Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals

ObjectivesTo quantify and characterise the usage of expanded access (EA) data in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs). EA offers patients who are ineligible for clinical trials or registered treatment options, access to investigational therapies. Although EA programmes are increasingly used to collect real-world data, it is unknown if and how these date are used in NICE health technology assessments.DesignCross-sectional study of NICE appraisals (2010–2020). We automatically downloaded and screened all available appraisal documentation on NICE website (over 8500 documents), searching for EA-related terms. Two reviewers independently labelled the EA usage by disease area, and whether it was used to inform safety, efficacy and/or resource use. We qualitatively describe the five appraisals with the most occurrences of EA-related terms.Primary outcome measureNumber of TAs that used EA data to inform safety, efficacy and/or resource use analyses.ResultsIn 54.2% (206/380 appraisals), at least one reference to EA was made. 21.1% (80/380) of the TAs used EA data to inform safety (n=43), efficacy (n=47) and/or resource use (n=52). The number of TAs that use EA data remained stable over time, and the extent of EA data utilisation varied by disease area (p=0.001).ConclusionNICE uses EA data in over one in five appraisals. In synthesis with evidence from well-controlled trials, data collected from EA programmes may meaningfully inform cost-effectiveness modelling.

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Real-World Data Study Focused on Chronic Lymphocytic Leukemia (CLL) Patient Treatment Options and Their Effectiveness

Case Medical Research ◽

10.31525/ct1-nct03881592 ◽

2019 ◽

Author(s):

Keyword(s):

Chronic Lymphocytic Leukemia ◽

Real World ◽

Treatment Options ◽

Lymphocytic Leukemia ◽

Patient Treatment ◽

Real World Data ◽

World Data

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R WE ready for reimbursement? A round up of developments in RWE relating to health technology assessment: part 2

Journal of Comparative Effectiveness Research ◽

10.2217/cer-2021-0144 ◽

2021 ◽

Author(s):

Alex Simpson ◽

Sreeram V Ramagopalan

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Real World ◽

Health Technology ◽

Health Inequity ◽

Real World Data ◽

World Data

In this round up, we cover how COVID-19 has been beneficial for improved access to real-world data, as well as how real-world data can be used to address health inequity, an area of increasing interest for health technology assessment.

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Evaluating renal cell carcinoma treatment options using real world data: Findings from an oncology survey across seven countries

Annals of Oncology ◽

10.1093/annonc/mdx711.002 ◽

2017 ◽

Vol 28 ◽

pp. xi6

Author(s):

S. Mpima ◽

S. Menon ◽

P. Nasuti ◽

H. Mistry

Keyword(s):

Renal Cell Carcinoma ◽

Cell Carcinoma ◽

Real World ◽

Renal Cell ◽

Treatment Options ◽

Real World Data ◽

World Data

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Real-world cost-effectiveness of pertuzumab (P) with trastuzumab + chemo (T+Chemo) in patients (pts) with metastatic breast cancer (MBC): A population-based retrospective cohort study by the Canadian Real-world Evidence for Value in Cancer Drugs (CanREValue) collaboration.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.1048 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. 1048-1048

Author(s):

Wei Fang Dai ◽

Jaclyn Marie Beca ◽

Chenthila Nagamuthu ◽

Ning Liu ◽

Maureen E. Trudeau ◽

...

Keyword(s):

Breast Cancer ◽

Cost Effectiveness ◽

Incremental Cost ◽

Real World ◽

Retrospective Cohort ◽

Health Technology ◽

Population Based ◽

Net Benefit ◽

Real World Data ◽

World Data

1048 Background: Addition of P to T+chemo for MBC pts has been shown to improve overall survival (OS) in a pivotal randomized trial (hazard ratio [HR] = 0.66, 95% CI: 0.52, 0.84) (Baselga et al., NEJM 2012). In Canada, the manufacturer submission to the health technology assessment agency estimated that P produced 0.64 life years gained (LYG) with an incremental cost-effectiveness ratio (ICER) of $187,376/LYG over 10 years (CADTH-pCODR, 2013). This retrospective cohort analysis aims to determine the comparative real-world population-based effectiveness and cost-effectiveness of P among MBC pts in Ontario, Canada. Methods: MBC pts were identified from the Ontario Cancer Registry and linked to the New Drug Funding Program database to identify receipt of treatment between 1/1/2008 and 3/31/2018. Cases received P-T-chemo after universal public funding of P (Nov 2013) and controls received T-chemo before. Demographic (age, socioeconomic, rurality) and clinical (comorbidities, prior adjuvant treatments, prior breast cancer surgery, prior radiation, stage at diagnosis, ER/PR status) characteristics were identified from linked admin databases balanced between cases and controls using propensity score matching. Kaplan-Meier methods and Cox regressions accounting for matched pairs were used to estimate median OS and HR. 5-year mean total costs from the public health system perspective were estimated from admin claims databases using established direct statistical methods and adjusted for censoring of both cost and effectiveness using inverse probability weighting. ICERs and 95% bootstrapped CIs were calculated, along with incremental net benefit (INB) at various willingness-to-pay values using net benefit regression. Results: We identified 1,823 MBC pts with 912 cases and 911 controls (mean age = 55 years), of which 579 pairs were matched. Cases had improved OS (HR = 0.66; 95% CI: 0.57, 0.78), with median 3.4 years, compared to controls median OS of 2.1. P provided an additional 0.63 (95% CI: 0.48 – 0.84) LYG at an incremental cost of $196,622 (95% CI: $180,774, $219,172), with a mean ICER = $312,147/LYG (95% CI: $260,752, $375,492). At threshold of $100,000/LYG, the INB was -$133,632 (95% CI: -$151,525, -$115,739) with < 1% probability of being cost-effective. Key drivers of incremental cost increase between groups included drug and cancer clinic costs. Conclusions: The addition of P to T-chemo for MBC increased survival but at significant costs. The ICER based on direct real-world data was higher than the initial economic model due to higher total costs for pts receiving P. This study demonstrated feasibility to derive ICER from person-level real-world data to inform cancer drug life-cycle health technology reassessment.

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VP08 Real-World Data Use In Health Technology Assessments: A Comparison Of Five Health Technology Assessment Agencies

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317003051 ◽

2017 ◽

Vol 33 (S1) ◽

pp. 149-150

Author(s):

Amr Makady ◽

Ard van Veelen ◽

Anthonius de Boer ◽

Hans Hillege ◽

Olaf Klunger ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

Real World ◽

Relative Effectiveness ◽

Internal Validity ◽

Health Technology ◽

Real World Data ◽

World Data ◽

The Impact

INTRODUCTION:Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.METHODS:Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.RESULTS:In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).CONCLUSIONS:Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.

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

Journal of Burn Care & Research ◽

10.1093/jbcr/iraa024.178 ◽

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

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Application of Health Technology Assessment (HTA) to Evaluate New Laboratory Tests in a Health System: A Case Study of Bladder Cancer Testing

Academic Pathology ◽

10.1177/2374289520968225 ◽

2020 ◽

Vol 7 ◽

pp. 237428952096822

Author(s):

Erik J. Landaas ◽

Ashley M. Eckel ◽

Jonathan L. Wright ◽

Geoffrey S. Baird ◽

Ryan N. Hansen ◽

...

Keyword(s):

Bladder Cancer ◽

Pilot Study ◽

Health Technology Assessment ◽

Technology Assessment ◽

Real World ◽

Health Technology ◽

Test System ◽

Real World Data ◽

World Data

We describe the methods and decision from a health technology assessment of a new molecular test for bladder cancer (Cxbladder), which was proposed for adoption to our send-out test menu by urology providers. The Cxbladder health technology assessment report contained mixed evidence; predominant concerns were related to the test’s low specificity and high cost. The low specificity indicated a high false-positive rate, which our laboratory formulary committee concluded would result in unnecessary confirmatory testing and follow-up. Our committee voted unanimously to not adopt the test system-wide for use for the initial diagnosis of bladder cancer but supported a pilot study for bladder cancer recurrence surveillance. The pilot study used real-world data from patient management in the scenario in which a patient is evaluated for possible recurrent bladder cancer after a finding of atypical cytopathology in the urine. We evaluated the type and number of follow-up tests conducted including urine cytopathology, imaging studies, repeat cystoscopy evaluation, biopsy, and repeat Cxbladder and their test results. The pilot identified ordering challenges and suggested potential use cases in which the results of Cxbladder affected a change in management. Our health technology assessment provided an objective process to efficiently review test performance and guide new test adoption. Based on our pilot, there were real-world data indicating improved clinician decision-making among select patients who underwent Cxbladder testing.

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Comparative analysis of methods for identifying multimorbidity patterns: a study of ‘real-world’ data

BMJ Open ◽

10.1136/bmjopen-2017-018986 ◽

2018 ◽

Vol 8 (3) ◽

pp. e018986 ◽

Cited By ~ 12

Author(s):

Albert Roso-Llorach ◽

Concepción Violán ◽

Quintí Foguet-Boreu ◽

Teresa Rodriguez-Blanco ◽

Mariona Pons-Vigués ◽

...

Keyword(s):

Real World ◽

Hierarchical Cluster ◽

International Classification Of Diseases ◽

Cross Sectional Study ◽

Real World Data ◽

Cross Sectional ◽

World Data ◽

Data Usage ◽

Classification Of Diseases ◽

Depth Study

ObjectiveThe aim was to compare multimorbidity patterns identified with the two most commonly used methods: hierarchical cluster analysis (HCA) and exploratory factor analysis (EFA) in a large primary care database. Specific objectives were: (1) to determine whether choice of method affects the composition of these patterns and (2) to consider the potential application of each method in the clinical setting.DesignCross-sectional study. Diagnoses were based on the 263 corresponding blocks of the International Classification of Diseases version 10. Multimorbidity patterns were identified using HCA and EFA. Analysis was stratified by sex, and results compared for each method.Setting and participantsElectronic health records for 408 994 patients with multimorbidity aged 45–64 years in 274 primary health care teams from 2010 in Catalonia, Spain.ResultsHCA identified 53 clusters for women, with just 12 clusters including at least 2 diagnoses, and 15 clusters for men, all of them including at least two diagnoses. EFA showed 9 factors for women and 10 factors for men. We observed differences by sex and method of analysis, although some patterns were consistent. Three combinations of diseases were observed consistently across sex groups and across both methods: hypertension and obesity, spondylopathies and deforming dorsopathies, and dermatitis eczema and mycosis.ConclusionsThis study showed that multimorbidity patterns vary depending on the method of analysis used (HCA vs EFA) and provided new evidence about the known limitations of attempts to compare multimorbidity patterns in real-world data studies. We found that EFA was useful in describing comorbidity relationships and HCA could be useful for in-depth study of multimorbidity. Our results suggest possible applications for each of these methods in clinical and research settings, and add information about some aspects that must be considered in standardisation of future studies: spectrum of diseases, data usage and methods of analysis.

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