Assessing the properties of patient-specific treatment effect estimates from causal forest algorithms under essential heterogeneity.

BACKGROUND: Treatment variation from observational data has been used to estimate patient-specific treatment effects. Causal Forest Algorithms (CFAs) developed for this task have unknown properties when treatment effect heterogeneity from unmeasured patient factors influences treatment choice - essential heterogeneity. METHODS: We simulated eleven populations with identical treatment effect distributions based on patient factors. The populations varied in the extent that treatment effect heterogeneity influenced treatment choice. We used the generalized random forest application (CFA-GRF) to estimate patient-specific treatment effects for each population. Average differences between true and estimated effects for patient subsets were evaluated. RESULTS: CFA-GRF performed well across the population when treatment effect heterogeneity did not influence treatment choice. Under essential heterogeneity, however, CFA-GRF yielded treatment effect estimates that reflected true treatment effects only for treated patients and were on average greater than true treatment effects for untreated patients. CONCLUSIONS: Patient-specific estimates produced by CFAs are sensitive to why patients in real-world practice make different treatment choices. Researchers using CFAs should develop conceptual frameworks of treatment choice prior to estimation to guide estimate interpretation ex post.

Association between household opioid prescriptions and risk for overdose among family members not prescribed opioids.

BACKGROUND: Prescription opioids have contributed to the rise in opioid-related overdoses and deaths. The presence of opioids within households may increase the risk of overdose among family members who were not prescribed an opioid themselves. Larger quantities of opioids may further increase risk. OBJECTIVES: To determine the risk of opioid overdose among individuals who were not prescribed an opioid but were exposed to opioids prescribed to other family members in the household, and evaluate the risk in relation to the total morphine milligram equivalents (MMEs) present in the household. METHODS: We conducted a cohort study using a large database of commercial insurance claims from 2001 to 2021. For inclusion in the cohort, we identified individuals not prescribed an opioid in the prior 90 days from households with two or more family members, and determined the total MMEs prescribed to other family members. Individuals were stratified into monthly enrollment strata defined by household opioid exposure and other confounders. A generalized linear model was used to estimate incidence rate ratios (IRRs) for overdose. RESULTS: Overall, the incidence of overdose among enrollees in households where a family member was prescribed an opioid was 1.73 (95% confidence interval [CI]: 1.67-1.78) times greater than households without opioid prescriptions. The risk of overdose increased continuously with the level of potential MMEs in the household from an IRR of 1.23 (95% CI: 1.16-1.32) for 1-100 MMEs to 4.67 (95% CI: 4.18-5.22) for >12,000 MMEs. The risk of overdose associated with household opioid exposure was greatest for ages 1-2 years (IRR: 3.46 [95% CI: 2.98-4.01]) and 3-5 years (IRR: 3.31 [95% CI: 2.75-3.99]). CONCLUSIONS: The presence of opioids in a household significantly increases the risk of overdose among other family members who were not prescribed an opioid. Higher levels of MMEs, either in terms of opioid strength or quantity, were associated with increased levels of risk. Risk estimates may reflect accidental poisonings among younger family members.

The Iowa Health Data Resource (IHDR): an innovative framework for transforming the clinical health data ecosystem.

IMPORTANCE: This manuscript will be of interest to most Clinical and Translational Science Awards (CTSA) as they retool for the increasing emphasis on translational science from translational research. This effort is an extension of the EDW4R work that most CTSAs have done to deploy infrastructure and tools for researchers to access clinical data. OBJECTIVES: The Iowa Health Data Resource (IHDR) is a strategic investment made by the University of Iowa to improve access to real-world health data. The goals of IHDR are to improve the speed of translational health research, to boost interdisciplinary collaboration, and to improve literacy about health data. The first objective toward this larger goal was to address gaps in data access, data literacy, lack of computational environments for processing Personal Health Information (PHI) and the lack of processes and expertise for creating transformative datasets. METHODS: A three-pronged approach was taken to address the objective. The approach involves integration of an intercollegiate team of non-informatics faculty and staff, a data enclave for secure patient data analyses, and novel comprehensive datasets. RESULTS: To date, all five of the health science colleges (dentistry, medicine, nursing, pharmacy, and public health) have had at least one staff and one faculty member complete the two-month experiential learning curriculum. Over the first two years of this project, nine cohorts totaling 36 data liaisons have been trained, including 18 faculty and 18 staff. IHDR data enclave eliminated the need to duplicate computational infrastructure inside the hospital firewall which reduced infrastructure, hardware and human resource costs while leveraging the existing expertise embedded in the university research computing team. The creation of a process to develop and implement transformative datasets has resulted in the creation of seven domain specific datasets to date. CONCLUSION: The combination of people, process, and technology facilitates collaboration and interdisciplinary research in a secure environment using curated data sets. While other organizations have implemented individual components to address EDW4R operational demands, the IHDR combines multiple resources into a novel, comprehensive ecosystem IHDR enables scientists to use analysis tools with electronic patient data to accelerate time to science.

Patient preferences and willingness-to-pay for community pharmacy-led Medicare Part D consultation services: A discrete choice experiment.

INTRODUCTION: Community pharmacies currently offer Medicare Part D consultation services, often at no-cost. Despite facilitating plan-switching behavior, identifying potential cost-savings, and increasing medication adherence, patient uptake of these services remains low. OBJECTIVES: To investigate patient preferences for specific service-offering attributes and marginal willingness-to-pay (mWTP) for an enhanced community pharmacy Medicare Part D consultation service. METHODS: A discrete choice experiment (DCE) guided by the SERVQUAL framework was developed and administered using a national online survey panel. Study participants were English-speaking adults (≥65 years) residing in the United States enrolled in a Medicare Part D or Medicare Advantage plan and had filled a prescription at a community pharmacy within the last 12 months. An orthogonal design resulted in 120 paired-choice tasks distributed equally across 10 survey blocks. Data were analyzed using mixed logit and latent class models. RESULTS: In total, 540 responses were collected, with the average age of respondents being 71 years. The majority of respondents were females (60%) and reported taking four or more prescription medication (51%). Service attribute levels with the highest utility were: 15-min intervention duration (0.392), discussion of services + a follow-up phone call (0.069), in-person at the pharmacy (0.328), provided by a pharmacist the patient knew (0.578), and no-cost (3.382). The attribute with the largest mWTP value was a service provided by a pharmacist the participant knew ($8.42). Latent class analysis revealed that patient preferences for service attributes significantly differed by gender and difficulty affording prescription medications. CONCLUSIONS: Quantifying patient preference using discrete choice methodology provides pharmacies with information needed to design service offerings that balance patient preference and sustainability. Pharmacies may consider providing interventions at no-cost to subsets of patients placing high importance on a service cost attribute. Further, patient preference for 15-min interventions may inform Medicare Part D service delivery and facilitate service sustainability.

Generating Practice-Based Evidence in the Use of Guideline-Recommended Combination Therapy for Secondary Prevention of Acute Myocardial Infarction.

Background: Clinical guidelines recommend beta-blockers, angiotensin-converting enzyme inhibitors/angiotensin-receptor blockers, and statins for the secondary prevention of acute myocardial infarction (AMI). It is not clear whether variation in real-world practice reflects poor quality-of-care or a balance of outcome tradeoffs across patients. Methods: The study cohort included Medicare fee-for-service beneficiaries hospitalized 2007-2008 for AMI. Treatment within 30-days post-discharge was grouped into one of eight possible combinations for the three drug classes. Outcomes included one-year overall survival, one-year cardiovascular-event-free survival, and 90-day adverse events. Treatment effects were estimated using an Instrumental Variables (IV) approach with instruments based on measures of local-area practice style. Pre-specified data elements were abstracted from hospital medical records for a stratified, random sample to create "unmeasured confounders" (per claims data) and assess model assumptions. Results: Each drug combination was observed in the final sample (N = 124,695), with 35.7% having all three, and 13.5% having none. Higher rates of guideline-recommended treatment were associated with both better survival and more adverse events. Unmeasured confounders were not associated with instrumental variable values. Conclusions: The results from this study suggest that providers consider both treatment benefits and harms in patients with AMIs. The investigation of estimator assumptions support the validity of the estimates.

Assessing the ability of an instrumental variable causal forest algorithm to personalize treatment evidence using observational data: the case of early surgery for shoulder fracture.

BACKGROUND: Comparative effectiveness research (CER) using observational databases has been suggested to obtain personalized evidence of treatment effectiveness. Inferential difficulties remain using traditional CER approaches especially related to designating patients to reference classes a priori. A novel Instrumental Variable Causal Forest Algorithm (IV-CFA) has the potential to provide personalized evidence using observational data without designating reference classes a priori, but the consistency of the evidence when varying key algorithm parameters remains unclear. We investigated the consistency of IV-CFA estimates through application to a database of Medicare beneficiaries with proximal humerus fractures (PHFs) that previously revealed heterogeneity in the effects of early surgery using instrumental variable estimators. METHODS: IV-CFA was used to estimate patient-specific early surgery effects on both beneficial and detrimental outcomes using different combinations of algorithm parameters and estimate variation was assessed for a population of 72,751 fee-for-service Medicare beneficiaries with PHFs in 2011. Classification and regression trees (CART) were applied to these estimates to create ex-post reference classes and the consistency of these classes were assessed. Two-stage least squares (2SLS) estimators were applied to representative ex-post reference classes to scrutinize the estimates relative to known 2SLS properties. RESULTS: IV-CFA uncovered substantial early surgery effect heterogeneity across PHF patients, but estimates for individual patients varied with algorithm parameters. CART applied to these estimates revealed ex-post reference classes consistent across algorithm parameters. 2SLS estimates showed that ex-post reference classes containing older, frailer patients with more comorbidities, and lower utilizers of healthcare were less likely to benefit and more likely to have detriments from higher rates of early surgery. CONCLUSIONS: IV-CFA provides an illuminating method to uncover ex-post reference classes of patients based on treatment effects using observational data with a strong instrumental variable. Interpretation of treatment effect estimates within each ex-post reference class using traditional CER methods remains conditional on the extent of measured information in the data.

Increased opioid use following rotator cuff repair associated with prior opioid use and surgeon prescription patterns.

BACKGROUND: Prescription opioids are standard of care for postoperative pain management after musculoskeletal surgery, but there is no guideline or consensus on best practices. Variability in the intensity of opioids prescribed for postoperative recovery has been documented, but it is unclear whether this variability is clinically motivated or associated with provider practice patterns, or how this variation is associated with patient outcomes. This study described variation in the intensity of opioids prescribed for patients undergoing rotator cuff repair (RCR) and examined associations with provider prescribing patterns and patients' long-term opioid use outcomes. METHODS: Medicare data from 2010 to 2012 were used to identify 16,043 RCRs for patients with new shoulder complaints in 2011. Two measures of perioperative opioid use were created: (1) any opioid fill occurring 3 days before to 7 days after RCR and (2) total morphine milligram equivalents (MMEs) of all opioid fills during that period. Patient outcomes for persistent opioid use after RCR included (1) any opioid fill from 90 to 180 days after RCR and (2) the lack of any 30-day gap in opioid availability during that period. Generalized linear regression models were used to estimate associations between provider characteristics and opioid use for RCR, and between opioid use and outcomes. All models adjusted for patient clinical and demographic characteristics. Separate analyses were done for patients with and without opioid use in the 180 days before RCR. RESULTS: In this sample, 54% of patients undergoing RCR were opioid naive at the time of RCR. Relative to prior users, a greater proportion of opioid naive users had any opioid fill (85.7% vs. 75.4%), but prior users received more MMEs than naive users (565 vs. 451 MMEs). Providers' opioid prescribing for other patients was associated with the intensity of perioperative opioids received for RCR. Total MMEs received for RCR were associated with higher odds of persistent opioid use 90-180 days after RCR. CONCLUSIONS: The intensity of opioids received by patients for postoperative pain appears to be partially determined by the prescribing habits of their providers. Greater intensity of opioids received is, in turn, associated with greater odds of patterns of chronic opioid use after surgery. More comprehensive, patient-centered guidance on opioid prescribing is needed to help surgeons provide optimal postoperative pain management plans, balancing needs for short-term symptom relief and risks for long-term outcomes.

Risk Adjustment of the Modified Low Back Pain Disability Questionnaire and Neck Disability Index to Benchmark Physical Therapist Performance: Analysis From an Outcomes Registry.

BACKGROUND: Patient-reported outcomes (PROs) have been touted as the ultimate assessment of quality medical care and have been proposed as performance measures after appropriate risk adjustment. Although spine conditions represent the most common orthopedic disorders, the most used PROs for disabilities related to the back and neck-the Modified Low Back Pain Disability Questionnaire (MDQ) and the Neck Disability Index (NDI)-have not been evaluated as performance measures. OBJECTIVE: The objective of this study was to benchmark physical therapists' performance in the management of spine conditions not involving surgery through the use of risk-adjusted MDQ and NDI outcomes. DESIGN: This was a retrospective observational study. METHODS: Data were accessed for patients seeking physical therapy with no history of related surgery for back or neck pain (315,274 treatment episodes) between January 2015 and June 2018. Patients with complete data, including initial and matched final MDQ or NDI, were considered for analysis (182,276 patients; 2799 physical therapists). Linear models controlling for baseline PRO and patient characteristics predicted PRO change for each patient. An aggregated performance ratio of actual PRO change to predicted PRO change was calculated for each physical therapist, and then empirical bootstrapping was used to develop the median performance ratio and its confidence intervals. Physical therapists who met a 40-patient threshold for either cohort (MDQ or NDI) were classified as "outperforming," "meeting expectations," or "underperforming" relative to predicted values using these 95% confidence intervals. RESULTS: Performance ratios indicated that 10% and 11% of physical therapists outperformed, 79% and 78% met expectations, and 11% and 11% underperformed relative to the risk-adjusted predicted change in the MDQ (1240 therapists; 97,908 patients) and NDI (461 therapists; 26,123 patients), respectively. To demonstrate the clinical importance of risk adjustment, clinical performance was evaluated in the seemingly homogeneous subset of 208 physical therapists within 0.5 SD of the median baseline MDQ and the median actual change in the MDQ. Following risk adjustment, 2 physical therapists were classified in each of the outperforming and underperforming cohorts. LIMITATIONS: The secondarily obtained observational data used were not collected for research purposes. Additionally, the analyses were limited by missing baseline information and follow-up PROs. CONCLUSIONS: The risk-adjusted performance ratios for the MDQ and NDI resulted in disparate conclusions regarding the quality of care compared with the raw, unadjusted change scores. According to the baseline and unadjusted change in the MDQ, even physical therapists in the most homogeneous sample were differentiated following appropriate risk adjustment. Clinically important improvements in actual PROs were observed in the outperforming but not in the underperforming physical therapists. Clinically meaningful differences in the performance ratio are unknown and are a limitation to clinical application and an opportunity for future research.

Geographic variation in the treatment of proximal humerus fracture: an update on surgery rates and treatment consensus.

BACKGROUND: Using 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. METHODS: This 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. RESULTS: Among 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. CONCLUSIONS: Persistent geographic variation in surgery rates for proximal humerus fracture across the USA suggests no treatment consensus has been reached.

Athletic Trainers' Effect on Population Health: Improving Access to and Quality of Care.

CONTEXT: The scope of athletic training practice combined with the magnitude of scholastic athletic injuries means that the scholastic athletic trainer (AT) is uniquely positioned to positively affect the overall health care of this population. The AT is equipped to serve in the prevention and primary management of injuries and return to activity of scholastic athletes. However, to optimize the musculoskeletal health of all athletes within a given setting, the gaps in clinical care must be continuously evaluated. Quality improvement (QI) approaches are often used to establish a framework for delivering care that promotes the best health status of the targeted population. OBJECTIVE: To describe the creation, implementation, and early results of a QI initiative aimed at advancing the health of the scholastic athletes served in the Greenville County, South Carolina, school district. DESIGN: Cohort study. PATIENTS OR OTHER PARTICIPANTS: A total of 49 793 athletes. MAIN OUTCOME MEASURE(S): The QI framework consisted of a process that documented the magnitude of athletic injuries, established risk factors for injury, defined intervention steps for at-risk athletes, and evaluated the QI process before and after implementation. The results were regularly reported to participating stakeholders, including ATs, athletic directors, coaches, parents, and athletes. RESULTS: After the QI process, injury rates decreased (absolute risk difference between the 2011-2012 and 2016-2017 academic years = 22%) and resources were more strategically allocated, which resulted in a decrease in health care costs of more than 50%. CONCLUSIONS: Collectively, the QI framework as described provides a systematic process for empowering the AT as the foundation of the scholastic sports medicine team.

Treatment for Rotator Cuff Tear Is Influenced by Demographics and Characteristics of the Area Where Patients Live.

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.

A comparison of one-year treatment utilization for shoulder osteoarthritis patients initiating care with non-orthopaedic physicians and orthopaedic specialists.

BACKGROUND: In this paper we investigate patients seeking care for a new diagnosis of shoulder osteoarthritis (OA) and the association between a patient's initial physician specialty choice and one-year surgical and conservative treatment utilization. METHODS: Using retrospective data from a single large regional healthcare system, we identified 572 individuals with a new diagnosis of shoulder OA and identified the specialty of the physician which was listed as the performing physician on the index shoulder visit. We assessed treatment utilization in the year following the index shoulder visit for patients initiating care with a non-orthopaedic physician (NOP) or an orthopaedic specialist (OS). Descriptive statistics were calculated for each group and subsequent one-year surgical and conservative treatment utilization was compared between groups. RESULTS: Of the 572 patients included in the study, 474 (83%) received care from an OS on the date of their index shoulder visit, while 98 (17%) received care from a NOP. There were no differences in baseline patient age, gender, BMI or pain scores between groups. OS patients reported longer symptom duration and a higher rate of comorbid shoulder diagnoses. Patients initiating care with an OS on average received their first treatment much faster than patients initiating care with NOP (16.3 days [95% CI, 12.8, 19.7] vs. 32.3 days [95% CI, 21.0, 43.6], Z = 4.9, p < 0.01). Additionally, patients initiating care with an OS had higher odds of receiving surgery (OR = 2.65, 95% CI: 1.42, 4.95) in the year following their index shoulder visit. CONCLUSIONS: Patients initiating care with an OS received treatment much faster and were treated with more invasive services over the year following their index shoulder visit. Future work should compare patient-reported outcomes across patient groups to assess whether more expensive and invasive treatments yield better outcomes for patients with shoulder OA.

Use of Angiotensin-Converting Enzyme Inhibitors and Angiotensin Receptor Blockers for Geriatric Ischemic Stroke Patients: Are the Rates Right?

BACKGROUND: Our objective is to estimate the effects associated with higher rates of renin-angiotensin system antagonists, angiotensin-converting enzyme inhibitors and angiotensin receptor blockers (ACEI/ARBs), in secondary prevention for geriatric (aged >65 years) patients with new ischemic strokes by chronic kidney disease (CKD) status. METHODS AND RESULTS: The effects of ACEI/ARBs on survival and renal risk were estimated by CKD status using an instrumental variable (IV) estimator. Instruments were based on local area variation in ACEI/ARB use. Data abstracted from charts were used to assess the assumptions underlying the instrumental estimator. ACEI/ARBs were used after stroke by 45.9% and 45.2% of CKD and non-CKD patients, respectively. ACEI/ARB rate differences across local areas grouped by practice styles were nearly identical for CKD and non-CKD patients. Higher ACEI/ARB use rates for non-CKD patients were associated with higher 2-year survival rates, whereas higher ACEI/ARB use rates for patients with CKD were associated with lower 2-year survival rates. While the negative survival estimates for patients with CKD were not statistically different from zero, they were statistically lower than the estimates for non-CKD patients. Confounders abstracted from charts were not associated with the instrumental variable used. CONCLUSIONS: Higher ACEI/ARB use rates had different survival implications for older ischemic stroke patients with and without CKD. ACEI/ARBs appear underused in ischemic stroke patients without CKD as higher use rates were associated with higher 2-year survival rates. This conclusion is not generalizable to the ischemic stroke patients with CKD, as higher ACEI/ARBS use rates were associated with lower 2-year survival rates that were statistically lower than the estimates for non-CKD patients.

2SLS versus 2SRI: Appropriate methods for rare outcomes and/or rare exposures.

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.

Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome.

BACKGROUND: Patient-centred care requires evidence of treatment effects across many outcomes. Outcomes can be beneficial (e.g. increased survival or cure rates) or detrimental (e.g. adverse events, pain associated with treatment, treatment costs, time required for treatment). Treatment effects may also be heterogeneous across outcomes and across patients. Randomized controlled trials are usually insufficient to supply evidence across outcomes. Observational data analysis is an alternative, with the caveat that the treatments observed are choices. Real-world treatment choice often involves complex assessment of expected effects across the array of outcomes. Failure to account for this complexity when interpreting treatment effect estimates could lead to clinical and policy mistakes. OBJECTIVE: Our objective was to assess the properties of treatment effect estimates based on choice when treatments have heterogeneous effects on both beneficial and detrimental outcomes across patients. METHODS: Simulation methods were used to highlight the sensitivity of treatment effect estimates to the distributions of treatment effects across patients across outcomes. Scenarios with alternative correlations between benefit and detriment treatment effects across patients were used. Regression and instrumental variable estimators were applied to the simulated data for both outcomes. RESULTS: True treatment effect parameters are sensitive to the relationships of treatment effectiveness across outcomes in each study population. In each simulation scenario, treatment effect estimate interpretations for each outcome are aligned with results shown previously in single outcome models, but these estimates vary across simulated populations with the correlations of treatment effects across patients across outcomes. CONCLUSIONS: 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.

Shoulder arthroplasty in the US Medicare population: a 1-year evaluation of surgical complications, hospital admissions, and revision surgery.

BACKGROUND: The objective of this study was to describe patients receiving each shoulder arthroplasty procedure and to assess surgical complications, hospital admissions for surgical complications, and surgical revisions among Medicare beneficiaries undergoing shoulder arthroplasty. METHODS: Medicare patients receiving shoulder arthroplasty in the United States in 2011 were identified from Medicare administrative data and classified by surgery type: shoulder hemiarthroplasty (HA), anatomic total shoulder arthroplasty (TSA), or reverse shoulder arthroplasty (RSA). Surgical complications, hospital admissions, and revisions were identified during the year after the index arthroplasty procedure. RESULTS: There were 24,441 patients who met all inclusion criteria, and of those, 20.0% received HA, 42.5% received TSA, and 37.4% received RSA. Compared with RSA and TSA recipients, HA recipients tended to be older and sicker and were more likely to be Medicaid eligible. The rate of new surgical complications and related hospital admissions was greatest during the first 50 days after surgery but remained significant and stable throughout the remainder of the year. Rates of complications and related hospital admissions were greatest for HA recipients (17.4% and 6.6%, respectively), followed by RSA (14.2% and 5.1%) and TSA (9.4% and 4.0%). CONCLUSIONS: The rate of adverse surgical outcomes after shoulder arthroplasty differed across populations that received HA, TSA, and RSA and across patients within each group by comorbidity burden. The finding that the rate of surgical complications and related hospital admissions remained meaningful during the entire year after surgery suggests that a postoperative follow-up period longer than the traditional 90 days may be warranted.

The Influence of Patient Choice of First Provider on Costs and Outcomes: Analysis From a Physical Therapy Patient Registry.

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.