Real-world evidence in the reassessment of oncology therapies: payer perceptions from five countries.

Aim: This study explored the perceived value of real-world evidence (RWE) in the reassessment of oncology therapies by collecting the perspectives of health technology assessment/payer decision-makers. Materials & methods: A web-based survey was conducted using the Market Access Transformation Rapid Payer Response online portal. 30 participants from France, Germany, Spain, the UK and the USA were recruited based on their expertise. Results: Participants agreed that the most common uses of RWE are to confirm efficacy and safety results from randomized controlled trials and to reevaluate the projected utilization of an oncology therapy. We found variability in other reported uses of RWE. Conclusion: The organizations developing RWE should ensure that their plans recognize the heterogeneity in payer perceptions.

Maintenance and Concomitant Therapy Use with Chlormethine Gel Among Patients with Stage IA/IB Mycosis Fungoides-Type Cutaneous T-Cell Lymphoma (MF-CTCL): A Real-World Evidence Study.

INTRODUCTION: Chlormethine (CL) gel is a skin-directed therapy approved for treatment of stage IA/IB mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) in the USA. MF-CTCL has a chronic clinical course, requiring long-term maintenance therapy with one or more therapies. This analysis describes real-world patterns of maintenance therapy and use of concomitant therapy with CL gel among patients with stage IA/IB MF-CTCL. METHODS: In a US-based registry, MF-CTCL patients treated with CL gel were enrolled between 3/2015 and 10/2018 across 46 centers and followed for up to 2 years. Patient demographics, clinical characteristics, CL gel treatment patterns, concomitant treatments, clinical response, and adverse events (AEs) were collected from medical records. Descriptive statistics are reported. RESULTS: Of the 206 patients with stage IA/IB MF-CTCL, 58.7% were male, and average age was 60.7 years with 4.6 years since diagnosis. Topical steroids, phototherapy, and topical retinoids were used concomitantly with CL gel in 62.6%, 26.2%, and 6.3% of patients, respectively. Most concomitant therapies (up to 85%) were started before CL gel initiation and, in about half of the cases (up to 57%), were used concurrently for ≥ 12 months. Overall, 158 (76.7%) patients experienced partial response (PR) and 144 continued with maintenance therapy. After achieving PR, most patients (74.3%) kept the same maintenance therapy schedule, most commonly once daily. Of patients who had any skin-related AE (31.6%) or skin-related AEs associated with CL gel (28.2%), nearly half experienced CL gel treatment interruption and ~40% had a dosing reduction. The observed real-world treatment patterns were concordant with National Comprehensive Cancer Network (NCCN) guidelines. CONCLUSION: The study results suggest that continuing CL gel maintenance therapy and combining treatments with CL gel are common practice in the real-world setting, with most maintained on a stable dosing schedule. Careful management of AEs may help patients maintain long-term optimal dosing with less treatment interruptions and dosing reductions.

The Role of the Surgeon on Outcomes of Vaginal Prolapse Surgery With Mesh.

INTRODUCTION: Adverse outcomes after surgery for pelvic organ prolapse (POP) with mesh are often attributed to the mesh material with little attention paid to the influence of surgeon factors. We used a national data set to determine whether surgeon case volume and specialty influenced vaginal prolapse surgery outcomes with mesh. MATERIALS AND METHODS: Public Use File data on a 5% random national sample of female Medicare beneficiaries were obtained from the Centers for Medicare and Medicaid Services. Women with a diagnosis of POP who underwent surgery with mesh between 2007 and 2008 were identified by relevant International Classification of Diseases, 9th Revision, Clinical Modification and Current Procedural Terminology, 4th Edition procedure codes. Outcomes were compared by surgeon case volume and specialty. RESULTS: From 2007 to 2008, 1657 surgeries for POP were performed with mesh. Low-, intermediate-, and high-volume surgeons performed 881 (53%), 408 (25%), and 368 (22%) of the cases with mesh, respectively. The cumulative reoperation rates for low-, intermediate-, and high-volume providers were 6%, 2%, and 3%, respectively. The difference in reoperation rates between low and intermediate and low- and high-volume surgeons was statistically significant (P = 0.007 and 0.003, respectively). There was no significant difference in reoperation rates between gynecologists and urologists when vaginal mesh was implanted for POP surgery. CONCLUSIONS: Low-volume surgeons performed most of the vaginal prolapse repairs with mesh and had significantly higher reoperation rates. Surgeon experience must be a consideration when reporting mesh-related complications of POP surgery.

Caring for Children With Tuberous Sclerosis Complex: What Is the Physical and Mental Health Impact on Caregivers?

Disease burden associated with tuberous sclerosis complex, a genetic disorder characterized by benign tumor growth including lesions in multiple organs, puts tremendous demands on families. This analysis examines the physical and mental health burden of tuberous sclerosis complex caregivers in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex caregivers collected information; descriptive analyses were conducted on age-based subgroups. A total of 275 caregivers of tuberous sclerosis complex patients responded. Mean patient age ≤ 18 years was 6.9 (±4.4) and 42.3 (±18.2) for patients >18 years of age. Caregivers reported multiple tuberous sclerosis complex manifestations and high health care utilization for patients. Caregivers spending more time on doctor visits or researching tuberous sclerosis complex had lower physical and mental health-related quality of life scores and more depressive symptoms. Tuberous sclerosis complex caregivers had significantly lower physical and mental health-related quality of life scores and more depressive symptomatology compared to US healthy adult population norms.

Characteristics and temporal trends in patient registries: focus on the life sciences industry, 1981-2012.

PURPOSE: Patient registries are used to monitor safety, examine real-world effectiveness, and may potentially contribute to comparative effectiveness research. To our knowledge, life sciences industry (LSI)-sponsored registries have not been systematically categorized. This study represents a first step toward understanding such registries over time. METHODS: Studies described as registries were identified in the ClinicalTrials.gov database. Characteristics from these registry records were abstracted and analyzed. RESULTS: Of 1202 registries identified, approximately 47% reported LSI sponsorship. These 562 LSI registries varied in focus: medical devices (n = 193, 34%), specific drugs (n = 173, 31%), procedures (n = 29, 5%), or particular diseases (n = 139, 25%). Thirty-three registries (<6%) evaluated pregnancy outcomes. The most common therapeutic area was cardiovascular (n = 234, 42%); others included endocrinology, immunology, oncology, musculoskeletal disorders, and neurology. The two most often measured outcomes were clinical effectiveness and safety, each of which appeared in 363/562 (65%) of LSI registries. Other outcomes included real-world clinical practice patterns (n = 122, 22%), patient-reported outcomes (n = 106, 19%), disease epidemiology/natural history (n = 69, 12%), and economic outcomes (n = 30, 5%). The number of LSI registries and their geographic diversity has increased over time. CONCLUSIONS: The LSI registries represent a substantial proportion of all patient registries documented in ClinicalTrials.gov. These prospective studies are growing in number and encompass diverse therapeutic areas and geographic regions. Most registries measure multiple outcomes and capture real-world data that may be unavailable through other study designs. This classification of LSI registries documents their use for studying heterogeneity of diseases, examining treatment patterns, measuring patient-reported outcomes, examining economic outcomes, and performing comparative effectiveness research.

Trends in management of pelvic organ prolapse among female Medicare beneficiaries.

OBJECTIVE: In the last decade, many new surgical treatments have been developed to achieve less-invasive approaches to prolapse management. However, limited data exist on how the patterns of care for women with pelvic organ prolapse (POP) may have changed over the last decade, and whether mesh implantation techniques have influenced the type of specific compartment repair performed. We used a national data set to analyze the temporal trends in patterns of care for women with POP. STUDY DESIGN: Data were obtained from Public Use Files from the Centers for Medicare and Medicaid Services for a 5% random sample of national beneficiaries with an International Classification of Diseases, Ninth Revision, Clinical Modification diagnosis of POP from 1999 through 2009. Current Procedural Terminology, 4th Edition and International Classification of Diseases, Ninth Revision, Clinical Modification procedure codes were used to evaluate nonsurgical and surgical management trends for this cohort. Types of surgery were categorized by prolapse compartment and combinations of repairs. After 2005, when applicable codes became available, mesh or graft repairs were also analyzed. RESULTS: Over the study time period, the number of women with a diagnosis of POP in any 1 year in our 5% sample of Medicare beneficiaries remained relatively stable (range, 21,245-23,268 per year). Rates of pessary insertion were also consistent at 11-13% over the study period. Of the women with a prolapse diagnosis, 14-15% underwent surgical repair, and there was little change over time in surgical management patterns based on compartment. Most commonly, multiple compartments were repaired simultaneously. There was a rapid increase in mesh use such that in 2009, 41% of all women who underwent surgery (5.8% of the total cohort) had mesh or graft inserted in their repair. Hysterectomy rates for prolapse decreased over time. Rates of vault suspension at the time of hysterectomy for prolapse were low; however, they showed a relative increase over time (22% in 1999 to 26% in 2009). CONCLUSION: Patterns and rates of prolapse repairs remained relatively unchanged from 1999 through 2009, with an exception of a rapid rise in mesh use. These data suggest that the majority of mesh techniques were used for augmentation purposes only, but did not result in an increase in apical repairs performed in the United States. There remains a disappointingly low rate of vault suspension repairs concomitantly at time of hysterectomy for POP.

The humanistic burden of head and neck cancer: a systematic literature review.

BACKGROUND: Head and neck cancer (HNC) and its treatment can affect communication, nutrition, and physical appearance, and the global impact of this disease on patients' quality of life may be substantial. OBJECTIVE: The aim of this systematic literature review was to describe the impact of HNC and its treatment on the physical, emotional, and social well-being of patients over time, by examining longitudinal studies of patient-reported outcomes (PRO) evaluating these domains. METHODS: Databases (MEDLINE and Embase) were searched to identify studies published in English between January 2004 and January 2014 analyzing the humanistic aspects of HNC in adult patients. Additional relevant publications were identified through manual searches of abstracts from recent conference proceedings. RESULTS: Of 1,566 studies initially identified, 130 met the inclusion criteria and were evaluated in the assessment. Investigations using a variety of PRO instruments in heterogeneous patient populations consistently reported that PRO scores decrease significantly from diagnosis through the treatment period, but generally recover to baseline in the first year post-treatment. This trend was observed for many functional domains, although some side effects, such as xerostomia, persisted well beyond 1 year. In addition, considerable evidence exists that baseline PRO scores can predict clinical endpoints such as overall and progression-free survival. CONCLUSIONS: Many aspects of HNC, both disease and treatment specific, profoundly affect patients' quality of life. Improved knowledge of these effects on PRO may allow for more informed treatment decisions and can help physicians to better prepare patients for changes they may experience during therapy. Furthermore, the predictive value of baseline PRO data may enable healthcare providers to identify at-risk patients in need of more intensive intervention.

The economic burden of head and neck cancer: a systematic literature review.

BACKGROUND: This systematic literature review aimed to evaluate and summarize the existing evidence on resource use and costs associated with the diagnosis and treatment of head and neck cancer (HNC) in adult patients, to better understand the currently available data. The costs associated with HNC are complex, as the disease involves multiple sites, and treatment may require a multidisciplinary medical team and different treatment modalities. METHODS: Databases (MEDLINE and Embase) were searched to identify studies published in English between October 2003 and October 2013 analyzing the economics of HNC in adult patients. Additional relevant publications were identified through manual searches of abstracts from recent conference proceedings. RESULTS: Of 606 studies initially identified, 77 met the inclusion criteria and were evaluated in the assessment. Most included studies were conducted in the USA. The vast majority of studies assessed direct costs of HNC, such as those associated with diagnosis and screening, radiotherapy, chemotherapy, surgery, side effects of treatment, and follow-up care. The costs of treatment far exceeded those for other aspects of care. There was considerable heterogeneity in the reporting of economic outcomes in the included studies; truly comparable cost data were sparse in the literature. Based on these limited data, in the US costs associated with systemic therapy were greater than costs for surgery or radiotherapy. However, this trend was not seen in Europe, where surgery incurred a higher cost than radiotherapy with or without chemotherapy. CONCLUSIONS: Most studies investigating the direct healthcare costs of HNC have utilized US databases of claims to public and private payers. Data from these studies suggested that costs generally are higher for HNC patients with recurrent and/or metastatic disease, for patients undergoing surgery, and for those patients insured by private payers. Further work is needed, particularly in Europe and other regions outside the USA; prospective studies assessing the cost associated with HNC would allow for more systematic comparison of costs, and would provide valuable economic information to payers, providers, and patients.

Short-term outcomes of vaginal mesh placement among female Medicare beneficiaries.

OBJECTIVE: To compare short-term outcomes between prolapse repairs with and without mesh using a national data set. Mesh use in surgical treatment of pelvic organ prolapse has gained wide popularity. However, mesh complications have increased concomitantly with its use. METHODS: Public Use File data were obtained for a 5% random national sample of female Medicare beneficiaries aged 65 years and older. Women who underwent prolapse surgery were identified using Current Procedural Terminology Coding System, Fourth Edition (CPT-4) codes. Because the code for mesh placement was effected in 2005, we separated patients into 3 cohorts as follows: those who underwent prolapse repairs from 1999 to 2000 (presumably without mesh), those who underwent repairs from 2007 to 2008 (presumably without mesh), and those with mesh (based on CPT-4 code 57267) from 2007 to 2008. One-year outcomes were identified using International Classification of Diseases, Ninth Revision diagnosis and procedure codes and CPT-4 procedure codes. RESULTS: A total of 9180 prolapse repairs without mesh were performed from 1999 to 2000, 7729 without mesh from 2007 to 2008, and 1804 prolapse repairs with mesh from 2007 to 2008. Prolapse reoperation within 1 year of surgery was higher in nonmesh vs mesh cohorts (6%-7% vs 4%, P <.02). Mesh removal rates were higher in mesh vs nonmesh group (4% vs 0%-1%, P <.001). Mesh use was associated with more dyspareunia, mesh-related complications, and urinary retention, even when controlling for concomitant sling. CONCLUSION: Mesh to treat pelvic organ prolapse and stress urinary incontinence was associated with a small decrease in early reoperation for prolapse. This decrease came at the expense of increased rates of pelvic pain, retention, mesh-related complications, and mesh removal.

Outcomes of vaginal prolapse surgery among female Medicare beneficiaries: the role of apical support.

OBJECTIVE: Recurrent pelvic organ prolapse (POP) has been attributed to many factors, one of which is lack of vaginal apical support. To assess the role of vaginal apical support and POP, we analyzed a national dataset to compare long-term reoperation rates after prolapse surgery performed with and without apical support. METHODS: Public use file data on a 5% random national sample of female Medicare beneficiaries were obtained from the Centers for Medicare and Medicaid Services. Women with POP who underwent surgery during 1999 were identified by relevant International Classification of Diseases, 9th Revision, Clinical Modification, and Current Procedural Terminology, Fourth Edition codes. Individual patients were followed-up through 2009. Prolapse repair was categorized as anterior, posterior, or anterior-posterior with or without a concomitant apical suspension procedure. The primary outcome was the rate of retreatment for POP. RESULTS: In 1999, 21,245 women had a diagnosis of POP. Of these, 3,244 (15.3%) underwent prolapse surgery that year. There were 2,756 women who underwent an anterior colporrhaphy, posterior colporrhaphy, or both with or without apical suspension. After 10 years, cumulative reoperation rates were highest among women who had an isolated anterior repair (20.2%) and significantly exceeded reoperation rates among women who had a concomitant apical support procedure (11.6%; P<.01). CONCLUSION: Ten years after surgery for POP, the reoperation rate was significantly reduced when a concomitant apical suspension procedure was performed. This analysis of a national cohort suggests that the appropriate use of a vaginal apical support procedure at the time of surgical treatment of POP might reduce the long-term risk of prolapse recurrence. LEVEL OF EVIDENCE: II.

Association of health-related quality of life with gender in patients with B-cell chronic lymphocytic leukemia.

PURPOSE: This analysis examined associations between gender and health-related quality of life (HRQOL) in patients with B-cell chronic lymphocytic leukemia (CLL) as they initiate therapy for CLL outside the clinical trial setting. METHODS: Baseline data were collected as part of Connect® CLL Registry, a prospective observational study initiated in community, academic, and government centers. Patient demographics and clinical characteristics were provided by clinicians. Patients reported HRQOL using the Brief Fatigue Inventory (BFI), EQ-5D, and Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu). Mean scores were analyzed, with statistical significance of differences determined by ANOVA. Multivariate analysis also considered age and line of therapy. RESULTS: Baseline HRQOL data were available for 1,140 patients: 710 (62 %) men and 430 (38 %) women from 161 centers. Patients were predominantly white (89 %) with mean age 69 ± 11 years. Women reported significantly worse global fatigue (P <0.0001), fatigue severity (P <0.0001), and fatigue-related interference (P = 0.0005) versus men (BFI). Pain/discomfort (P = 0.0077), usual activities (P = 0.0015), and anxiety/depression (P = 0.0117) were significantly worse in women than in men (EQ-5D). With women reporting a better social/family score (P = 0.0238) and men reporting a better physical score (P = 0.0002), the mean FACT-G total score did not differ by gender. However, the mean FACT-Leu total score was better among men versus women (P = 0.0223), primarily because the mean leukemia subscale score was significantly better among men (P <0.0001). Multivariate analysis qualitatively confirmed these findings. CONCLUSIONS: Connect® CLL Registry results indicate that significant differences exist in certain HRQOL domains, as women reported greater levels of fatigue and worse functioning in physical domains.

Comparative outcomes of open versus laparoscopic sacrocolpopexy among Medicare beneficiaries.

INTRODUCTION AND HYPOTHESIS: Since the first reported laparoscopic sacrocolpopexy in 1991, a limited number of single-center studies have attempted to assess the procedure's effectiveness and safety. Therefore, we analyzed a national Medicare database to compare real-world short-term outcomes of open and laparoscopically assisted (including robotic) sacrocolpopexy in a United States sample of patients. METHODS: Public Use File data for a 5% random national sample of all Medicare beneficiaries aged 65 and older were obtained from the Centers for Medicare and Medicaid Services for the years 2004-2008. Women with pelvic organ prolapse were identified using ICD-9 diagnosis codes. CPT-4 procedure codes were used to identify women who underwent open (code 57280) or laparoscopic (code 57425) sacrocolpopexy. Individual subjects were followed for 1 year post-operatively. Outcomes measured, using ICD-9 and CPT-4 codes, included medical and surgical complications and re-operation rates. RESULTS: Seven hundred and ninety-four women underwent open and 176 underwent laparoscopic (including robotic) sacrocolpopexy. Laparoscopic sacrocolpopexy was associated with a significantly increased rate of re-operation for anterior vaginal wall prolapse (3.4% vs 1.0%, p = 0.018). However, more medical (primarily cardiopulmonary) complications occurred post-operatively in the open group (31.5% vs 22.7%, p = 0.023). When sacrocolpopexy was performed with concomitant hysterectomy, mesh-related complications were significantly higher in the laparoscopic group (5.4% vs 0%, p = 0.026). CONCLUSION: Laparoscopic sacrocolpopexy resulted in an increased rate of reoperation for prolapse in the anterior compartment. When hysterectomy was performed at the time of sacrocolpopexy, the laparoscopic approach was associated with an increased risk of mesh-related complications.

Patterns of pessary care and outcomes for medicare beneficiaries with pelvic organ prolapse.

OBJECTIVES: Using a national data set, we sought to assess patterns of pessary care in older women with pelvic organ prolapse (POP) and subsequent outcomes, including rates of complications and surgical treatment of POP. METHODS: Public use files from the US Centers for Medicare and Medicaid Services were obtained for a 5% random national sample of beneficiaries from 1999 to 2000. Diagnostic and procedural codes (International Classification of Diseases, Ninth Revision, Clinical Modification and Current Procedural Terminology, 4th Edition) were used to identify women with POP and those treated with pessary. Individual subjects were followed longitudinally for 9 years. Across this duration, patient care and outcomes (eg, return clinic visits, repeated pessary placements, complications, and rate of surgical treatment of prolapse) were assessed. RESULTS: Of 34,782 women with a condition diagnosed as POP, 4019 women (11.6%) were treated with a pessary. In the initial 3 months after pessary placement, 40% underwent a follow-up visit with the provider who had placed the pessary, and through 9 years after the initial fitting, 69% had such a visit. During this period, 3% of the subjects developed vesicovaginal or rectovaginal fistulas, and 5% had a mechanical genitourinary device complication. Twelve percent of women underwent surgery for POP by 1 year; with 24% by 9 years. CONCLUSIONS: Pessary can be effectively used for the management of POP in older women. Despite this, a low percentage of Medicare beneficiaries undergo pessary fitting. Lack of continuity of care is associated with a small but unacceptable rate of vaginal fistulas.

Effect of a patient support program on access to oral therapy for hematologic malignancies.

PURPOSE: The results of a study assessing the effectiveness of a manufacturer-sponsored assistance program for patients prescribed oral cancer therapies are presented. METHODS: Rates of dispensing success were evaluated in a random sample of patients (n = 1000) who enrolled in the Celgene Patient Support (CPS) program for assistance obtaining lenalidomide or thalidomide over a two-year period and a control group of patients (n = 1000) who registered to receive the drugs under restricted-distribution protocols but did not receive CPS assistance. The main study outcomes were (1) the proportion of patients who actually received medication and (2) the time from prescription approval to the initial dispensing of medication. RESULTS: Despite the complex access issues faced by program enrollees, the proportion of CPS participants who received medication (89%) was comparable to the proportion of patients who received medication in the control cohort (91%). The median time from the approval of prescriptions to the initial dispensing of medication was also comparable in the CPS and control groups (eight days versus five days). The study also evaluated the reasons why medication was not dispensed to CPS enrollees in some cases. CONCLUSION: The percentage of patients who were dispensed prescriptions for lenalidomide or thalidomide did not differ significantly between those who were enrolled in a patient assistance program and those who were not. The median time between prescription authorization and first dispensing was comparable among program and nonprogram patients.

National Ambulatory Medical Care Survey: tobacco intervention practices in outpatient clinics.

Tobacco use remains the leading cause of preventable death. The outpatient medical clinic represents an important venue for delivering evidence-based interventions to large numbers of tobacco users. Extensive evidence supports the effectiveness of brief interventions. In a retrospective database analysis of 11,827 adult patients captured in the 2005 National Ambulatory Medical Care Survey (of which 2,420 were tobacco users), we examined the degree to which a variety of patient demographic, clinical and physician-related variables predict the delivery of tobacco counseling during a routine outpatient visit in primary care settings. In 2005, 21.7% of identified tobacco users received a tobacco intervention during their visit. The probability of receiving an intervention differed by gender, geographic region and source of payment. Individuals presenting with tobacco-related health conditions were more likely to receive an intervention. Most physicians classified as specialists were less likely to intervene. The provision of tobacco intervention services appears to be increasing at a modest rate, but remains well below desirable levels. It is a priority that brief interventions be routinely implemented to reduce the societal burden of tobacco use.

A cost-effectiveness analysis of using azacitidine vs. decitabine in treating patients with myelodysplastic syndromes.

OBJECTIVE: Azacitidine and decitabine are used to treat patients with myelodysplastic syndromes (MDS) in the United States (US). This study sought to assess their relative cost-effectiveness. DESIGN AND METHODS: The authors developed a cost-effectiveness Markov model (1-month cycles) tracking hypothetical cohorts of MDS patients treated with azacitidine or decitabine over 2 years. The model used a US payer perspective and 2009 costs. Health states modeled included MDS with Transfusion Dependence, MDS with Transfusion Independence, Progression to Acute Myelogenous Leukemia (AML), and Death. Incremental cost-effectiveness outcomes included cost per quality-adjusted life year (QALY), cost per life year (LY), cost per patient-month of transfusion independence, and cost per case of AML progression avoided. One-way sensitivity analyses were performed on key model parameters. RESULTS: Compared to decitabine, azacitidine was associated with better survival (1.512 LYs vs 1.292), more QALYs gained (1.041 vs 0.870), more patient-months with transfusion independence (8.328 vs 6.224), and a greater proportion of patients avoiding progression to AML (50.9% vs 28.5%). Total per-patient costs over 2 years for azacitidine were lower than for decitabine ($150,322 vs $166, 212). LIMITATIONS: To inform and update the model over time, it will be important that randomized or observational clinical studies be conducted to directly compare azacitidine and decitabine, provide new information on how these medicines are used, and on their relative clinical effectiveness. CONCLUSION: Results demonstrate that azacitidine provides greater clinical benefit and costs less than decitabine across all key outcomes. These results accentuate the positive role of azacitidine in providing cost-effective care for MDS.

Outcomes of erythropoiesis-stimulating agents in cancer patients with chemotherapy-induced anemia.

PURPOSE: To assess the clinical and economic outcomes among patients with chemotherapy-induced anemia (CIA) treated with United States Food and Drug Administration-approved fixed dosing regimens of erythropoiesis-stimulating agents (ESA). METHODS: Data were employed from the Dosing and Outcomes Study of Erythropoiesis-Stimulating Therapies (DOSE) registry to evaluate CIA patients who were initiated on either epoetin alfa (EPO) 40,000 Units (U) or darbepoetin alfa (DARB) 500 micrograms (mcg) between January 1, 2006 and May 8, 2009. Study measurements included ESA treatment dose and dose ratio, changes in hemoglobin (Hb) levels from baseline, and cumulative ESA costs. RESULTS: Five hundred forty patients treated in 44 clinical centers were evaluated, of which 420 were initiated on EPO 40,000 U and 120 were initiated on DARB 500 mcg. Both cohorts had similar baseline characteristics, although EPO patients were less likely than DARB patients to have received iron supplementation before ESA initiation (11.4% EPO vs. 20.0% DARB, p = 0.015). The EPO-to-DARB dose ratio based on cumulative ESA dose was 169:1 (U EPO: mcg DARB). EPO patients showed statistically greater Hb improvement compared to DARB patients, and compared to EPO patients, a greater proportion of DARB patients required a blood transfusion (13.9% EPO vs. 22.5% DARB, p = 0.026). Mean cumulative ESA cost was significantly lower for EPO patients than DARB patients ($4,261 EPO vs. $8,643 DARB, p < 0.0001). CONCLUSIONS: These findings reported that patients with CIA achieved more favorable clinical and economic outcomes if initiated with EPO 40,000 U vs. DARB 500 mcg.

Pharmacoeconomic considerations in treating iron overload in patients with ß-thalassaemia, sickle cell disease and myelodysplastic syndromes in the US: a literature review.

Patients with ß-thalassaemia, sickle cell disease (SCD) and myelodysplastic syndromes (MDS) require chronic blood transfusions, which can lead to iron overload and substantial morbidity and mortality. To reduce the excess iron and its deleterious effects, available iron chelation therapy (ICT) in the US includes oral deferasirox or infusional deferoxamine (DFO). The aim of this study was to review and synthesize the available pharmacoeconomic evidence on ICT in patients with ß-thalassaemia, SCD and MDS in the US. We systematically identified and reviewed pharmacoeconomic studies of ICT in patients with ß-thalassaemia, SCD and MDS that either were published in MEDLINE-indexed, English-language journals from 1999 to 2009, or appeared in medical society websites and scientific meeting abstracts. We assessed available cost-of-illness, cost-of-treatment, cost-consequence, cost-effectiveness, utility and patient-satisfaction studies. The majority of the 20 identified studies assessed cost of treatment, mainly focusing on acquisition and administration costs of ICTs. Gaps in the published literature include current data on direct medical costs for patients with MDS, direct medical costs associated with complications of iron overload, direct non-medical costs, indirect costs and patient utilities. Different underlying model assumptions, methodologies and comparators were found in the cost-effectiveness studies, which yielded a broad range of incremental cost-effectiveness ratios for different ICTs. Comprehensive cost-of-illness studies are needed to address data gaps in the published literature regarding the economic burden of iron overload. Comparative-effectiveness studies that evaluate clinical, economic and patient-reported outcomes would help the medical community to better understand the value of different ICTs.

Thromboembolic complications of sling surgery for stress urinary incontinence among female Medicare beneficiaries.

OBJECTIVES: To determine the rate of thromboembolic complications after sling surgery for stress urinary incontinence among female Medicare beneficiaries aged 65 and older. METHODS: We analyzed the 1999-2001 Medicare public use files provided by the Centers for Medicare and Medicaid Services on a 5% national random sample of beneficiaries. Women undergoing sling procedures from January 1, 1999 to July 31, 2000 were identified by the Physicians Current Procedural Terminology Coding System (4th edition) codes and tracked for 12 months. Diagnoses of postoperative thromboembolism were identified with International Classification of Diseases (9th revision) codes. Multivariate analysis was used to determine independent risk factors for developing a thromboembolic event. RESULTS: A total of 1356 slings were performed on patients in the 5% sample of female Medicare beneficiaries during the 18-month index period. Concomitant prolapse surgery was performed in 467 (34.4%) cases. At 3 months after surgery, thromboembolic complications had occurred in 0.9% women undergoing a sling alone and in 2.2% women undergoing concomitant prolapse surgery (P = .05). Multivariate analysis revealed that concomitant prolapse surgery was associated with nearly 3 times the odds of thromboembolic complications (odds ratio 2.86, 95% confidence interval 1.10-7.45). CONCLUSIONS: Our results show a low rate of thromboembolism after an isolated sling procedure. However, we found an increased rate of deep venous thrombosis and pulmonary embolism among women undergoing sling surgery with prolapse repair, which emphasizes the need for appropriate deep venous thrombosis prophylaxis in this patient group.

The effect of concomitant prolapse repair on sling outcomes.

PURPOSE: We analyzed the effect of concomitant prolapse surgery performed at the time of sling surgery on short-term postoperative outcomes in women with urinary incontinence. MATERIALS AND METHODS: We analyzed 1999 to 2001 Medicare claims data on a 5% national random sample of female beneficiaries who underwent sling procedures. Subjects were tracked for 12 months after surgery to assess short-term complications. Concomitant prolapse repairs and prolapse repairs performed in the first 12 months after sling surgery were identified by CPT-4 procedure codes. Postoperative complications and treatments were identified by ICD-9 diagnosis codes and CPT-4 procedure codes, respectively. Bivariate and multivariate analyses were performed to measure the effect of concomitant prolapse surgery on sling outcomes. RESULTS: Concomitant prolapse repairs were performed in 34.4% of sling cases. Women who underwent prolapse repair at the time of the sling surgery were significantly more likely to be diagnosed with postoperative outlet obstruction (9.4% vs 5.5%, p <0.007) than those who did not. Women who underwent concomitant prolapse repair were less likely to undergo a repeat procedure for stress incontinence in postoperative year 1 (4.7% vs 10.2%, p = 0.0005). Multivariate analysis revealed that women who underwent prolapse repair at the time of the sling surgery were significantly less likely to undergo a reoperation for prolapse within 1 year after the sling surgery (OR 0.31, 95% CI 0.22-0.44). CONCLUSIONS: Our findings suggest that addressing prolapse at the time of stress incontinence surgery may avoid an early repeat operation for either prolapse or stress incontinence. However, rates of postoperative outlet obstruction are higher.