Clinical and Economic Implications of Interventions in Pancreatic Fluid Collections: An Assessment From a National Claims Database.

OBJECTIVE: To compare clinical and economic implications of percutaneous and endoscopic treatment approaches in patients with pancreatic fluid collections (PFCs). MATERIALS AND METHODS: This is a retrospective claims analysis of Medicare beneficiaries who underwent inpatient endoscopic or percutaneous PFC drainage procedures (2016-2020). We performed longitudinal analysis of claims for all-cause mortality and rehospitalization during 180-day follow-up. Main outcome was mortality. Other outcomes were rehospitalization and direct costs. RESULTS: A total of 1311 patients underwent endoscopic (n = 727) or percutaneous (n = 584) drainage. Percutaneous as compared with endoscopic approach was associated with higher mortality (23.08% vs 16.7%, P = 0.004), rehospitalization (58.9% vs 53.3%, P = 0.04), and mean direct hospital costs ($37,107 [SD = $67,833] vs $27,800 [SD = $43,854], P = 0.004). On multivariable analysis, percutaneous drainage (adjusted hazard ratio [HR], 1.38; 95% confidence interval [CI], 1.02-1.86; P = 0.039), older age (hazard ratio [HR], 1.04; 95% CI, 1.01-1.04; P < 0.001), intensive care unit stay (HR, 1.02; 95% CI, 1.01-1.03; P < 0.001), and multiple comorbidities (HR, 1.07; 95% CI, 1.05-1.09; P < 0.001) were significantly associated with mortality. Percutaneous drainage (adjusted odds ratio [OR], 1.30; 95% CI, 1.04-1.63; P = 0.027) and older age (OR, 0.98; 95% CI, 0.97-0.99; P < 0.001) were significantly associated with rehospitalizations. CONCLUSIONS: As percutaneous drainage may be associated with higher mortality, rehospitalization, and costs, when requisite expertise is available, endoscopy should be preferred for treatment of PFC amenable to such an approach. Randomized trials are required to validate these findings.

Sampling of Thoracic Lymph Nodes and Lung Lesions: Trends in Procedural Utilization.

BACKGROUND: Advances in bronchoscopy have impacted the practice patterns in the sampling of thoracic lymph nodes and lung lesions. OBJECTIVES: The aim of the study was to study the trends in utilization of mediastinoscopy, transthoracic needle aspiration (TTNA), and bronchoscopic transbronchial sampling. METHODS: We conducted an analysis of patient claims for sampling of thoracic lymph nodes and lung lesions in the Medicare population and a sample of the commercial population between 2016 and 2020. We used Current Procedural Terminology codes to identify mediastinoscopy, TTNA, and bronchoscopic transbronchial sampling. Post-procedural pneumothorax rates were assessed by procedure type including subset analyses for patients with chronic obstructive pulmonary disease (COPD). RESULTS: Between 2016 and 2020, utilization of mediastinoscopy has decreased in both the Medicare and commercial populations (-47.3% and -65.4%, respectively), while linear endobronchial ultrasound (EBUS)-guided transbronchial needle aspiration (TBNA) has increased only in the Medicare population (+28.2%). Percutaneous lung biopsy claims dropped by -17.0% in the Medicare and -41.22% in the commercial population. The use of bronchoscopic TBNA and forceps biopsy declined in both populations, but the reliance on a combination of guided technology (radial EBUS-guided and navigation) grew in the Medicare and commercial populations (+76.3% and +25%). Rates of post-procedural pneumothorax were significantly higher following percutaneous biopsy compared to bronchoscopic transbronchial biopsy. CONCLUSIONS: Linear EBUS-guided sampling has surpassed mediastinoscopy as the technique for sampling thoracic lymph nodes. Transbronchial lung sampling is increasingly being performed with guidance technology. This trend is aligned with favorable rates of post-procedure pneumothorax for transbronchial biopsy.

Evolving Use of Health Technology Assessment in Medical Device Procurement-Global Systematic Review: An ISPOR Special Interest Group Report.

OBJECTIVES: To review the current academic evidence describing how data from health technology assessments (HTAs) informs procurement decisions for medical devices. METHODS: A systematic literature review was performed to identify relevant studies and criteria used in medical device purchasing or procurement decisions. Included articles were screened for relevancy and risk of bias. The included studies were summarized qualitatively. RESULTS: A total of 292 studies were screened, of which 11 matched the inclusion criteria. Included studies' geographies and HTA maturity varied. Some studies described hospital-level HTA processes, whereas others focused on national-level recommendations. Criteria for procurement decisions included standard HTA factors, such as efficacy, cost, cost-effectiveness, and budget impact; broader issues were also noted, including impact on the organization, ethical aspects, staff workload, and volume. There was little consideration of device-specific characteristics, such as life cycle, learning curve, or incremental technical innovation. Few decisions referred to HTA reports as part of the procurement decision; similarly, few HTA reports included a procurement perspective to help guide the procurement bodies. CONCLUSIONS: There is minimal evidence that notes HTA influencing medical device procurement. Procurement bodies and hospitals may not be incentivized to publish their work and transparency could be improved; further research would better describe the link between HTA and procurement. Such research would enable the HTA agencies to meaningfully assess devices to target procurement bodies and allow device sponsors to prioritize evidence. This could limit redundancy, improve evidence, and ultimately promote savings to healthcare systems and expand access.

Indirect comparison of bronchial thermoplasty versus omalizumab for uncontrolled severe asthma.

OBJECTIVE: Bronchial thermoplasty (BT) as an add-on therapy for uncontrolled severe asthma is an alternative to biologic therapies like omalizumab (OM). We conducted an indirect treatment comparison (ITC) to appraise comparative effectiveness of BT and OM. METHODS: A systematic literature review identified relevant randomized controlled trials. The ITC followed accepted methodology. RESULTS: The ITC comprised a sham-controlled trial of BT (AIR2) and two placebo-controlled trials of OM (INNOVATE; EXTRA). Comparing the BT post-treatment period to ongoing treatment with OM, showed no significant differences in the rate ratios (RRs) for severe exacerbations (RR of BT versus OM = 0.91 [95% CI: 0.64, 1.30]; p = 0.62) or hospitalizations (RR = 0.57 [95% CI: 0.17, 1.86]; p = 0.53); emergency department visits were significantly reduced by 75% with BT (RR = 0.25 [95% CI: 0.07, 0.91]; p = 0.04); the proportions of patients with clinically meaningful response on the asthma quality-of-life questionnaire were comparable (RR = 1.06 [95% CI: 0.86, 1.34]; p = 0.59). The RR for exacerbations statistically favours OM over the total study period in AIR2 (RR = 1.50 [95% CI: 1.11, 2.02]; p = 0.009) likely reflecting a transient increase in events during the BT peri-treatment period. CONCLUSIONS: The ITC should be interpreted cautiously considering the differences between patient populations in the included trials. However, based on the analysis, BT compares well with a potentially more costly pharmacotherapy for asthma. Clinicians evaluating the relative merits of using these treatments should consider the totality of evidence and patient preferences to make an informed decision.

Investing in New Technology in Pulmonary Medicine: Navigating the Tortuous Path to Success.

The introduction of new technologies offers the promise to advance medicine. This occurs alongside improved efforts to control costs of health care by hospital administrators, the Centers for Medicare & Medicaid Services' (CMS) pivot to value programs, and commercial payers' efforts to reduce reimbursement. These trends present a challenge for the pulmonologist, among others, who must navigate increasingly complex and highly scrutinized evaluation processes used to secure new technology (NT). Health-care providers are turning toward value assessments while simultaneously tasked with the mission of offering state of the art technologies and services. Pulmonologists desiring NT are thus faced with increased scrutiny in their evaluation of costs and clinical data to support investments. Consideration of this scrutiny and further evidence to temper the evaluation will improve the likelihood of adoption and patient access to clinically impactful technology. The identification of this evidence may provide a comprehensive view of the clinical and economic benefits of such technologies to both administrators and pulmonary clinicians. It is imperative that all parties involved in the decision process work collaboratively to deploy value added and clinically impactful technologies. Although a physician group might invest in such NT, the capital required often leads such decisions to a larger organization such as a hospital, health-care system, or privately owned entity. This article aims to provide a framework for pulmonary clinicians to better understand the processes that purchasers use to evaluate NT, the pressures that influence their consideration, and what resources may be leveraged toward success.

Clinical Prediction Models for Cardiovascular Disease: Tufts Predictive Analytics and Comparative Effectiveness Clinical Prediction Model Database.

BACKGROUND: Clinical prediction models (CPMs) estimate the probability of clinical outcomes and hold the potential to improve decision making and individualize care. For patients with cardiovascular disease, there are numerous CPMs available although the extent of this literature is not well described. METHODS AND RESULTS: We conducted a systematic review for articles containing CPMs for cardiovascular disease published between January 1990 and May 2012. Cardiovascular disease includes coronary heart disease, heart failure, arrhythmias, stroke, venous thromboembolism, and peripheral vascular disease. We created a novel database and characterized CPMs based on the stage of development, population under study, performance, covariates, and predicted outcomes. There are 796 models included in this database. The number of CPMs published each year is increasing steadily over time. Seven hundred seventeen (90%) are de novo CPMs, 21 (3%) are CPM recalibrations, and 58 (7%) are CPM adaptations. This database contains CPMs for 31 index conditions, including 215 CPMs for patients with coronary artery disease, 168 CPMs for population samples, and 79 models for patients with heart failure. There are 77 distinct index/outcome pairings. Of the de novo models in this database, 450 (63%) report a c-statistic and 259 (36%) report some information on calibration. CONCLUSIONS: There is an abundance of CPMs available for a wide assortment of cardiovascular disease conditions, with substantial redundancy in the literature. The comparative performance of these models, the consistency of effects and risk estimates across models and the actual and potential clinical impact of this body of literature is poorly understood.

Medicare is scrutinizing evidence more tightly for national coverage determinations.

We examined Medicare national coverage determinations for medical interventions to determine whether or not they have become more restrictive over time. National coverage determinations address whether particular big-ticket medical items, services, treatment procedures, and technologies can be paid for under Medicare. We found that after we adjusted for the strength of evidence and other factors known to influence the determinations of the Centers for Medicare and Medicaid Services (CMS), the evidentiary bar for coverage has risen. More recent coverage determinations (from mid-March 2008 through August 2012) were twenty times less likely to be positive than earlier coverage determinations (from February 1999 through January 2002). Furthermore, coverage during the study period was increasingly and positively associated both with the degree of consistency of favorable findings in the CMS reviewed clinical evidence and with recommendations made in clinical guidelines. Coverage policy is an important payer tool for promoting the appropriate use of medical interventions, but CMS's rising evidence standards also raise questions about patients' access to new technologies and about hurdles for the pharmaceutical and device industries as they attempt to bring innovations to the market.

Cost-effectiveness of bronchial thermoplasty in commercially-insured patients with poorly controlled, severe, persistent asthma.

OBJECTIVES: We examined the cost-effectiveness of treating poorly controlled, severe, persistent asthma patients with bronchial thermoplasty (BT), a novel technology that uses thermal energy to reduce airway smooth muscle mass, with 5-year outcome data demonstrating a durable reduction in asthma exacerbations. STUDY DESIGN: We conducted a model-based cost-effectiveness analysis assessing 5-year healthcare utilization, patient quality of life and adverse events. METHODS: We utilized Markov modeling to estimate the costs and quality-of-life impact of BT compared with high-dose combination therapy among poorly controlled, severe, persistent asthma patients: those requiring high-dose combination therapy and having experienced an asthma exacerbation-related ER visit in the past year. RESULTS: The cost-effectiveness of BT was US$5495 per quality-adjusted life year; and approximately 22% of sensitivity analysis iterations estimated BT to reduce costs and increase quality of life. CONCLUSIONS: BT is a cost-effective treatment option for patients with poorly controlled, severe, persistent asthma.

CHANGING FACE OF MEDICARE'S NATIONAL COVERAGE DETERMINATIONS FOR TECHNOLOGY.

OBJECTIVES: The Centers for Medicare and Medicaid Services (CMS) issues National Coverage Determinations (NCDs) for medical interventions expected to have a significant impact on Medicare, the health insurance program for US citizens aged 65 years and older and certain people with disabilities under the age of 65 years. The objective of this study was to evaluate NCDs issued from 1999 to 2013 to identify key trends, and to discuss implications for future CMS policy. METHODS: We used the Tufts Medical Center Medicare National Coverage Determination Database to examine characteristics of NCDs from 1999 through 2013. We examined various characteristics of NCDs, including: whether the intervention under review is used for prevention or treatment of disease, the type of intervention considered, evidence limitations cited by CMS, and coverage determination outcome. We evaluated longitudinal trends in categorical and continuous variables in the database, using Cochran-Armitage trend tests and linear regression, respectively. RESULTS: We found that NCDs increasingly focus on preventive care (p = 0.072), pertain to diagnostic imaging (p = 0.033), and evaluate health education/behavioral therapy interventions (p = 0.051). CMS increasingly cites the lack of relevant outcomes (p = 0.019) and the lack of applicability of study results to the Medicare population (p < 0.001) as evidence limitations. CMS less often restricts coverage to certain population subgroups in NCDs (p < 0.001), but increasingly applies coverage with evidence development policies (p < 0.001). CONCLUSIONS: Identified trends reflect broader changes in Medicare as CMS shifts its focus from treatment to prevention of disease, addresses potentially overutilized technologies, and attempts to issue flexible coverage policies.

Medicare's use of cost-effectiveness analysis for prevention (but not for treatment).

CONTEXT: Medicare currently pays for 23 preventive services in its benefits package, the majority of which were added since 2005. In the past decade, the program has transformed from one essentially administering treatment claims, to one increasingly focused on health promotion and maintenance. What is largely unappreciated is the role cost-effectiveness analysis has played in the coverage of preventive services. METHODS: We review the role of cost-effectiveness analysis in Medicare coverage of preventive services and contrast it to the lack of such consideration in the coverage of treatments. FINDINGS: While not considered for coverage of treatment, cost-effectiveness analysis played a role in the coverage of nine preventive services, and was evaluated in a number of instances when the service was not added. Pneumococcal vaccine, the first preventive service added to the benefit (1981), followed a Congressionally requested cost-effectiveness analysis, which showed it to be cost-saving. More recently, the Centers for Medicare and Medicaid Services (CMS) reviewed cost-effectiveness evidence when covering preventive services such as HIV screening (2010) and screening and behavioral counseling for alcohol misuse (2011) (studies reported cost-effectiveness ratios of $55,440 per QALY, and $1755 per QALY, respectively). CONCLUSIONS: Cost-effectiveness analysis has played a longstanding role in informing the addition of preventive services to Medicare. It offers Medicare officials information they can use to help ensure health gains are achieved at reasonable cost. However, limiting cost-effectiveness evidence to prevention and not treatment is inconsistent and potentially inefficient.

Potential role of network meta-analysis in value-based insurance design.

OBJECTIVES: Value-based insurance design (V-BID) has emerged as an approach to improve health outcomes and contain healthcare costs by encouraging use of high-value care. We estimated the impact of a V-BID for osteoporosis treatments using comparative effectiveness evidence and real-world data from a California health insurance plan to estimate the benefits of the design's implementation. METHODS: This study consisted of 4 steps. First, we reviewed randomized clinical trials including osteoporosis treatments-alendronate, ibandronate, risedronate, raloxifene, and teriparatide-reported in a recent Agency for Health Research Quality systematic review. Second, we performed a network meta-analysis to synthesize data from the clinical trials and estimate the comparative effectiveness of included treatments. Third, we implemented a V-BID by removing co-payments for the most effective treatments. Fourth, using a Monte Carlo simulation, we estimated the impact of the V-BID in terms of fracture reduction and cost-savings. RESULTS: Thirty-two randomized controlled trials were included in the network meta-analysis. We estimated that alendronate, risedronate, and teriparatide have the highest probability of being most effective across each fracture type-vertebral, hip, and nonvertebral/ nonhip. After eliminating co-payments, (ie, reducing them to zero), for these treatments, we estimated the health plan would experience a 7% (n = 287) decrease in fractures and an 8% ($6.8 million) decrease in costs. CONCLUSIONS: Our study illustrates the benefits of comparative effectiveness evidence in V-BID development. We show that where clinical trials are lacking, network meta-analysis can provide valuable insights into the potential clinical and economic benefits of V-BID.

Evaluation of the economic burden of diseases associated with poor nutrition status.

BACKGROUND: Geriatric acutely ill patients may frequently have insufficient nutrition intake, leading to malnutrition and increased susceptibility to additional morbidity. This compromised health status can increase costs by extending length of stay in the hospital, promoting the development of comorbidities, and requiring more intensive care. Understanding the value of measures improving the health of these patients depends in part on estimating the costs of prevented adverse outcomes. Because clinical trials often do not record these costs, it is difficult to assess the value of nutrition interventions. MATERIALS AND METHODS: This article comprehensively reviews 15 years of clinical trials of nutrition interventions to identify salient diseases and complications measured by previous clinical trials and then estimates costs corresponding with these conditions associated with poor nutrition status. RESULTS: The most costly complication associated with poor nutrition status is acute respiratory infections ($13,350-$19,530 per hospitalization), while institutional long-term care is the greatest chronic cost contributor across many diseases ($77,000 per year of care). CONCLUSION: This review can facilitate imputation of nutrition intervention benefits for acutely ill patients by characterizing the costs of clinical outcomes often reported in trials.

Willingness to pay for diagnostic technologies: a review of the contingent valuation literature.

OBJECTIVES: To understand how people value information from diagnostic technologies, we reviewed and analyzed published willingness-to-pay (WTP) studies on the topic. METHODS: We searched PubMed for English-language articles related to WTP for diagnostic laboratory tests published from 1985 through 2011. We characterized methodological differences across studies, examined individual- and technology-level factors associated with WTP, and summarized median WTP values across different diagnostic tests. RESULTS: We identified 66 relevant WTP studies. Half focused on oncology, while others analyzed infectious diseases (n = 11, 16.1%) and obstetric or gynecological conditions (n = 8, 11.7%), among others. Most laboratory tests included in studies were biological samples/genetic testing (n = 44, 61.1%) or imaging tests (n = 23, 31.9%). Approximately one third of the analyses (n = 20, 30.3%) used discrete-choice questions to elicit WTP values. Higher income, education, disease severity, perceived disease risk, family history, and more accurate tests were in general associated with higher WTP values for diagnostic information. Of the 44 studies with median WTP values available, most reported a median WTP value below $100. The median WTP value for colon or colorectal cancer screening ranged from below $100 to over $1000. CONCLUSIONS: The contingent valuation literature in diagnostics has grown rapidly, and suggests that many respondents place considerable value on diagnostic information. There exists, however, great variation in studies with respect to the type of technologies and diseases assessed, respondent characteristics, and study methodology. The perceived value of diagnostic technologies is also influenced by the study design and elicitation methods.

Economic impact of childhood and adult attention-deficit/hyperactivity disorder in the United States.

OBJECTIVE: Attention-deficit/hyperactivity disorder (ADHD) is one of the most prevalent mental disorders in children in the United States and often persists into adulthood with associated symptomatology and impairments. This article comprehensively reviews studies reporting ADHD-related incremental (excess) costs for children/adolescents and adults and presents estimates of annual national incremental costs of ADHD. METHOD: A systematic search for primary United States-based studies published from January 1, 1990 through June 30, 2011 on costs of children/adolescents and adults with ADHD and their family members was conducted. Only studies in which mean annual incremental costs per individual with ADHD above non-ADHD controls were reported or could be derived were included. Per-person incremental costs were adjusted to 2010 U.S. dollars and converted to annual national incremental costs of ADHD based on 2010 U.S. Census population estimates, ADHD prevalence rates, number of household members, and employment rates by age group. RESULTS: Nineteen studies met the inclusion criteria. Overall national annual incremental costs of ADHD ranged from $143 to $266 billion (B). Most of these costs were incurred by adults ($105 B-$194 B) compared with children/adolescents ($38 B-$72 B). For adults, the largest cost category was productivity and income losses ($87 B-$138 B). For children, the largest cost categories were health care ($21 B-$44 B) and education ($15 B-$25 B). Spillover costs borne by the family members of individuals with ADHD were also substantial ($33 B-$43 B). CONCLUSION: Despite a wide range in the magnitude of the cost estimates, this study indicates that ADHD has a substantial economic impact in the United States. Implications of these findings and future directions for research are discussed.

Imputing productivity gains from clinical trials.

OBJECTIVE: To respond to employer and payer interest in the extent to which productivity gains offset therapy costs by identifying clinical trials that did not include such measures and using their clinical data to impute productivity impact. METHODS: A PubMed search identified the sample of 25 clinical trials of musculoskeletal pain medications and antidepressants. Next, we applied regression coefficients, quantifying the empirical relationship between clinical measures to each trial's clinical outcomes data. This validated methodology provides estimates of Work Limitations Questionnaire Productivity Loss scores. RESULTS: Based on imputation, musculoskeletal medications and antidepressants achieved median productivity gains of approximately 0.5% and 1.0%, respectively. CONCLUSION: Accounting for productivity gains based on the Work Limitations Questionnaire could substantially influence cost-effectiveness results reported in the health economics literature.

A clinical and economic evaluation of enteral nutrition.

MOTIVATION: The American Society of Parenteral and Enteral Nutrition (ASPEN) guidelines advise use of enteral nutrition (EN) for critically ill hospital patients requiring nutritional support, but no studies have comprehensively estimated economic benefits from adherence to this recommendation. METHODS: We systematically reviewed studies comparing EN to alternative nutritional support therapies among adult, critically ill patients. We reviewed 1200 abstracts, selected 243 for further review, and included 48 studies in our analysis. Most retained studies compared EN and parenteral nutrition (PN). Using meta-analysis, we estimated the absolute impact of EN on adverse event risk and its impact on treatment duration and length of stay. These estimates were converted to population economic impacts by assuming 10% of PN patients are suitable candidates for EN. RESULTS: Compared to PN, EN reduces the risk of major, potentially life-threatening infections (RR = 0.58, 95% confidence interval [CI] 0.44 to 0.77), the risk of major, potentially life-threatening non-infection events (RR = 0.73, CI 0.59 to 0.91), and suggests a reduction in mortality, although this result did not achieve statistical significance (RR = 0.70, CI 0.45 to 1.09). EN also reduces inpatient length of stay, time in the ICU, and length of nutritional treatment. Compared to PN, EN savings from reduced adverse event risks average nearly $1500 per patient; savings from reduced hospital length of stay amount to nearly $2500 per patient. Shifting 10% of parenterally treated adult patients in the U.S. to EN would save $35 million annually due to reduced adverse events and another $57 million due to shorter hospital stays. CONCLUSION: The evidence of both clinical and economic gains from EN is consistent with ASPEN guidelines recommending use of EN in critically ill hospital patients when possible.