lin ◽  
Melissa Lingohr-Smith
Keyword(s):  
Clinical Trial ◽  
Medical Cost ◽  
Real World ◽  
Standard Therapy ◽  
Clinical Trial Data ◽  
Medical Costs ◽  
Trial Data ◽  
Real World Data ◽  
World Data ◽  
Event Rates

Abstract Introduction: Clinical trials have demonstrated that the new oral anticoagulants (NOACs), dabigatran, rivaroxaban, apixaban, and edoxaban are noninferior to standard therapies for treatment of acute symptomatic venous thromboembolism (VTE). We have previously published the medical costs avoided when NOACs are used instead of standard therapies based on rates of clinical events reported in clinical trials. However, the rates of recurrent VTE and major bleeding (MB) in the real-world settings may differ from those from the clinical trials. In this study, we estimated the real-world medical cost avoidance from a U.S. payer perspective when NOACs are used instead of standard therapy for the treatment of patients with VTE. Methods: Reduction of real-world event rates of recurrent VTE and MB were obtained by applying rate reductions observed in clinical studies to the Worcester population. Incremental annual medical costs among patients with VTE and MB from a U.S. payer perspective were obtained from the literature or healthcare claims databases and inflation adjusted to 2013 costs. Differences in total medical costs associated with clinical endpoints for patients treated with NOACs vs. standard therapy were then estimated. One-way univariate and Monte Carlo sensitivity analyses were additionally carried out. Univariate analysis varied the estimates of the clinical event rates between the ranges of confidence intervals and the estimates of event costs ±30% when such confidence intervals were not reported. Ten thousand cycles of Monte-Carlo simulations were used for additional sensitivity analysis where all model parameters were allowed to vary simultaneously. Results: Real-world event rates of recurrent VTE and MB in the Worcester VTE study were 11.2% and 10.8% respectively. Differences in real-world event rates of recurrent VTE among VTE patients treated with NOACs instead of standard therapy were estimated at -1.80% for apixaban, -1.23% for rivaroxaban, -2.02% for edoxaban, and 1.02% for dabigatran. Differences in real-world event rates of MB among VTE patients treated with NOACs instead of standard therapy were estimated at -7.48% for apixaban, -4.97% for rivaroxaban, -1.73% for edoxaban, and -2.57% for dabigatran. Based on the real-world data, the annual total medical cost avoidances vs. standard therapy were greatest for VTE patients treated with apixaban (-$4,440 per patient year-ppy), followed by those treated with rivaroxaban (-$2,971 ppy), edoxaban (-$1,957 ppy), and dabigatran (-$572 ppy). In comparison to data previously reported based on clinical trials, these medical cost avoidances are substantially greater for any of the NOACs vs. standard therapy (Table). The medical cost avoidances remained consistent under univariate (one-way) sensitivity. Additionally, the mean cost estimates of 10,000 random cycles of Monte-Carlo simulations for each of the NOACs were similar to the default estimated medical cost avoidances, demonstrating the robustness of the model estimates. Conclusions: Based on real-world data, when any of the evaluated NOACs are used instead of standard therapy for treatment of patients with acute VTE annual medical costs are reduced. In the real-world setting, the use of NOACs vs. standard therapy is predicted to be associated with even greater annual medical cost reductions than that previously estimated based on clinical trial data. Of the NOACs, apixaban has the greatest real-world medical cost avoidance, as its use is associated with substantial reductions in both VTE and MB event rates. Abstract 687. Table 1 Estimates of Medical Cost Differences Among VTE Patients Treated with NOACs vs. Standard Therapy Based on Clinical Trial Data vs. Real-World Data Outcome Apixaban ($/patient-yr) Rivaroxaban ($/patient-yr) Edoxaban ($/patient-yr) Dabigatran ($/patient-yr) Recurrent VTE* Clinical trial data -$252 -$132 -$197 $114 Real-world data -$1,047 -$717 -$1,173 $595 Major bleedings* Clinical trial data -$572 -$354 -$109 -$195 Real-world data -$3,392 -$2,254 -$784 -$1,167 Total Medical Cost* Clinical trial data -$824 -$486 -$306 -$80 Real-world data -$4,440 -$2,971 -$1,957 -$572 *Negative values mean the NOAC is associated with lower total medical cost vs. standard therapy. Disclosures Amin: Bristol-Myers Squibb, Pfizer: Consultancy. Off Label Use: Apixaban and edoxaban for the indication of VTE. Bruno:Bristol-Myers Squibb: Employment, Equity Ownership. Trocio:Pfizer: Employment, Equity Ownership. Lin:Bristol-Myers Squibb, Pfizer: Consultancy, Research Funding. Lingohr-Smith:Bristol-Myers Squibb, Pfizer: Consultancy, Research Funding.

PP81 Real World Data: The Early Access To Medicines Scheme Catches The Worm

2019 ◽  
Vol 35 (S1) ◽  
pp. 53-53
Author(s):  
Adam Hall ◽  
Lok Wan Liu ◽  
Richard Macaulay ◽  
Sean Walsh
Keyword(s):  
Clinical Trial ◽  
Clinical Practice ◽  
Real World ◽  
Clinical Trial Data ◽  
Access To Medicines ◽  
Trial Data ◽  
Real World Data ◽  
World Data ◽  
Early Access ◽  
Nice Guidance

IntroductionThe Early Access to Medicines Scheme (EAMS) aims to provide access to medicines prior to market authorization for patients with severe, life-threatening diseases who do not have adequate treatment options. An EAMS designation enables the potential collection of United Kingdom-specific real world evidence (RWE) prior to health technology assessment (HTA) by the National Institute for Health and Care Excellence (NICE). This research evaluates whether RWE is being gathered through the EAMS and utilized to support HTA submissions.MethodsAll EAMS designations as of 7 November 2018 were identified from the Medicines and Healthcare products Regulatory Agency website. For products with final NICE guidance, all publicly-available NICE documentation was reviewed.ResultsSixteen product and indication pairings with an EAMS designation were identified, with 12 having received final NICE guidance (11 were recommended, 3 were recommended for temporary reimbursement via the Cancer Drugs Fund, and 2 were not recommended). Of the 11 recommended products, seven had references to the number of patients or sites with product access through the EAMS, but only one (dupilumab for atopic dermatitis) had detailed data collected during the EAMS period. The manufacturer of dupilumab reported baseline demographics and disease characteristics from a cohort of 35 patients treated under the EAMS to inform the generalizability of trial populations for clinical practice. Follow-up results from this cohort demonstrated that real-world data on dupilumab effectiveness was comparable with the clinical trial data, despite a higher proportion of patients in the real-world cohort receiving immunosuppressant therapy, which makes improvements in efficacy harder to achieve. The committee also noted that the RWE presented supported the understanding of dupilumab's long-term clinical effectiveness and informed assumptions for the economic model.ConclusionsTo date, the majority of products receiving an EAMS designation have not presented RWE at NICE reappraisal. The case of dupilumab illustrated how RWE collected through the EAMS can be used to reduce uncertainty around how clinical trial data can be translated into clinical practice. In the future, RWE may increasingly be used to help inform NICE decisions.

Survival analysis by tumor response from real-world data in advanced gastric cancer treated with nivolumab: The DELIVER trial (JACCRO GC-08).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4044-4044
Author(s):  
Yosuke Kito ◽  
Eisuke Inoue ◽  
Yusuke Akamaru ◽  
Masazumi Takahashi ◽  
Jin Matsuyama ◽  
...  
Keyword(s):  
Gastric Cancer ◽  
Clinical Trial ◽  
Survival Time ◽  
Advanced Gastric Cancer ◽  
Real World ◽  
Tumor Response ◽  
Phase Iii ◽  
Clinical Factors ◽  
Real World Data ◽  
World Data

4044 Background: The phase III ATTRACTION-2 trial demonstrated survival benefit of nivolumab (Nivo) as third- or later-line treatment in previously treated advanced gastric or gastroesophageal junction (GEJ) cancer, with response rate (RR) of 11% (Kang YK, et al. Lancet 2017). It has been shown that some tumors grow rapidly after Nivo treatment, but the proportion and survival are still uncertain. We therefore prospectively investigated clinical outcomes from real-world data of Nivo treatment in advanced gastric cancer (GC). Methods: The DELIVER trial was a prospective, multicenter, observational study which assessed patients (pts) with advanced gastric or GEJ adenocarcinoma treated with Nivo alone and ECOG PS 0-2 (UMIN000030850). The aims were to evaluate the efficacy and safety of Nivo treatment in real world. Primary endpoint was overall survival (OS), secondary endpoints were RR, disease control rate (DCR), progression-free survival (PFS), tumor growth rate (TGR) at 1st evaluation, and safety. The sub-group analyses were performed for survival according to tumor response and clinical factors. The survival data was fixed at the timepoint of 1 year after the last enrollment. Results: In 501 pts enrolled from Mar 2018 to Aug 2019, 487 pts were evaluable (median age 70y, 71% male, ECOG PS0/1/2 42/44/14%, no. of prior regimen 1/2/≥3 2/39/59%, 21% HER2-pos, 42% pts with ascites). Median OS was 5.8 months (m) (95%CI 5.3-7.0) with 1y-survival rate of 30%, and median PFS was 1.8 m (95%CI 1.7-2.0), at 454 events for PFS and 389 events for OS. The DCR were 39.4%, and RR was 14.2% in 282 pts with measurable lesions. Median OS and PFS by tumor response (CR/PR/SD/PD) were Not Reached (NR)/NR/11.3/4.1m and NR/11.7/3.8/1.4m, respectively. A sub-group analysis of OS by clinical factors is the following: male/female; 6.5/5.0m ( p= 0.002), tub/por/sig; 8.1/5.4/4.1m ( p< 0.0001), albumin < 3.5/≥3.5; 4.2/8.9m ( p< 0.0001), w/peritoneal mets/w/o; 4.9/8.4m ( p< 0.0001), and w/ascites/w/o; 3.7/8.9m ( p< 0.0001). These findings were also observed in PFS. In 219 evaluable pts for TGR, 20.5% pts were identified as hyper-progressive disease (HPD). An exploratory approach by logistic regression analysis indicated that level of free-T3 in blood before Nivo treatment was higher in the HPD compared to the non-HPD group (2.5 vs. 2.2 pg/ml, p= 0.005). Survival time was comparable between the HPD group and PD without HPD group. Median period from 1st evaluation to death was 2.8 m for HPD, 5.7 m for non-HPD, and 2.4 m for PD at 1st evaluation without HPD. Conclusions: The real-world data of Nivo treatment in advanced GC indicated comparable survival to previous result in a clinical trial. Differences in survival time by tumor response or some clinical factors were observed in Nivo treatment. In addition, our study revealed the rate of HPD and the prognosis in advanced GC pts treated with Nivo. Clinical trial information: UMIN000030850.

scholarly journals Comparing SDTM and FHIR® for Real World Data from Electronic Health Records for Clinical Trial Submissions

2021 ◽  
Author(s):  
Sarah Riepenhausen ◽  
Cornelia Mertens ◽  
Martin Dugas
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Electronic Health Records ◽  
Real World ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Real World Data ◽  
Health Records ◽  
World Data ◽  
Data Submission

Real world data for use in clinical trials is promising. We compared the SDTM for clinical trial data submission with FHIR® for routine documentation. After categorization of variables by relevance, clinically relevant SDTM items were mapped to FHIR®. About 30% in both were seen as clinically relevant. The majority of these SDTM items were mappable to FHIR® Observation resource.

928-P: Dulaglutide Has Higher Adherence and Persistence than Semaglutide and Exenatide QW: 6-Month Follow-Up from U.S. Real-World Data

Diabetes ◽  
2020 ◽  
Vol 69 (Supplement 1) ◽  
pp. 928-P
Author(s):  
REEMA MODY ◽  
MARIA YU ◽  
BAL K. NEPAL ◽  
MANIGE KONIG ◽  
MICHAEL GRABNER
Keyword(s):  
Real World ◽  
Real World Data ◽  
World Data ◽  
Exenatide Qw
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