What are the challenges for building real world comparative cohorts in oncology?

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19285-e19285
Author(s):  
Nikita Jeswani ◽  
Amanda McDonell ◽  
Finlay MacDougall ◽  
Jeff Paul Hodge
Keyword(s):  
Language Processing ◽  
Real World ◽  
International Prognostic Index ◽  
Evaluation Criteria ◽  
Prognostic Index ◽  
Cost Effective ◽  
Patient Characteristics ◽  
Outcome Evaluation ◽  
Real World Data ◽  
Indolent Lymphomas

e19285 Background: There has been a proliferation of single arm trials in oncology being presented to regulators. An external comparator using real world data (RWD) can help establish context to trial results and direct comparison to the treatment arm by mirroring the inclusion/exclusion criteria for the trial and examining trial outcomes in an RWD external comparator cohort. RWD has successfully supported some regulatory submissions, although there have been failures as well, with comparability of patients and endpoints under scrutiny. Methods: IQVIA has partnered with life sciences companies on > 20 external comparator projects from 2017-present using a mix of data acquisition strategies (e.g., chart review, database extraction) and methods (e.g., real-world cohort vs. real-world benchmark, propensity matching vs. inverse probability treatment weighting). We collated, reviewed, and synthesised the challenges and risk mitigations documented throughout study execution in aggressive and indolent lymphomas, multiple myeloma, Merkel cell carcinoma, and synovial sarcoma. Results: Patient characteristics used to assess trial eligibility (e.g., biomarker expression, International Prognostic Index, ECOG status) and outcomes (e.g., response rate) are not often captured in a structured format in RWD or recorded in routine clinical practice. As a result, there is a greater reliance on chart reviews today over database extractions to fulfil external comparator requirements, though in some diseases, databases have proven to be a faster and more cost-effective solution. Conclusions: To improve the success of external comparator studies, trial design should be informed by existing RWD so that relevant real-world endpoints and outcome evaluation criteria are included alongside RCT standards. Advances are needed to facilitate RWD capture that matches trial data more closely. This could include bespoke data collaborations, biobank linkage, and natural language processing to drive study execution in databases in the future.

Assessment and design of real world driving cycles targeted to the calibration of vehicles with electrified powertrain

2021 ◽  
pp. 146808742110387
Author(s):  
Stylianos Doulgeris ◽  
Zisimos Toumasatos ◽  
Maria Vittoria Prati ◽  
Carlo Beatrice ◽  
Zissis Samaras
Keyword(s):  
Real World ◽  
Simulation Models ◽  
Cost Effective ◽  
Operational Parameters ◽  
Virtual Design ◽  
Passenger Cars ◽  
Real World Data ◽  
Vehicle Simulation ◽  
Driving Cycles ◽  
The Impact

Vehicles’ powertrain electrification is one of the key measures adopted by manufacturers in order to develop low emissions vehicles and reduce the CO2 emissions from passenger cars. High complexity of electrified powertrains increases the demand of cost-effective tools that can be used during the design of such powertrain architectures. Objective of the study is the proposal of a series of real-world velocity profiles that can be used during virtual design. To that aim, using three state of the art plug-in hybrid vehicles, a combined experimental, and simulation approach is followed to derive generic real-world cycles that can be used for the evaluation of the overall energy efficiency of electrified powertrains. The vehicles were tested under standard real driving emissions routes, real-world routes with reversed order (compared to a standard real driving emissions route) of urban, rural, motorway, and routes with high slope variation. To enhance the experimental activities, additional virtual mission profiles simulated using vehicle simulation models. Outcome of the study consists of specific driving cycles, designed based on standard real-world route, and a methodology for real-world data analysis and evaluation, along with the results from the assessment of the impact of different operational parameters on the total electrified powertrain.

Abstract W P273: The Cost-Effectiveness of Telestroke Varies by Implementation Cost and Stroke Severity: Real World Data From a Pacific Northwest Network

Stroke ◽  
2015 ◽  
Vol 46 (suppl_1) ◽  
Author(s):  
Elizabeth Baraban ◽  
Richard Nelson ◽  
Alexandra Lesko ◽  
Jennifer Majersik ◽  
Archit Bhatt ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Pacific Northwest ◽  
Real World ◽  
Cost Effective ◽  
Stroke Severity ◽  
Real World Data ◽  
World Data ◽  
Life Years ◽  
Implementation Costs ◽  
The Cost

Objective: An obstacle for community hospitals in joining a telestroke network is often the cost of implementation. Yet, previous analyses examining the cost and cost-effectiveness have only used estimates from the literature. Using real-world data from a Pacific Northwest telestroke network, we examined the cost-effectiveness of telestroke for spokes by level of financial responsibility for these costs and how this changes with patient stroke severity. Methods: We constructed a decision analytic model and parameterized it using patient-level clinical and financial data from the Providence Telestroke Network (PTN) pre and post telestroke implementation. Data included patients presenting at 17 spokes within 4.5 hours of symptom onset. Probability inputs included observed IV-tPA treatment rates, transfer status and hospital costs and reimbursements. Effectiveness, measured as quality-adjusted life years (QALYs), and cost per patient were used to calculate incremental cost effectiveness ratios (ICERs). ICER’s of <$50,000-$120,000/QALY are considered cost-effective. Outcomes were generated overall and separately by admit NIHSS, defined as low (0-10), medium (11-20) and high (>20) and percentage of implementation costs paid by spokes (0%, 50%, 100%). Results: Data for 594 patients, 105 pre- and 489 post-implementation, were included. See Table 1. Conclusions: Our results support previous theoretic models showing good value, overall. However, costs and ICERs varied by stroke severity, with telestroke being most cost-effective for severe strokes. Telestroke was least cost effective if spokes paid for half or more of implementation costs.

scholarly journals PDB66 - INSULIN GAP IN TYPE 2 DIABETES: IS IT COST EFFECTIVE TO INTENSIFY TREATMENT? EVIDENCE FROM PORTUGAL USING REAL WORLD DATA

2018 ◽  
Vol 21 ◽  
pp. S129
Author(s):  
F. Fiorentino ◽  
R. Ascenção ◽  
J. Costa ◽  
M. Gouveia ◽  
M. Borges
Keyword(s):  
Type 2 Diabetes ◽  
Real World ◽  
Cost Effective ◽  
Real World Data ◽  
World Data

P0816CLINICAL CHARACTERISTICS AND EGFR AND UACR DISTRIBUTION ACCORDING TO THE 2012 KDIGO CKD CLASSIFICATION: A REPORT FROM THE US DISCOVER CKD COHORT

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Juan Jose Garcia Sanchez ◽  
Juan Jesus Carrero ◽  
Supriya Kumar ◽  
Roberto Pecoits-Filho ◽  
Glen James ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Real World ◽  
Patient Characteristics ◽  
Study Cohort ◽  
Diagnostic Code ◽  
Inclusion Criteria ◽  
Real World Data ◽  
Electronic Health Record Data ◽  
World Data ◽  
Co Morbidity

Abstract Background and Aims In 2012, the Kidney Disease Improving Global Outcomes (KDIGO) guidelines recommended categorising and prognosticating chronic kidney disease (CKD) based on estimated glomerular filtration rate (eGFR) and urine albumin to creatinine ratio (UACR). Contemporary studies describing the prevalence and characteristics of patients with CKD categorised according KDIGO 2012 and how studies of new pharmacotherapies relate to these categories are scarce. One such new therapy class of key interest are the sodium glucose co-transporter 2 inhibitors (SGLT-2i), shown to delay the progression to renal failure and prevent cardiovascular/renal death in patients with CKD. We aimed to describe patient characteristics and the prevalence of CKD according to the 2012 KDIGO categories in a large real-world US cohort of patients with CKD (part A). We also describe a subset of the population according to the DAPA-CKD trial inclusion criteria (eGFR [25-75ml/min/1.73m2] and UACR [200-5000mg/g]) (part B). Method DISCOVER-CKD is an international observational study in patients with CKD. The DISCOVER-CKD retrospective US cohort of patients was extracted using real-world data from the integrated Limited Claims and Electronic Health Record data (IBM Health, Armonk, NY) and HealthVerity. Patients were aged ≥18 years, with ≥1 UACR measure. For part A, required first diagnostic code of CKD (Stages 3A, 3B, 4, 5, or ESRD) or two eGFR of &lt;75 mL/min/1.73 m2 recorded at least 90 days apart and for part B, two measures of eGFR 25-75 mL/min/1.73 m2 recorded at least 90 days apart between 1st January 2008 and September 2018. Index date was diagnostic code or 2nd eGFR. The first UACR, recorded +/-12 months of index, was used to categorise patients. Descriptive analyses were used to summarise prevalence and patient characteristics. Results Of the overall study cohort (N=4330, 49.1% women, mean age 65.3±10.64 years), by KDIGO categories (part A): 85.7% (n=3601) had normal to mildly increased albuminuria, 11.0% (n=463) had moderately increased albuminuria and 3.3% (n=137) had severely increased albuminuria (Figure 1). 4.6% (n=193) fulfilled DAPA-CKD trial inclusion criteria (part B). In both populations, the most common comorbidities were hypertension (HTN, 73.0% for both) and type 2 diabetes (T2D, 57.6% and 56.2%, respectively). Anti-hypertensive drugs were frequently used (76.4% and 76.9%, respectively). Conclusion This study, utilising real-world data, adds to the scarcity of knowledge reporting the characteristics of patients with CKD in different eGFR and UACR strata according to the KDIGO 2012 definitions. We observed a trend in higher UACR in the group of patients with lower eGFR and report a high prevalence of T2D and HTN in the study population, demonstrating the high co-morbidity burden in patients, for whom new therapies may be beneficial.

550 Real World Treatment Trends for Patients TBSA ≤ 20%: Evaluating Important Shifts in Care and Budget Impact of ASCS Adoption

2020 ◽  
Vol 41 (Supplement_1) ◽  
pp. S115-S115
Author(s):  
James H Holmes ◽  
Stacey Kowal ◽  
Cheryl P Ferrufino
Keyword(s):  
Survey Data ◽  
Resource Use ◽  
Real World ◽  
Budget Impact ◽  
Patient Characteristics ◽  
Real World Data ◽  
Treatment Pathways ◽  
World Data ◽  
Burn Care ◽  
The Impact

Abstract Introduction Treatment pathways in burn care are typically determined based on burn center (BC) and patient characteristics, although decisions may be influenced by anecdotal experience, personal preference, and hospital policies/purchasing decisions. Health economic (HE) evaluations can support improved decision-making, identifying the most cost-effective interventions for tailored care. A novel burn care model (BEACON) was developed with burn surgeons over several years and validated through numerous publications, including an assessment of the HE impacts of autologous skin cell suspension (ASCS) use for definitive burn closure. To ensure that BEACON accurately represents the current state of care, it is vital to update data that underpins model projections. This study collected real world data on practice patterns and patient outcomes for the most commonly seen burns (TBSA ≤ 20%) to update the current understanding of standard of care (SOC) costs and outcomes and to refine estimates on the impact of ASCS use in TBSA ≤ 20% patients. Methods Data was collected from a 10% sample of BCs, including: BC and patient characteristics, resource use, inpatient costs, and length of stay (LOS). NBR based inputs in BEACON were updated to reflect survey data for patients with TBSA ≤ 20%, with the ability to view data as a national aggregate sample and across BC characteristics. BEACON estimates patient and BC costs and outcomes across a spectrum of patient profiles (age, gender, inhalation injury, comorbidity status, burn depth, TBSA) and combines information on each patient profile to understand annual budget impact. Key outcomes were compared across the survey sample and published NBR trends. Using the updated BEACON, the BC budget impact of ASCS in burns TBSA ≤ 20% was assessed. Results The survey was collected from 16+ BCs, focusing on inpatient encounters in 2018. LOS was lower than NBR estimates, with some centers reporting LOS per %TBSA far below 1 d/%TBSA. Using the detailed bottom-up estimation of cost from BEACON with survey data, trends suggest total hospital costs for SOC are lower than published NBR charges given shorter LOS and updated cost and resource use assumption. Conclusions Compared to NBR 8.0, contemporary data suggests that fewer small TBSA burns are being treated in the inpatient setting; those treated have a LOS below NBR estimates. When using real world data, the impact of ASCS use in burns TBSA ≤ 20% was still calculated to be cost saving to a BC overall, given reductions in LOS and number of definitive closure procedures. Incorporating ASCS into appropriate TBSA ≤ 20% procedures can still result in a positive financial impact for BCs. Applicability of Research to Practice

scholarly journals Evaluating the Cost-Effectiveness of Early Compared with Late or No Biologic Treatment to Manage Crohn’s Disease using Real-World Data

2019 ◽  
Vol 14 (4) ◽  
pp. 490-500 ◽  
Author(s):  
Nadia Pillai ◽  
Judith E Lupatsch ◽  
Mark Dusheiko ◽  
Matthias Schwenkglenks ◽  
Michel Maillard ◽  
...  
Keyword(s):  
Crohn’S Disease ◽  
Cost Effectiveness ◽  
Crohn's Disease ◽  
Health System ◽  
Real World ◽  
Transition Probabilities ◽  
Cost Effective ◽  
Real World Data ◽  
World Data ◽  
The Cost

Abstract Background and Aims We evaluated the cost-effectiveness of early [≤2 years after diagnosis] compared with late or no biologic initiation [starting biologics &gt;2 years after diagnosis or no biologic use] for adults with Crohn’s disease in Switzerland. Methods We developed a Markov cohort model over the patient’s lifetime, from the health system and societal perspectives. Transition probabilities, quality of life, and costs were estimated using real-world data. Propensity score matching was used to ensure comparability between patients in the early [intervention] and late/no [comparator] biologic initiation strategies. The incremental cost-effectiveness ratio [ICER] per quality-adjusted life year [QALY] gained is reported in Swiss francs [CHF]. Sensitivity and scenario analyses were performed. Results Total costs and QALYs were higher for the intervention [CHF384 607; 16.84 QALYs] compared with the comparator [CHF340 800; 16.75 QALYs] strategy, resulting in high ICERs [health system: CHF887 450 per QALY; societal: CHF449 130 per QALY]. In probabilistic sensitivity analysis, assuming a threshold of CHF100 000 per QALY, the probability that the intervention strategy was cost-effective was 0.1 and 0.25 from the health system and societal perspectives, respectively. In addition, ICERs improved when we assumed a 30% reduction in biologic prices [health system: CHF134 502 per QALY; societal: intervention dominant]. Conclusions Early biologic use was not cost-effective, considering a threshold of CHF100 000 per QALY compared with late/no biologic use. However, early identification of patients likely to need biologics and future drug price reductions through increased availability of biosimilars may improve the cost-effectiveness of an early treatment approach.

scholarly journals Three handy tips and a practical guide to improve your propensity score models

RMD Open ◽  
2019 ◽  
Vol 5 (1) ◽  
pp. e000953 ◽  
Author(s):  
Sytske Anne Bergstra ◽  
Alexandre Sepriano ◽  
Sofia Ramiro ◽  
Robert Landewé
Keyword(s):  
Real World ◽  
Observational Studies ◽  
Propensity Scores ◽  
Patient Characteristics ◽  
Confounding By Indication ◽  
Real World Data ◽  
Treatment Allocation ◽  
Popular Method ◽  
Clinical Scenarios ◽  
Treatment Responses

Real-world data are increasingly available to investigate ‘real-world’ safety and efficacy. However, since treatment in observational studies is not randomly allocated, confounding by indication may occur, in which differences in patient characteristics may influence both treatment choices and treatment responses. A popular method to adjust for this type of bias is the use of propensity scores (PS). The PS is a score between 0 and 1 that reflects the likelihood per patient of receiving one of the treatment categories of interest conditional on a set of variables. At least in theory, in patients with similar PS, the treatment prescribed will be independent of these variables (pseudorandomisation). But researchers using PS sometimes fail to recognise important methodological flaws which can lead to spurious conclusions. These include perfect prediction of treatment allocation, untied observations and lack of generalisability due to oversimplification of complex clinical scenarios. In this viewpoint we will discuss the most commonly encountered flaws and provide a stepwise description on the estimation and use of PS, such that in future publications these flaws can be avoided.

Revised International Prognostic Index (R-IPI) Is a Better Predictor of Outcome Than the Standard IPI for Patients with Diffuse Large B-Cell Lymphoma (DLBCL) Treated with Rituximab and CHOP (R-CHOP).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 492-492 ◽  
Author(s):  
Laurie H. Sehn ◽  
Mukesh Chhanabhai ◽  
Catherine Fitzgerald ◽  
Karamjit Gill ◽  
Paul Hoskins ◽  
...  
Keyword(s):  
Overall Survival ◽  
International Prognostic Index ◽  
Young Patients ◽  
Prognostic Index ◽  
Progression Free Survival ◽  
B Cell Lymphoma ◽  
Patient Characteristics ◽  
Research Database ◽  
Clinical Tool ◽  
Field Radiation

Abstract Background: The IPI, which was developed prior to the availability of rituximab, remains the primary clinical tool used to predict outcome for patients with DLBCL. Since clinical trials investigating the use of rituximab and chemotherapy in this patient population have been confined to either elderly or young patients exclusively, the utility of the IPI in the era of immuno-chemotherapy remains unknown. Methods: We performed a retrospective analysis to assess the predictive value of the IPI in an unselected population of patients with DLBCL treated with R-CHOP. Patients were identified using the Lymphoid Cancer Research Database of the British Columbia (BC) Cancer Agency. We included all patients ≥16 years of age who were newly diagnosed with DLBCL prior to Jan 15, 2005, and were treated in BC with an R-CHOP protocol. Patients were excluded if they were HIV positive, had evidence of an active second malignancy or an underlying indolent lymphoma. Results: 365 patients were identified. Patient characteristics were as follows: median age 61 y (16–90); male, 61%; advanced stage III/IV, 59%; elevated LDH, 54%; PS≥2, 40%; &gt;1 extranodal site, 35%. Central pathology review was performed on 95% of patients; 324 DLBCL, 36 PMBCL, 5 other. All patients received R-CHOP; 9% were treated with R-CHOP x 3 and involved field radiation therapy for limited stage disease, remaining patients received 6–8 cycles of R-CHOP. Median follow-up for living patients is 22 months. Overall, the IPI remains predictive for both progression-free survival (p&lt;0.0001) and overall survival (p&lt;0.0001). However, the IPI no longer permits separation between the two low risk subgroups (low v low-intermediate) or between the two high risk subgroups (high-intermediate v high). (see Table) An alternate grouping of the IPI is proposed (R-IPI) that identifies three distinct prognostic groups. (see Table and Figure) Conclusions: The IPI remains predictive in the era of immuno-chemotherapy, but the R-IPI provides a simplified and more accurate prediction of outcome. The IPI factors can no longer be used to identify a group with less than a 50% chance of survival. Overall Survival According to Revised IPI (R-IPI) Overall Survival According to Revised IPI (R-IPI)

Dose-intensive cyclophosphamide, etoposide, cisplatin reinduction for relapsed/refractory aggressive non-Hodgkin lymphoma (r/r-aNHL).

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 6548-6548
Author(s):  
Jan-Willem Henning ◽  
Qiuli Duan ◽  
Nizar J. Bahlis ◽  
Andrew Daly ◽  
Peter Duggan ◽  
...  
Keyword(s):  
Stem Cell ◽  
Univariate Analysis ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Progression Free Survival ◽  
Patient Characteristics ◽  
Non Hodgkin Lymphoma ◽  
Cd34 Cells ◽  
Cell Mobilization ◽  
N 93

6548 Background: Approximately 2/3 r/r-aNHL patients (pts) respond to salvage R-ICE or R-DHAP, 1/2 proceed to autologous stem cell transplantation (ASCT), and 1/3 achieve 3 year (yr) progression-free survival (PFS); however, PFS is only 20% if prior Rituximab, time to progression (TTP)<1yr, or age-adjusted International Prognostic Index (aaIPI)=2-3 [JCO 2010;28: 4184-90]. Since 1995, we re-induced poor prognosis r/r-aNHL with dose-intensive Cyclophosphamide 5.25g/m2, Etoposide 1.05g/m2 and Cisplatin 105mg/m2 (DICEP), G-CSF days (d) 14-19, and apheresis d19,20, or 21. Rituximab was added d0,7 after 2006. Methods: We retrospectively analyzed 113 consecutive transplant eligible r/r-aNHL pts [diffuse large B-cell=95, transformed=9, peripheral T-cell=6, other=3] who received one cycle of DICEP (n=93) or R-DICEP (n=20) from 1995-2009. Patient characteristics included: median age=49yr (22-69); primary refractory=68; TTP<1yr=85; elevated LDH=60; ECOG 2-4=42; aaIPI 2-3=59; bulk>10cm=26. Results: Of 113 pts, 77% responded to DICEP and 90% (102) proceeded to ASCT. The median CD34+ cells collected was 19x106/kg (0.3-142). Early treatment-related mortality (TRM) occurred in 3 pts (2.7%), and 4 others developed late second cancers (MDS/AML=2). With 94 months median follow-up (26-194), 5 and 10yr OS rates for all 113pts are 48% and 41%, and PFS rates are 42% and 37%, respectively. 5 year PFS rates for ASCT vs no-ASCT are 46% vs 9%, for relapse aaIPI=0-1 vs aaIPI=2-3 are 53.3% vs 32.1% (p=0.01), and for TTP>1yr vs <1yr are 63.9% vs 35.2% (p=0.009). Other predictors of inferior PFS in univariate analysis were elevated LDH, ECOG 2-4, no response to DICEP; however, PFS for 27 pts who failed prior Rituximab-chemotherapy (56%) was similar to other 86 pts (38%) (logrank p=0.09). Predictors of PFS and OS in multivariate analysis include: TTP<1yr, elevated LDH, bulk, no response to DICEP. Conclusions: (R)DICEP is an effective re-induction regimen for r/r-aNHL, leading to excellent stem cell mobilization and a high chance of proceeding to ASCT. Long-term PFS and OS rates compare favourably to reports of other re-induction regimens, and a prospective multicentre trial is warranted.

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