Coverage and use of cancer therapies in the treatment of chronic myeloid leukemia.

OBJECTIVES: This study was designed to assess the effect of tyrosine kinase inhibitor (TKI) use on nonpharmaceutical medical spending for patients with chronic myeloid leukemia (CML), and estimate the association between cost-sharing and the TKI medication possession ratio (MPR). STUDY DESIGN: The retrospective study covered the 13 years from 1997 to 2009. METHODS: Analyses were conducted using a large administrative health insurance claims database covering 45 large employers. From this database, 995 unique patients with CML were identified, with 3,765 patient-years; of these patients, 415 (or 1,689 patientyears) were TKI users. We estimated the association of TKI use with total pharmaceutical spending and total non-pharmaceutical medical spending. In addition, we characterized plan-level cost-sharing rules for TKIs and assessed whether these were associated with the MPR for TKI therapy among CML patients. RESULTS: TKI users averaged $26,406 in annual non-pharmaceutical medical spending, compared with $38,194 for non-users; this was a difference of approximately 30%, which was statistically significant at the 5% level. The median patient out-ofpocket payment was $25, which increased to $63 at the 75th percentile and to $122 at the 95th percentile. MPRs were 94.8 at the median cost-sharing level and 100.0 at the 75th percentile and higher. There was no statistically significant association between cost-sharing and MPR. CONCLUSIONS: Use of TKIs was associated with a 30% reduction in non-pharmaceutical medical spending for CML patients. This difference is approximately equal to 40% of the incremental pharmaceutical cost associated with using TKI therapy. The net annual cost of TKI therapy is roughly $15,000. An informal calculation suggests that this is well within the range of conventional cost-effectiveness thresholds. On balance, coverage of TKIs is relatively generous, with the vast majority of patients exhibiting high levels of adherence to therapy.

Coverage and preventive screening.

CONTEXT: Preventive care has been shown as a high-value health care service. Many employers now offer expanded coverage of preventive care to encourage utilization. OBJECTIVE: To determine whether expanding coverage is an effective means to encourage utilization. DESIGN: Comparison of screening rates before and after introduction of deductible-free coverage. SETTING: People insured through large corporations between 2002 and 2006. PATIENTS OR OTHER PARTICIPANTS: Preferred Provider Organization (PPO) enrollees from an employer introducing deductible-free coverage, and a control group enrolled in a PPO from a second employer with no policy change. MAIN OUTCOME MEASURES: Adjusted probability of endoscopy, fecal occult blood test (FOBT), lipid screens, mammography, and Papanicolaou (pap) smears. INTERVENTION: Introduction of first-dollar coverage (FDC) of preventive services in 2003. RESULTS: After adjusting for demographics and secular trends, there were between 23 and 78 additional uses per 1,000 eligible patients of covered preventive screens (lipid screens, pap smears, mammograms, and FOBT), with no significant changes in the control group or in a service without FDC (endoscopy). CONCLUSIONS: FDC improves utilization modestly among healthy individuals, particularly those in lower deductible plans. Compliance with guidelines can be encouraged by lowering out-of-pocket costs, but patients' predisposing characteristics merit attention.

Cost sharing, family health care burden, and the use of specialty drugs for rheumatoid arthritis.

OBJECTIVES: To examine the impact of benefit generosity and household health care financial burden on the demand for specialty drugs in the treatment of rheumatoid arthritis (RA). DATA SOURCES/STUDY SETTING: Enrollment, claims, and benefit design information for 35 large private employers during 2000-2005. STUDY DESIGN: We estimated multivariate models of the effects of benefit generosity and household financial burden on initiation and continuation of biologic therapies. DATA EXTRACTION METHODS: We defined initiation of biologic therapy as first-time use of etanercept, adalimumab, or infliximab, and we constructed an index of plan generosity based on coverage of biologic therapies in each plan. We estimated the household's burden by summing up the annual out-of-pocket (OOP) expenses of other family members. PRINCIPAL FINDINGS: Benefit generosity affected both the likelihood of initiating a biologic and continuing drug therapy, although the effects were stronger for initiation. Initiation of a biologic was lower in households where other family members incurred high OOP expenses. CONCLUSIONS: The use of biologic therapy for RA is sensitive to benefit generosity and household financial burden. The increasing use of coinsurance rates for specialty drugs (as under Medicare Part D) raises concern about adverse health consequences.

The effectiveness of covering smoking cessation services for medicare beneficiaries.

OBJECTIVE: To examine whether reimbursement for Provider Counseling, Pharmacotherapies, and a telephone Quitline increase smoking cessation relative to Usual Care. STUDY DESIGN: Randomized comparison trial testing the effectiveness of four smoking cessation benefits. SETTING: Seven states that best represented the national population in terms of the proportion of those > or = 65 years of age and smoking rate. PARTICIPANTS: There were 7,354 seniors voluntarily enrolled in the Medicare Stop Smoking Program and they were followed-up for 12 months. INTERVENTION(S): (1) Usual Care, (2) reimbursement for Provider Counseling, (3) reimbursement for Provider Counseling with Pharmacotherapy, and (4) telephone counseling Quitline with nicotine patch. MAIN OUTCOME MEASURE: Seven-day self-reported cessation at 6- and 12-month follow-ups. PRINCIPAL FINDINGS: Unadjusted quit rates assuming missing data=smoking were 10.2 percent (9.0-11.5), 14.1 percent (11.7-16.5), 15.8 percent (14.4-17.2), and 19.3 percent (17.4-21.2) at 12 months for the Usual Care, Provider Counseling, Provider Counseling + Pharmacotherapy, and Quitline arms, respectively. Results were robust to sociodemographics, smoking history, motivation, health status, and survey nonresponse. The additional cost per quitter (relative to Usual Care) ranged from several hundred dollars to $6,450. CONCLUSIONS: A telephone Quitline in conjunction with low-cost Pharmacotherapy was the most effective means of reducing smoking in the elderly.

Kidney cancer.

PURPOSE: We quantified the burden of kidney cancer in the United States by identifying trends in the use of health care resources and estimating the economic impact of the disease. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. RESULTS: The incidence of all stages of kidney cancer is increasing in America, particularly T1 disease. Rates are increasing more rapidly in the black than in the white population and survival is worse for black individuals at all stages of diagnosis. Total expenditures for kidney cancer were $401 million in 2000, representing a 46% increase from 1994. Approximately 85% of health care dollars spent on kidney cancer were for inpatient care with steady increases through the 1990s. Regarding treatment, more partial nephrectomies were performed in Medicare patients as the 1990s progressed. Health Care Cost and Utilization Project data showed an increase in the number of inpatient hospitalizations but this trend was not seen in the Centers for Medicare and Medicaid Services data set. Length of stay decreased from 1994 to 2000 in the Health Care Cost and Utilization Project database. The adoption of laparoscopic techniques began to appear in the Veterans Affairs data set in 2001 and it increased thereafter. CONCLUSIONS: Increasing trends in the incidence of and costs associated with kidney cancer have been apparent for more than 10 years. As the population ages and the prevalence of risk factors such as obesity and hypertension increases, the burden of disease will increase significantly. Consideration should be given to expanding tumor registries such as Surveillance, Epidemiology and End Results. Treatment databases could better characterize the cost and effectiveness of treatment for metastatic disease and of trends in the adoption of laparoscopy.

Testis cancer.

PURPOSE: We quantified the burden of testis cancer in the United States by identifying trends in its incidence, its treatment and the use of health care resources to estimate the economic impact of the disease. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. RESULTS: The overall incidence of testis cancer in the United States increased 46% between 1975 and 2001. During the same period the ratio of seminoma to nonseminoma increased and there were fewer men presenting with stage II and III tumors. Survival rates increased successively, attaining the current level of 95.9%. Treatment patterns changed and active surveillance increased as a primary treatment modality. Overall hospitalization rates for men with testis cancer decreased from 1.8/100,000 in 1994 and 1.4/100,000 in 2000. Care for white men shifted to the outpatient setting, which did not occur for black men. The estimated annual expenditure for testis cancer for privately insured individuals between ages 18 and 54 years was $6,236. National estimates of annual medical expenditures placed the total cost of treatment at $21.8 million in 2000, representing an increase of 10% over the total in 1994. Of men with testis cancer 16% missed work for treatment of the disease with an average of 8.4 total hours of work missed. CONCLUSIONS: The cost of testis cancer is estimated at almost $21.8 million annually. It appears to be increasing with time despite a shift to active surveillance treatments and less hospitalization.

Interstitial cystitis and painful bladder syndrome.

PURPOSE: We quantified the burden of interstitial cystitis/painful bladder syndrome on the health care system in the United States. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. Interstitial cystitis was defined based on International Classification of Diseases, 9th revision code 595.1 (interstitial cystitis). For painful bladder syndrome we used the definition International Classification of Diseases, 9th revision code 788.41 (urinary frequency) with code 625.8 (other specified symptoms associated with female genital organs) or 625.9 (unspecified symptoms associated with female genital organs). RESULTS: Between 1992 and 2001 there was a 2-fold increase in the rate of hospital outpatient visits and a 3-fold increase in the rate of physician office visits related to interstitial cystitis. The annualized rate was 102 office visits per 100,000 population. Ambulatory surgery for interstitial cystitis decreased. A diagnosis of interstitial cystitis was associated with a 2-fold increase in direct medical costs. Between 1994 and 2000 annual national expenditures for interstitial cystitis were stable at $37 million but annual costs for painful bladder syndrome increased from $481 million to $750 million. CONCLUSIONS: Although interstitial cystitis accounts for a small percent of health care visits, its economic burden is substantial. Because of misdiagnosis, the true burden of interstitial cystitis/painful bladder syndrome on the health care system in the United States is probably underestimated in administrative data that rely only on physician coding to identify the disorder. The greatest part of the disease burden is likely not captured in this economic analysis.

Male infertility.

PURPOSE: We assessed male infertility and its treatment in the United States by identifying trends in the use of health care resources and estimating the economic impact of such care. MATERIALS AND METHODS: The analytical methods used to generate these results were previously described. RESULTS: Inpatient hospitalizations for male infertility were relatively few with an overall rate of 0.9/100,000 population. Of these stays 55% were associated with inpatient management of varicocele and 749 of 797 occurred in a rural setting. Between 1994 and 1996 there were 55,411 ambulatory surgery visits with men 25 to 34 years old having the highest use rate of 61/100,000. Men living in the West had the lowest rate of ambulatory surgical visits, which was significantly lower than that for men living in the Northeast and Midwest (29/100,000 vs 104/100,000 and 72/100,000, respectively). The Veterans Affairs health system showed no geographic trend in infertility diagnosis and Hispanic men had the highest frequency of treatment for male infertility, followed by black and then white men. The National Survey for Ambulatory Surgery estimated that 67% of patients undergoing ambulatory surgery for male infertility had a diagnosis of varicocele. In 2000 total expenditures for treating primary male infertility were approximately $17 million. However, adding the cost for assisted reproduction technology cycles placed total cost at about $18 billion. CONCLUSIONS: Infertile males generally seek infertility care outside of traditional reimbursement patterns. For this reason obtaining accurate data regarding the costs associated with male fertility care has proved to be challenging. Given the expense of in vitro fertilization and its attendant technologies, emphasis should be placed on addressing the underlying causes of male infertility. Further systematic examination of the demographics and management of male reproductive dysfunction is warranted.

Bladder and upper tract urothelial cancer.

PURPOSE: While there are data available indicating the incidence and prevalence of bladder and upper tract urothelial cancer, population level data on resource use, costs and patterns of care for these cancers are limited. We quantified the economic impact of caring for patients with bladder and upper tract urothelial cancer, and determined the primary drivers for such costs in the population in the United States. MATERIALS AND METHODS: The analytical methods used to generate these results have been described previously. RESULTS: An increasing proportion of patients with bladder and upper tract urothelial cancer were being treated in the outpatient setting. Most care was provided by urologists and visit frequency was directly related to disease stage. Only a small proportion of patients potentially eligible for chemotherapy, ie those with advanced disease, sought specialized care from oncologists. Office based diagnostic tests such as cytology were not commonly done, although a substantial number of patients with bladder cancer underwent cystoscopy. The use of excretory urography in these patients was decreasing, while the use of computerized tomography was increasing. Ileal conduits were the most frequently performed type of urinary diversion following cystectomy. The cystectomy rate remained unchanged for a decade. Intravesical therapy was done infrequently in patients with bladder cancer. Annual costs for treating bladder and upper urinary tract cancers were $1 billion and $64 million, respectively, in 2000. These costs represented a $164 million increase over 1994 levels, which outpaced inflation. CONCLUSIONS: The costs of treating bladder cancer increased steadily during a 6-year period despite a decrease in inpatient care. Coupled with a lack of substantial change in transurethral resection and cystectomy rates, this suggests that the primary cost drivers are increased outpatient testing, eg computerized tomography and cystoscopy, and an increase in the number of diagnosed cases. Greater focus on selective use of testing modalities, preventive care such as smoking cessation and earlier identification of patients at risk may help curtail further expenditure with regard to managing bladder and upper urinary tract cancers.

Cryptorchidism and hypospadias.

PURPOSE: We quantified the burden of cryptorchidism and hypospadias in the United States by identifying trends in the use of health care resources and estimating the economic impact of the disease. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. RESULTS: Cryptorchidism is managed almost exclusively in the outpatient setting and insufficient data were available on inpatient health care use. Annual inpatient hospitalizations for hypospadias decreased by 75% between 1994 and 2000 from 2,669 (2.2/100,000 children) to 849 (0.6/100,000). Between 1992 and 2000 there were 611,647 physician office visits (96/100,000 per year) with undescended testis listed as the primary diagnosis. The rate of physician office visits for hypospadias by commercially insured boys younger than 3 years increased significantly from 429/100,000 in 1994 to 655/100,000 in 2002. The annualized rate of 18/100,000 in 1994 to 1996 remained relatively constant during these 3 years. Orchiopexy rates were highest in 0 to 2-year-old children, as generally recommended, but a substantial minority of these procedures was done in 3 to 10-year-old children. Geographic variation was noted with higher ambulatory surgery rates in the Northeast and Midwest than in the South and West. Data on commercially insured boys younger than 3 years revealed a 1.5-fold overall increase in the rate of hypospadias surgery from 321/100,000 in 1994 to 468/100,000 in 2002, reflecting the known increase in hypospadias incidence in the United States during the late 1990 s. CONCLUSIONS: Average cost per hospitalization for hypospadias exceeded $5,389 with costs per case higher in children 3 years or older, although there were more cases in children younger than 3 years. The cost per case of hypospadias was higher in the Northeast and South than in the other regions. Data on cryptorchidism are too sparse to provide insights into its downstream economic costs.

Vesicoureteral reflux and ureteroceles.

PURPOSE: We quantified the burden of vesicoureteral reflux and ureteroceles in the United States by identifying trends in the use of health care resources and estimating the economic impact of the diseases. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. RESULTS: Annual inpatient hospitalizations for vesicoureteral reflux increased slightly between 1994 and 2000 from 6.4/100,000 to 7.0/100,000 children, although this trend did not attain statistical significance. Inpatient hospitalization for ureteroceles remained relatively stable between 1994 and 2000 at an average of approximately 2,818 cases annually (1.0/100,000 to 1.1/100,000 children). The rates of visits to physician offices doubled during the 1990 s for commercially insured children (12/100,000 in 1994 and 26/100,000 in 2002) and children covered by Medicaid (43/100,000 in 1996 and 85/100,000 in 2000). Overall the rate of ambulatory surgery visits by commercially insured children increased from 3.4/100,000 in 1998 to 4.8/100,000 in 2002. Similar estimates were not available for children covered by Medicaid. Emergency room use by children with a primary diagnosis of vesicoureteral reflux was rare, reflecting the trend toward delivery of care at physician offices, ambulatory surgery centers and inpatient hospitals. No reliable data could be obtained on outpatient visits or ambulatory surgery for ureteroceles. In 2000 total expenditures for inpatient pediatric vesicoureteral reflux were $47 million, an increase of more than $10 million since 1997. Based on data from 2000 the yearly national inpatient expenditures from ureterocele treatment were an estimated $4 million. CONCLUSIONS: The economic impact of inpatient treatment for pediatric vesicoureteral reflux is considerable. If other service types such as pharmaceuticals, and outpatient and ambulatory services were considered, the observed impact of this condition would certainly be greater. Importantly the costs of prophylactic medical therapy and emerging therapies such as Deflux are not accounted for in this estimate. Furthermore, indirect economic costs, such as work loss to parents of children with pediatric vesicoureteral reflux, were not considered, causing an even greater underestimation of the true costs associated with the condition. Although the National Association of Children's Hospitals and Related Institutions, and the Health Care Cost and Utilization Project Kids' Inpatient Database include data on ureteroceles, the data were limited and, thus, they could not be used to determine reliable cost trends. Available data indicate that the mean cost per ureterocele case was almost $8,000 with little variation observed across ages, regions or sexes.

Male urethral stricture disease.

PURPOSE: The incidence of urethral stricture disease in the United States is unknown. We estimated the impact of urethral stricture disease by determining its prevalence, costs and other measures of burden, including side effects and the need for surgical intervention. MATERIALS AND METHODS: Analyses of services for urethral stricture disease were performed in 10 public and private data sets by epidemiological, biostatistical and clinical experts. RESULTS: Male urethral stricture disease occurred at a rate as high as 0.6% in some susceptible populations and resulted in more than 5,000 inpatient visits yearly. Yearly office visits for urethral stricture numbered almost 1.5 million between 1992 and 2000. The total cost of urethral stricture diseases in 2000 was almost $200 million, not including medication costs. A diagnosis of urethral stricture increased health care expenditures by more than $6,000 per individual yearly in insured populations after controlling for comorbidities. Urethral stricture disease appeared to be more common in the elderly population and in black patients, as measured by health care use. In most data sets services provided for urethral stricture disease decreased with time. Patients with urethral stricture disease appeared to have a high rate of urinary tract infection (41%) and incontinence (11%). CONCLUSIONS: Despite decreasing rates of urethral strictures with time the burden of urethral stricture disease is still significant, resulting in hundreds of millions of dollars spent and hundreds of thousands of caregiver visits yearly.

Erectile dysfunction.

PURPOSE: Male sexual health has taken on increased importance as the United States population ages, develops coexisting medical conditions and undergoes interventions that can affect sexual function. We characterized the burden and severity of disease, treatment patterns and economic consequences of erectile dysfunction. MATERIALS AND METHODS: The analytical methods used to generate these results were described previously. RESULTS: Erectile dysfunction was self-reported by almost 1 of 5 men and it increased with age. Erectile dysfunction may have been more commonly reported in Hispanic men and in those with a history of diabetes, obesity, smoking and hypertension. In most databases black American men had rates of use for office visits and inpatient hospital care that were twice those of other racial groups, although these rates were not controlled for comorbid conditions or other regional and socioeconomic factors. The use of diagnostic tests markedly decreased, while pharmacological therapy, especially with oral phosphodiesterase-5 inhibitors, markedly increased. Penile implant surgery continued to be performed with most patients electing inflatable devices. Extrapolating from the population based estimates of erectile dysfunction prevalence and current use trends showed that the cost of treatment nationwide could reach $15 billion if all men sought treatment. CONCLUSIONS: The burden of disease due to erectile dysfunction in the United States will increase with the aging of the male population, increasing prevalence of comorbid conditions, expanded treatment seeking behavior and costs of pharmaceutical therapy. Accurate estimates of economic cost will require better understanding of pathogenesis, treatment seeking behavior, patient preference for therapies, success of treatments and relative satisfaction with oral pharmacotherapy and penile implants.

Benefit design and specialty drug use.

In this paper we examine spending by privately insured patients with four conditions often treated with specialty drugs: cancer, kidney disease, rheumatoid arthritis, and multiple sclerosis. Despite having employer-sponsored health insurance, these patients face substantial risk for high out-of-pocket spending. In contrast to traditional pharmaceuticals, we find that specialty drug use is largely insensitive to cost sharing, with price elasticities ranging from 0.01 to 0.21. Given the expense of many specialty drugs, care management should focus on making sure that patients who will most benefit receive them. Once such patients are identified, it makes little economic sense to limit coverage.

The Value of Elderly Disease Prevention.

Approximately 100 million elderly will enter Medicare over the next 25 years. We consider the potential benefits of interventions that would reduce or eliminate the most important risk factors for disease and spending. Effective control of hypertension could reduce health care spending $890 billion for these cohorts while adding 75 million disability-adjusted life years (DALYs). Eliminating diabetes would add 90 million life-year equivalents at a cost of $2,761 per DALY. Reducing obesity back to levels seen in the 1980's would have little effect on mortality, but yields great improvements in morbidity (especially heart disease and diabetes) with a cost savings of over $1 trillion. Smoking cessation will have the smallest impact, adding 32 million DALYs at a cost of $9.045 per DALY. While smoking cessation reduces lung disease and lung cancer, but these are relatively low prevalence compared to the other diseases. Its impact on heart disease is negligible. The effects on overall social welfare are unknown, since we do not estimate the costs of these interventions, the costs of any behavioral modification, or the welfare loss due to providers from lower medical spending.

Urologic diseases in America Project: analytical methods and principal findings.

PURPOSE: The burden of urological diseases on the American public is immense in human and financial terms but it has been under studied. We undertook a project, Urologic Diseases in America, to quantify the burden of urological diseases on the American public. MATERIALS AND METHODS: We identified public and private data sources that contain population based data on resource utilization by patients with benign and malignant urological conditions. Sources included the Centers for Medicare and Medicaid Services, National Center for Health Statistics, Medical Expenditure Panel Survey, National Health and Nutrition Examination Survey, Department of Veterans Affairs, National Association of Children's Hospitals and Related Institutions, and private data sets maintained by MarketScan Health and Productivity Management (MarketScan, Chichester, United Kingdom), Ingenix (Ingenix, Salt Lake City, Utah) and Center for Health Care Policy and Evaluation. Using diagnosis and procedure codes we described trends in the utilization of urological services. RESULTS: In 2000 urinary tract infections accounted for more than 6.8 million office visits and 1.3 million emergency room visits, and 245,000 hospitalizations in women with an annual cost of more than 2.4 billion dollars. Urinary tract infections accounted for more than 1.4 million office visits, 424,000 emergency room visits and 121,000 hospitalizations in men with an annual cost of more than 1 billion dollars. Benign prostatic hyperplasia was the primary diagnosis in more than 4.4 million office visits, 117,000 emergency room visits and 105,000 hospitalizations, accounting for 1.1 billion dollars in expenditures that year. Urolithiasis was the primary diagnosis for almost 2 million office visits, more than 600,000 emergency room visits, and more than 177,000 hospitalizations, totaling more than 2 billion dollars in annual expenditures. Urinary incontinence in women was the primary cause for more than 1.1 million office visits in 2000 and 452 million dollars in aggregate primary cause for more than 1.1 million office visits in 2000 and 452 million dollars in aggregate annual expenditures. Other manuscripts in this series present further detail for specific urologic conditions. CONCLUSIONS: Recent trends in epidemiology, practice patterns, resource utilization and costs for urological diseases have broad implications for quality of health care, access to care and the equitable allocation of scarce resources for clinical care and research.