If estimator assumptions are valid, estimates across outcomes can be used to assess the optimality of treatment rates in a study population. However, because true treatment effect parameters are sensitive to correlations of treatment effects across outcomes, decision makers should be cautious about generalizing estimates to other populations.
This study used Monte Carlo simulations to examine the ability of the two-stage least squares (2SLS) estimator and two-stage residual inclusion (2SRI) estimators with varying forms of residuals to estimate the local average and population average treatment effect parameters in models with binary outcome, endogenous binary treatment, and single binary instrument. The rarity of the outcome and the treatment was varied across simulation scenarios. Results showed that 2SLS generated consistent estimates of the local average treatment effects (LATE) and biased estimates of the average treatment effects (ATE) across all scenarios. 2SRI approaches, in general, produced biased estimates of both LATE and ATE under all scenarios. 2SRI using generalized residuals minimized the bias in ATE estimates. Use of 2SLS and 2SRI is illustrated in an empirical application estimating the effects of long-term care insurance on a variety of binary health care utilization outcomes among the near-elderly using the Health and Retirement Study.
Researchers are increasingly opting for nonlinear 2SRI to estimate treatment effects in models with binary and otherwise inherently nonlinear dependent variables, believing that it produces generally unbiased and consistent estimates. This research shows that positive properties of nonlinear 2SRI rely on assumptions about the relationships between treatment effect heterogeneity and choice.
Study Design Retrospective study. Background Alternative models of care that allow patients to choose direct access to physical therapy have shown promise in terms of cost reduction for neck and back pain. However, real-world exploration within the US health care system is notably limited. Objectives To compare total claims paid and patient outcomes for patients with neck and back pain who received physical therapy intervention via direct access versus medical referral. Methods Data were accessed for patients seeking care for neck or back pain (n = 603) between 2012 and 2014, who chose to begin care either through traditional medical referral or direct access to a physical therapy- led spine management program. All patients received a standardized, pragmatic physical therapy approach, with patient-reported measures of pain and disability assessed before and after treatment. Patient demographics and outcomes data were obtained from the medical center patient registry and combined with total claims paid calculated for the year after the index claim. Linear mixed-effects modeling was used to analyze group differences in pain and disability, visits/time, and annualized costs. Results Patients who chose to enter care via the direct-access physical therapy-led spine management program displayed significantly lower total costs (mean difference, $1543; 95% confidence interval: $51, $3028; P = .04) than those who chose traditional medical referral. Patients in both groups showed clinically important improvements in pain and disability, which were similar between groups (P>.05). Conclusion The initial patient choice to begin care with a physical therapist for back or neck pain resulted in lower cost of care over the next year, while resulting in similar improvements in patient outcomes at discharge from physical therapy. These findings add to the emerging literature suggesting that patients' choice to access physical therapy through direct access may be associated with lower health care expenditures for patients with neck and back pain. Level of Evidence Economic and decision analyses, level 4. J Orthop Sports Phys Ther 2018;48(2):63-71. Epub 26 Oct 2017. doi:10.2519/jospt.2018.7423.
BackgroundUsing a larger, more comprehensive sample, and inclusion of the reverse shoulder arthroplasty as a primary surgical approach for proximal humerus fracture, we report on geographic variation in the treatment of proximal humerus fracture in 2011 and comment on whether treatment consensus is being reached.MethodsThis was a retrospective cohort study of Medicare patients with an x-ray-confirmed diagnosis of proximal humerus fracture in 2011. Patients receiving reverse shoulder arthroplasty, hemiarthroplasty, or open reduction internal fixation within 60 days of their diagnosis were classified as surgical management patients. Unadjusted observed surgery rates and area treatment ratios adjusted for patient demographic and clinical characteristics were calculated at the hospital referral region level.ResultsAmong patients with proximal humerus fracture (N = 77,053), 15.4% received surgery and 84.6% received conservative management. Unadjusted surgery rates varied from 1.7 to 33.3% across hospital referral regions. Among patients receiving surgery, 22.3% received hemiarthroplasty, 65.8% received open reduction internal fixation, and 11.8% received reverse shoulder arthroplasty. Patients that were female, were younger, had fewer medical comorbidities, had a lower frailty index, were white, or were not dual-eligible for Medicaid during the month of their index fracture were more likely to receive surgery (p < .0001). Geographic variation in the treatment of proximal humerus fracture persisted after adjustment for patient demographic and clinical differences across local areas. Average surgery rates ranged from 9.9 to 21.2% across area treatment ratio quintiles.ConclusionsPersistent geographic variation in surgery rates for proximal humerus fracture across the USA suggests no treatment consensus has been reached.
Background and Objective Pancreatic resection is the standard therapy for patients with stage I/II pancreatic ductal adenocarcinoma (PDA), yet many studies demonstrate low rates of resection. The objective of this study is to evaluate whether increasing resection rates would result in an increase in average survival in patients with stage I/II PDA. Methods SEER data were analyzed for patients with stage I/II pancreatic head cancers treated from 2004–2009. Pancreatectomy rates were examined within Health Service Areas (HSA) across 18 SEER regions. An instrumental variables (IV) analysis was performed, using HSA rates as an instrument, to determine the impact of increasing resection rates on survival. Results Pancreatectomy was performed in 4,322 of the 8,323 patients evaluated with stage I/II PDA (overall resection rate=51.9%). The resection rate across HSAs ranged from an average of 38.6% in the lowest quintile to 67.3% in the highest quintile. Median survival was improved in HSAs with higher resection rates. IV analysis revealed that, for patients whose treatment choices were influenced by the rates of resection in their geographic region, pancreatectomy was associated with a statistically significant increase in overall survival. Conclusions When controlling for confounders using IV analysis, pancreatectomy is associated with a statistically significant increase in survival for patients with resectable PDA. Based on these results, if resection rates were to increase in select patients, then average survival would also be expected to increase. It is important that this information be provided to physicians and patients so they can properly weigh the risks and advantages of pancreatectomy as treatment for PDA.
Despite guidelines promoting the use of statins for secondary prevention for CVD patients, substantial local area variation suggests that patient and provider beliefs and preferences weigh heavily in statin prescribing decisions.
Background:Atraumatic rotator cuff tear is a common orthopaedic complaint for people >60 years of age. Lack of evidence or consensus on appropriate treatment for this type of injury creates the potential for substantial discretion in treatment decisions. To our knowledge, no study has assessed the implications of this discretion on treatment patterns across the United States.Methods:All Medicare beneficiaries in the United States with a new magnetic resonance imaging (MRI)-confirmed atraumatic rotator cuff tear were identified with use of 2010 to 2012 Medicare administrative data and were categorized according to initial treatment (surgery, physical therapy, or watchful waiting). Treatment was modeled as a function of the clinical and demographic characteristics of each patient. Variation in treatment rates across hospital referral regions and the presence of area treatment signatures, representing the extent that treatment rates varied across hospital referral regions after controlling for patient characteristics, were assessed. Correlations between measures of area treatment signatures and measures of physician access in hospital referral regions were examined.Results:Among patients who were identified as having a new, symptomatic, MRI-confirmed atraumatic rotator cuff tear (n = 32,203), 19.8% were managed with initial surgery; 41.3%, with initial physical therapy; and 38.8%, with watchful waiting. Patients who were older, had more comorbidity, or were female, of non-white race, or dual-eligible for Medicaid were less likely to receive surgery (p < 0.0001). Black, dual-eligible females had 0.42-times (95% confidence interval [CI], 0.34 to 0.50) lower odds of surgery and 2.36-times (95% CI, 2.02 to 2.70) greater odds of watchful waiting. Covariate-adjusted odds of surgery varied dramatically across hospital referral regions; unadjusted surgery and physical therapy rates varied from 0% to 73% and from 6% to 74%, respectively. On average, patients in high-surgery areas were 62% more likely to receive surgery than the average patient with identical measured characteristics, and patients in low-surgery areas were half as likely to receive surgery than the average comparable patient. The supply of orthopaedic surgeons and the supply of physical therapists were associated with greater use of initial surgery and physical therapy, respectively.Conclusions:Patient characteristics had a significant influence on treatment for atraumatic rotator cuff tear but did not explain the wide-ranging variation in treatment rates across areas. Local-area physician supply and specialty mix were correlated with treatment, independent of the patient’s measured characteristics.
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