A review of the application of propensity score methods yielded increasing use, advantages in specific settings, but not substantially different estimates compared with conventional multivariable methods

Stürmer, Til; Joshi, Manisha; Glynn, Robert J.; Avorn, Jerry; Rothman, Kenneth J.; Schneeweiß, Sebastian

doi:10.1016/j.jclinepi.2005.07.004

Cited by 578 publications

(529 citation statements)

References 226 publications

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“…The conclusions from these studies were inconsistent. Two studies 66,68 concluded that estimates obtained from regression analysis are similar to those obtained using propensity scoring. However, two studies 64,67 also came to the opposite conclusion, that estimates obtained from regression analysis and propensity scoring differ significantly.…”

Section: Effectiveness Of Adjustment Methodsmentioning

confidence: 91%

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The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

104

143

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BackgroundThe National Institute for Health and Care Excellence (NICE) commissioned a ‘mock technology appraisal’ to assess whether changes to its methods and processes are needed. This report presents the findings of independent research commissioned to inform this appraisal and the deliberations of a panel convened by NICE to evaluate the mock appraisal.MethodsOur research included reviews to identify issues, analysis methods and conceptual differences and the relevance of alternative decision frameworks, alongside the development of an exemplar case study of chimeric antigen receptor (CAR) T-cell therapy for treating acute lymphoblastic leukaemia.ResultsAn assessment of previous evaluations of regenerative medicines found that, although there were a number of evidential challenges, none was unique to regenerative medicines or was beyond the scope of existing methods used to conceptualise decision uncertainty. Regarding the clinical evidence for regenerative medicines, the issues were those associated with a limited evidence base but were not unique to regenerative medicines: small non-randomised studies, high variation in response and the intervention subject to continuing development. The relative treatment effects generated from single-arm trials are likely to be optimistic unless it is certain that the historical data have accurately estimated the efficacy of the control agent. Pivotal trials may use surrogate end points, which, on average, overestimate treatment effects. To reduce overall uncertainty, multivariate meta-analysis of all available data should be considered. Incorporating indirectly relevant but more reliable (more mature) data into the analysis can also be considered; such data may become available as a result of the evolving regulatory pathways being developed by the European Medicines Agency. For the exemplar case of CAR T-cell therapy, target product profiles (TPPs) were developed, which considered the ‘curative’ and ‘bridging to stem-cell transplantation’ treatment approaches separately. Within each TPP, three ‘hypothetical’ evidence sets (minimum, intermediate and mature) were generated to simulate the impact of alternative levels of precision and maturity in the clinical evidence. Subsequent assessments of cost-effectiveness were undertaken, employing the existing NICE reference case alongside additional analyses suggested within alternative frameworks. The additional exploratory analyses were undertaken to demonstrate how assessments of cost-effectiveness and uncertainty could be impacted by alternative managed entry agreements (MEAs), including price discounts, performance-related schemes and technology leasing. The panel deliberated on the range of TPPs, evidence sets and MEAs, commenting on the likely recommendations for each scenario. The panel discussed the challenges associated with the exemplar and regenerative medicines more broadly, focusing on the need for a robust quantification of the level of uncertainty in the cost-effective estimates and the potential value of MEAs in limiting the exposure of the NHS to high upfront costs and loss associated with a wrong decision.ConclusionsIt is to be expected that there will be a significant level of uncertainty in determining the clinical effectiveness of regenerative medicines and their long-term costs and benefits, but the existing methods available to estimate the implications of this uncertainty are sufficient. The use of risk sharing and MEAs between the NHS and manufacturers of regenerative medicines should be investigated further.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Section: Effectiveness Of Adjustment Methodsmentioning

confidence: 91%

“…A summary of the studies that have looked at methods of adjustment for confounding bias in NRSs and how reliable they are [62][63][64][65][66][67][68][69][70] is presented in Appendix 3. Overall, it is unclear which methods are most appropriate in certain circumstances and further research is needed.…”

Section: Adjustment For Bias In Non-randomised Studiesmentioning

confidence: 99%

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Hettle

Corbett

Hinde

et al. 2017

Health Technol Assess

104

143

View full text Add to dashboard Cite

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“…Indeed, systematic differences between statin users and non-users have been found to influence estimates of the effect of exposure to the drugs [17,18]. A propensity score is a measure of how likely an individual is to be prescribed a particular drug.Use of propensity scores offers a method of reducing selection bias and confounding in pharmacoepidemiological studies [19,20]. Propensity scores were estimated for all patients using conditional logistic regression, with statin prescription at the index date as the outcome, taking the matching into account.…”

Section: Discussionmentioning

confidence: 99%

Effect of statins on a wide range of health outcomes: a cohort study validated by comparison with randomized trials

Smeeth

Douglas

Hall

et al. 2008

Brit J Clinical Pharma

213

201

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WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT• The effect of statins on cardiovascular outcomes is well established.• Effects on numerous other health outcomes have been claimed, but results have been inconsistent.• Previous observational studies may have been affected by selection bias and confounding. WHAT THIS STUDY ADDS• This study was larger and more statistically powerful than the combined total of data from all randomized trials of statins undertaken to date.• The validity of the study is supported by findings for vascular outcomes being comparable to those observed in large randomized controlled trials.• There was little evidence to support effects of statins beyond their established effects on cardiovascular disease. AIMSTo assess the effect of statins on a range of health outcomes. METHODSWe undertook a population-based cohort study to assess the effect of statins on a range of health outcomes using a propensity score-based method to control for differences between people prescribed and not prescribed statins. We validated our design by comparing our results for vascular outcomes with the effects established in large randomized trials. The study was based on the United Kingdom Health Improvement Network database that includes the computerized medical records of over four and a half million patients. RESULTSPeople who initiated treatment with a statin (n = 129 288) were compared with a matched sample of 600 241 people who did not initiate treatment, with a median follow-up period of 4.4 years. Statin use was not associated with an effect on a wide range of outcomes, including infections, fractures, venous thromboembolism, gastrointestinal haemorrhage, or on specific eye, neurological or autoimmune diseases. A protective effect against dementia was observed (hazard ratio 0.80, 99% confidence interval 0.68, 0.95). There was no effect on the risk of cancer even after Ն8 years of follow-up. The effect sizes for statins on vascular end-points and mortality were comparable to those observed in large randomized trials, suggesting bias and confounding had been well controlled for. CONCLUSIONSWe found little evidence to support wide-ranging effects of statins on health outcomes beyond their established beneficial effect on vascular disease.

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“…Propensity scores are increasingly being used in pharmacoepidemiologic studies to control for confounding (15). The propensity score is calculated as the probability of receiving one treatment compared with another, i.e., a TNF␣ antagonist versus a nonbiologic DMARD.…”

Section: Control For Potential Confoundingmentioning

confidence: 99%

“…If more covariates can be included in the propensity score model as compared with the conventional outcomes regression model (which is limited by the number of outcomes [16]), then adjustments in the propensity score may provide better adjustment for otherwise incompletely measured confounders. However, in practice, propensity score analyses have almost never produced results significantly different from those produced by conventional outcomes models in pharmacoepidemiology (15).…”

Section: Control For Potential Confoundingmentioning

confidence: 99%

The risk of infection associated with tumor necrosis factor α antagonists: Making sense of epidemiologic evidence

Solomon¹,

Lunt²,

Schneeweiß³

2008

Arthritis & Rheumatism

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A review of the application of propensity score methods yielded increasing use, advantages in specific settings, but not substantially different estimates compared with conventional multivariable methods

Abstract: Publication of results based on propensity score methods has increased dramatically, but there is little evidence that these methods yield substantially different estimates compared with conventional multivariable methods.

Cited by 578 publications

References 226 publications

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

Effect of statins on a wide range of health outcomes: a cohort study validated by comparison with randomized trials

The risk of infection associated with tumor necrosis factor α antagonists: Making sense of epidemiologic evidence

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