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BACKGROUND: Evidence that long-term hormone therapy (HT) may increase the risk of serious adverse events led to a sharp reduction in all HT use, including short-term use for vasomotor symptom relief. We estimated women's willingness to accept adverse event risks in exchange for vasomotor symptom relief when risks are communicated in absolute vs. relative terms. METHODS: We developed a conjoint survey to elicit preferences across benefit and risk attributes of HT. The survey was administered via the Internet to 523 U.S. women aged 46-60 years. Participants evaluated pairs of hypothetical treatments and indicated preferences using a Likert-based scale. Risks were presented in absolute and relative terms. Satisfaction scores for HT risks and benefits were estimated using random-effects, ordered-probit regression. Maximum acceptable risk (MAR) was calculated as the increase in risk that reduces the satisfaction score for a given level of HT benefit to 0. RESULTS: For both risk versions, the least important attribute (smallest difference in satisfaction scores) was night sweat frequency; heart attack risk was the most important (largest difference in scores). Participants were more willing to accept risks in return for symptom relief when shown absolute vs. relative risks, although differences in MAR were statistically significant only for breast cancer risk. MARs for breast cancer and heart attack exceeded reported rates in most cases. CONCLUSIONS: Many women may be willing to accept risks to control vasomotor symptoms. However, describing risks in different, but technically equivalent, ways affects women's willingness to trade risks for benefits. [ABSTRACT FROM AUTHOR]

BACKGROUND: In an effort to control drug spending, health plans are increasingly shifting specialty drugs from their medical benefit to the pharmacy benefit. One consequence of this trend is that some health plans have both a medical and a pharmacy coverage policy for the same drug. OBJECTIVE: To examine how frequently health plans issue medical and pharmacy benefit policies for the same specialty drug and to evaluate the concordance between plans' medical and pharmacy policies when plans issue both policy types. METHODS: We identified specialty drug coverage policies from the Tufts Medical Center Specialty Drug Evidence and Coverage Database, which includes policies issued by 17 of the largest US commercial health plans. Policies were current as of August 2020. We determined plans that issued both medical and pharmacy policies. Next, we identified drugs with "medical-pharmacy policy pairs," ie, drugs for which a plan issued both a medical and a pharmacy policy. For these pairs, we compared the plan's policies while accounting for the following coverage criteria: patient subgroups (patients must meet certain clinical criteria), prescriber requirements (a specialist must prescribe the drug), and step therapy protocols (patients must first fail alternative treatments). We considered medical-pharmacy policy pairs to be discordant if coverage criteria differed, eg, the medical policy included a prescriber requirement but the pharmacy policy did not. RESULTS: Eight plans issued separate medical and pharmacy benefit coverage policies for the same specialty drug and indication. Among these 8 plans, we identified 1,619 medical-pharmacy policy pairs. Eighty-six percent of pairs were concordant (1,386/1,619), and 14% were discordant (233/1,619). Discordance was most often due to differences in plans' application of step therapy protocols (184/233), followed by prescriber requirements (52/233) and patient subgroups (25/233). Forty pairs were discordant in multiple ways. Of discordant pairs, medical policies were more restrictive 41% (96/233) of the time; pharmacy policies were more restrictive 54% (125/233) of the time; 5% of the time (12/233), the medical policy was more restrictive in some ways, but the pharmacy policy was more restrictive in others. Overall, plans imposed coverage restrictions in their medical and pharmacy policies with similar frequencies. CONCLUSIONS: Commercial health plans' medical and pharmacy coverage policies for the same specialty drugs tended to be concordant, although we found coverage criteria to be discordant 14% of the time. Medical and pharmacy policies that are inconsistent in their coverage criteria and restrictions complicate, and potentially hinder, patients' access to specialty drugs. DISCLOSURES: Drs Kauf, O'Sullivan, and Strand were employees of Alkermes, Inc., when the study was conducted and may own stock in the company. Mx Levine, Mr Panzer, and Dr Chambers have no conflicts of interest to disclose. This research study was supported by Alkermes, Inc.

Background: Prolonged postoperative ileus (POI) is the predominant cause of extended hospitalization after bowel resection surgery. Alvimopan accelerates gastrointestinal recovery, potentially reducing health care costs. We examined the value of alvimopan in reducing prolonged POI and length of stay for patients undergoing abdominal surgery using different definitions of POI., Study Design: We developed a decision analytic model to examine costs and outcomes associated with postoperative treatment with either an accelerated care pathway (ACP) only or alvimopan+ACP. To represent an overall perspective for alvimopan, data from four phase 3 bowel resection trials and one phase 4 radical cystectomy trial were used to populate the model with 3 different definitions of POI. The period analyzed included start of surgery to 7 days post discharge. Costs were obtained from standard US costing sources and are reported in 2015 US dollars. Due to variations in published definitions of POI, alternative definitions based on adverse event reports, NG tube insertion, and time to food toleration were examined., Results: The combined clinical trial data included 1,003 ACP and 1,013 alvimopan+ACP patients. When POI was reported as an adverse event, the incidence of POI was significantly lower with alvimopan+ACP (n = 70 [7%]) vs ACP alone (n = 148 [15%]; p < 0.0001). Time to discharge order written was shorter for patients with POI who were treated with alvimopan+ACP than with ACP (202 ± 115 hours vs 266 ± 138 hours; p < 0.0001). As a result, costs were $731 lower with alvimopan+ACP ($17,835) vs ACP ($18,566). Alternative definitions of POI produced similar results., Conclusions: The addition of alvimopan to existing treatment pathways for patients undergoing abdominal surgery can reduce overall hospital costs., (Copyright © 2015 American College of Surgeons. Published by Elsevier Inc. All rights reserved.)

Purpose: We evaluated the effect of alvimopan treatment vs placebo on health care utilization and costs related to gastrointestinal recovery in patients treated with radical cystectomy in a randomized, phase 4 clinical trial., Materials and Methods: Resource utilization data were prospectively collected and evaluated by cost consequence analysis. Hospital costs were estimated from 2012 Medicare reimbursement rates and medication wholesale acquisition costs. Differences in base case mean costs between the study cohorts for total postoperative ileus related costs (hospital days, study drug, nasogastric tubes, postoperative ileus related concomitant medication and postoperative ileus related readmissions) and total combined costs (postoperative ileus related, laboratory, electrocardiograms, nonpostoperative ileus related concomitant medication and nonpostoperative ileus related readmission) were evaluated by probabilistic sensitivity analysis using a bootstrap approach., Results: Mean hospital stay was 2.63 days shorter for alvimopan than placebo (mean±SD 8.44±3.05 vs 11.07±8.23 days, p=0.005). Use of medications or interventions likely intended to diagnose or manage postoperative ileus was lower for alvimopan than for placebo, eg total parenteral nutrition 10% vs 25% (p=0.001). Postoperative ileus related health care costs were $2,340 lower for alvimopan and mean total combined costs were decreased by $2,640 per patient for alvimopan vs placebo. Analysis using a 10,000-iteration bootstrap approach showed that the mean difference in postoperative ileus related costs (p=0.04) but not total combined costs (p=0.068) was significantly lower for alvimopan than for placebo., Conclusions: In patients treated with radical cystectomy alvimopan decreased hospitalization cost by reducing the health care services associated with postoperative ileus and decreasing the hospital stay., (Copyright © 2014 American Urological Association Education and Research, Inc. Published by Elsevier Inc. All rights reserved.)

Background: Thrombocytopenia is associated with the natural history of hepatitis C virus (HCV) infection and anti-viral therapy. Recent, national estimates of the clinical burden of thrombocytopenia among HCV-infected individuals in the United States are unavailable. Bi-yearly data from the 1999-2000 to 2007-2008 National Health and Nutrition Examination Surveys (NHANES) were used to examine the prevalence of thrombocytopenia among HCV-infected individuals in the United States., Results: Among 467 HCV-infected individuals in the survey (weighted population = 3,597,039), mean weighted age was 46.7 years (standard deviation = 15.5) and 61.7% were male. Overall, 7.6% met the study definition of TCP at the 150 × 10(9)/L threshold; 4.5%, 2.0%, and 0.8% had platelet counts below 125, 100, and 75 × 10(9)/L, respectively. The 2-year weighted prevalences of thrombocytopenia (150 × 10(9)/L threshold) from 1999-2008 were 4.9%, 8.6%, 6.5%, 4.1%, and 12.9%. The unadjusted biannual time trend (odds ratio) was 1.16 (95% confidence interval = 0.82-1.64). In the two adjusted models, the odds by time ranged from 1.24-1.40, depending on whether the model included demographic or laboratory variables or both, but did not reach statistical significance. Age was positively and significantly related to thrombocytopenia status., Conclusions: As the HCV-infected population ages, the prevalence of thrombocytopenia is expected to rise. This study provides limited evidence of such an effect at the national level.

Background: Some patients with chronic hepatitis C virus (HCV) infection forego treatment due to concerns about treatment efficacy, treatment duration, and side effects., Objective: The purpose of this study was to quantify patient preferences among possible outcomes associated with new, direct-acting antiviral agents (DAAs) for the treatment of HCV infection and determine which treatment features are most important to patients in making treatment decisions., Methods: Adult participants with a self-reported physician diagnosis of HCV infection in five countries completed a web-enabled, choice-format conjoint analysis survey. The survey presented participants with 20 treatment-choice questions. Each treatment-choice question included a pair of hypothetical treatment profiles with varying levels of six attributes: treatment duration; chance of getting rid of the virus completely (i.e. likelihood of a sustained virologic response [SVR]); weeks on an additional, third medicine (i.e. a DAA); risk of a severe rash; risk of severe anemia; and number of times a day the third medicine is taken. Treatment-choice questions were based on a pre-determined experimental design with known statistical properties. Random-parameters logit was used to estimate preference weights for all attribute levels and the mean relative importance of each attribute., Results: 284 participants completed the survey. Likelihood of an SVR was the most important outcome to participants, followed by severe anemia risk, severe rash risk, therapy type (a combination of total weeks of treatment and weeks on the third medicine), and dosing of the third medicine. Controlling for other factors, preferences were similar across all therapy types examined., Conclusion: Patients with HCV infection indicate a willingness to accept an increased risk of side effects for sufficient improvement in the likelihood of treatment response.

Objectives: To evaluate the cost-effectiveness of immunoprophylaxis against respiratory syncytial virus (RSV) infections with palivizumab based on actual cost and observed incidence rates in various pediatric risk groups., Design: Decision tree analysis comparing children with various combinations of the following indications: chronic lung disease, congenital heart disease, or prematurity (≤32 weeks gestation), and children with none of these indications. One-way sensitivity analyses and Monte Carlo simulations were used to quantify parameter uncertainty., Setting: Florida during the 2004-2005 RSV season., Participants: A total of 159,790 Medicaid-eligible children aged 0 to 2 years., Intervention: Palivizumab prophylaxis compared with no prophylaxis., Outcomes Measure: Incremental cost (2010 US dollars) per hospitalization for RSV infection avoided., Results: The mean cost of palivizumab per dose ranged from $1661 for infants younger than 6 months of age to $2584 for children in their second year of life. Among preterm infants younger than 6 months of age without other indications, immunoprophylaxis with palivizumab cost $302,103 (95% confidence interval, $141,850-$914,798) to prevent 1 RSV-related hospitalization. Given a mean cost of $8910 for 1 RSV-related hospitalization in this subgroup, palivizumab would be cost-neutral at a per-dose cost of $47. Incremental cost-effectiveness ratios for the other subgroups ranged from $361,727 to more than $1.3 million per RSV-related hospitalization avoided in children up to 2 years of age with chronic lung disease and no additional risk factors. Younger age and multiple indications were associated with improvements in the incremental cost-effectiveness ratio., Conclusions: The cost of immunoprophylaxis with palivizumab far exceeded the economic benefit of preventing hospitalizations, even in infants at highest risk for RSV infection.

Background: Abacavir sulfate (abacavir) is associated with a hypersensitivity reaction (HSR) that affects 5-8% of patients. While serious complications are rare, failure to identify it, or abacavir re-challenge following HSR, can be fatal. Genetic screening for HLA-B*5701 can identify patients who are likely to experience an HSR and reduces the incidence of the reaction., Objective: We assessed the intrinsic and practical value, from the US healthcare system perspective, of prospective HLA-B*5701 screening among a population of antiretroviral-naive patients without elevated risk factors for cardiovascular disease, plasma HIV RNA >100,000 copies/mL, or pre-existing renal insufficiency., Methods: Two approaches were used to evaluate the costs and benefits of prospective screening. First, the efficiency of HLA-B*5701 screening compared with no screening prior to abacavir initiation (intrinsic value of screening) was evaluated using a 60-day decision-tree model. Next, the practical value of screening was assessed using a lifetime discrete-event simulation model that compared HLA-B*5701 screening prior to abacavir use versus initiation with a tenofovir-containing regimen. Screening-effectiveness parameters were taken from an open-label trial that incorporated screening prior to abacavir initiation and other published studies. Treatment efficacy was derived from clinical trials. Modelling assumptions, costs ($US, year 2007 values) and other parameters were derived from published sources, primary data analysis and expert opinion. Multiple one-way sensitivity and scenario analyses were performed to assess parameter uncertainty. The primary outcome measure for the short-term screening versus no screening analysis was cost per patient. For the long-term analysis, outcomes were presented as QALYs. Costs and effects were discounted at 3% per year., Results: Over the first 60 days of treatment, prospective screening prior to abacavir initiation cost an additional $US17 per patient and avoided 537 HSRs per 10,000 patients. The per-patient cost of screening was sensitive to the cost of the genetic test, HSR costs and screening performance. In the lifetime model, screening-informed abacavir use was more effective and less costly than initiation with a tenofovir-containing regimen in the base case and in sensitivity analyses., Conclusions: Our results suggest that prospective HLA-B*5701 screening prior to abacavir initiation produces cost savings and should become a standard component of HIV care.

Although sickle cell disease (SCD) is marked by high utilization of medical resources, the full cost of care for patients with SCD, including care not directly related to SCD, is unknown. The purpose of this study was to estimate the total cost of medical care for a population of children and adults with SCD. We used data from individuals diagnosed with SCD enrolled in the Florida Medicaid program during 2001-2005 to estimate total, SCD-related, and non-SCD-related cost per patient-month based on patient age at the time of health care use. Across the 4,294 patient samples, total health care costs generally rose with age, from $892 to $2,562 per patient-month in the 0-9- and 50-64-year age groups, respectively. Average cost per patient-month was $1,389. Overall, 51.8% of care was directly related to SCD, the majority of which (80.5%) was associated with inpatient hospitalizations. Notably, non-SCD-related costs were substantially higher than those reported for the general US population. These results suggest a discounted (3% discount rate) lifetime cost of care averaging $460,151 per patient with SCD. Interventions designed to prevent SCD complications and avoid hospitalizations may reduce the significant economic burden of the disease.

Background: Although clinical research is integral to the advancement of medical knowledge, physicians face a variety of obstacles to their participation as investigators in clinical trials. We examined factors that influence the participation of gastroenterologists and hepatologists in clinical research., Methods: We surveyed 1050 members of the American Association for the Study of Liver Diseases regarding their participation in clinical research. We compared the survey responses by specialty and level of clinical trial experience., Results: A majority of the respondents (71.6%) reported involvement in research activities. Factors most influential in clinical trial participation included funding and compensation (88.3%) and intellectual pursuit (87.8%). Barriers to participation were similar between gastroenterologists (n = 160) and hepatologists (n = 189) and between highly experienced (n = 62) and less experienced (n = 159) clinical researchers. These barriers included uncompensated research costs and lack of specialized support. Industry marketing was a greater influence among respondents with less trial experience, compared to those with extensive experience (15.7% vs 1.6%; P < .01). Hepatologists and respondents with extensive clinical trial experience tended to be more interested in phase 1 and 2 studies, whereas gastroenterologists and less experienced investigators were more interested in phase 4 studies., Conclusion: This study suggests that the greatest barrier to participation in clinical research is lack of adequate resources. Respondents also favored industry-sponsored research with less complex trial protocols and studies of relatively short duration.

Objective: To quantify the burden of pediatric hepatitis C virus (HCV) disease over the coming decade., Study Design: Using national Census results and published and unpublished data, we constructed estimates of HCV prevalence, incidence, rate of vertical transmission, sustained viral response (SVR), and severe complications of infection. Using these figures, we generated a projection model for pediatric HCV outcomes, and we then performed a sensitivity analysis by altering the rates of fibrosis development and SVR., Results: A prevalence of 23,048 to 42,296 pediatric patients with chronic HCV combined with 7200 new cases from vertical transmission was used for further calculations. Over the next decade, estimated screening costs were 26 million US dollars, monitoring costs ranged from 117 million US dollars to 206 million US dollars, and treatment costs ranged from 56 million US dollars to 104 million US dollars., Conclusions: To date, pediatric HCV has received relatively little attention, but it will have a significant economic impact over the next 10 years if changes in practice are not made.

This retrospective cohort study used North Carolina Medicaid paid-claims data to assess clinical and economic outcomes of treatments for urinary tract infection (UTI). The study population comprised female Medicaid recipients, between 15 and 64 years of age, with a paid claim filed for a primary diagnosis of UTI or acute UTI from January 1 to June 30, 1994, who were treated with ciprofloxacin, nitrofurantoin, or trimethoprim/sulfamethoxazole (TMP/SMZ). Patients had follow-up for 6 months after the primary diagnosis. Patients who did not receive further treatment for UTI with 1 of the 3 drugs within 30 days after initial treatment were assumed to be cured. Costs were measured as the sum of reimbursements for UTI-related medical services and drug treatments. Outcomes for 409 patients were assessed. Cure rates of initial treatment with ciprofloxacin, nitrofurantoin, and TMP/SMZ were 81%, 88%, and 93%, respectively. Cost-effectiveness ratios of initial treatment with the 3 drugs were $150.80, $81.20, and $69.00, respectively. When efficacy rates generated from published randomized clinical studies were applied, cost-effectiveness ratios for the 3 drugs were $130.96, $86.17, and $72.00, respectively. A decision model of treatment pattern and associated costs is presented. Several patient variables indicate that the ciprofloxacin group included more severe cases of UTI than did the other groups. Study limitations, confounders, and future research suggestions are discussed. Our results show that treatment for >7 days results in a better cure rate regardless of the drug used than does treatment for < or =7 days and that TMP/SMZ is the most cost-effective of the 3 drugs for UTI or acute UTI.