This retrospective cohort analysis was conducted to examine the cost components of administering IV chemotherapy to peripheral T-cell lymphoma (PTCL) patients in the US to inform decision makers.
Methods:
Patients diagnosed with PTCL (ICD-9 code 202.7X) between 1 October 2007 and 30 September 2012 were identified from a US administrative claims database. Costs for patients receiving at least one NCCN recommended IV chemotherapy were assessed using the allowed payment from claim line items, categorized into cost components (study drug costs, IV administration costs and other visit-related services).
Results:
The mean costs to the payer for IV cancer therapy administration in a PTCL patient population averaged about $5735 per visit and $9356 per member per month (PMPM). Across all therapies, mean IV administration costs accounted for $127–$794 per visit and $594–$1808 PMPM, contributing an additional 2–32% to the total costs of the drug alone. Mean other visit-related services costs for treating PTCL accounted for $70–$2487 per visit and $444–$3094 PMPM, contributing an additional 2–74% to the total costs. Combined, these additional costs represent an additional mean cost of $220–$3150 per visit and $1193–$4609 PMPM to the base price of the drug alone.
Limitations:
This study used a convenience sample to identify PTCL patients and only included visits where at least one NCCN recommended IV chemotherapy was administered.
Conclusions:
The costs of IV administration and other visit-related services add measurable costs to the total cost of IV therapy for treating PTCL. When considering the cost of the drug, these additional costs can represent a substantial proportion of the overall costs and must be considered when evaluating the costs of IV treatment options for PTCL. 相似文献
To estimate the direct medical costs associated with managing complications, hypoglycemia episodes, and infections associated with type 2 diabetes expressed in 2012 United States dollars (USD).
Methods:
Direct data analysis and microcosting were used to estimate the costs for an event leading to either a hospital admission or outpatient care, and the post-acute care associated with managing macrovascular and microvascular complications, hypoglycemia episodes, and infections. Data were obtained from many sources, including inpatient and emergency department databases, national physician and laboratory fee schedules, government reports, and literature. Event-year costs reflect the resource use during an acute care episode (initial management in an inpatient or outpatient setting) and any subsequent care provided in the first year. State costs reflect annual resource use required beyond the first year for the ongoing management of complications and other conditions. Costs were assessed from the perspective of a comprehensive US healthcare payer and expressed in 2012 USD.
Results:
Event-year costs (and state costs) for macrovascular complications were as follows: myocardial infarction $56,445 ($1904); ischemic stroke $42,119 ($15,541); congestive heart failure $23,758 ($1904); ischemic heart disease $21,406 ($1904); and transient ischemic attack $7388 ($179). For two microvascular complications the event-year and state costs were assumed the same: $71,714 for end stage renal disease, and $2862 blindness. The event-year cost was $9041 for lower extremity amputations, and $2147 for diabetic foot ulcers. Costs were also determined for managing hypoglycemic episodes: $176–$16,478 (depending on treatment required), and infections: vulvovaginal candidiasis $111, lower urinary tract infection $105.
Conclusions:
This study, which provides up-to-date cost estimates per patient, found that managing macrovascular and microvascular complications results in substantial costs to the healthcare system. This study facilitates conduct of other research studies such as modeling the management of diabetes and estimating the economic burden associated with complications. 相似文献
Evaluate the cost-effectiveness of primary vs secondary prophylaxis (PP vs SP) with pegfilgrastim to reduce the risk of febrile neutropenia (FN) in Non-Hodgkin’s Lymphoma (NHL) patients receiving myelosuppressive chemotherapy from a US payer perspective.
Methods:
A Markov model was used to compare PP vs SP with pegfilgrastim in a cohort of patients receiving six cycles of cyclophosphamide, vincristine, doxorubicin, and prednisone (CHOP) or CHOP plus rituximab (CHOP-R) chemotherapy. Model inputs, including efficacy of pegfilgrastim in reducing risk of FN and costs, were estimated from publicly available sources and peer-reviewed publications. Incremental cost-effectiveness was evaluated in terms of net cost per life-year saved (LYS), per quality-adjusted life-year (QALY) gained, and per FN event avoided over a lifetime horizon. Deterministic and probabilistic analyses were performed to assess sensitivity and robustness of results.
Results:
Lifetime costs for PP were $5000 greater than for SP; however, PP was associated with fewer FN events and more LYs and QALYs gained vs SP. Incremental cost-effectiveness ratios (ICERs) for PP vs SP for CHOP were $13,400 per FN event avoided, $29,500 per QALY gained, and $25,800 per LYS. CHOP-R results were similar ($15,000 per FN event avoided, $33,000 per QALY gained, and $28,900 per LYS). Results were most sensitive to baseline FN risk, cost per FN episode, and odds ratio for reduced relative dose intensity due to prior FN event. PP was cost-effective vs SP in 85% of simulations at a $50,000 per QALY threshold.
Limitations:
In the absence of NHL-specific data, estimates for pegfilgrastim efficacy and relative risk reduction of FN were based on available data for neoadjuvant TAC in patients with breast cancer. Baseline risks of FN for CHOP and CHOP-R were assumed to be equivalent.
Conclusions:
PP with pegfilgrastim is cost-effective compared to SP with pegfilgrastim in NHL patients receiving CHOP or CHOP-R. 相似文献
Rivaroxaban is the first oral factor Xa inhibitor approved in the US to reduce the risk of stroke and blood clots among people with non-valvular atrial fibrillation, treat deep vein thrombosis (DVT), treat pulmonary embolism (PE), reduce the risk of recurrence of DVT and PE, and prevent DVT and PE after knee or hip replacement surgery. The objective of this study was to evaluate the costs from a hospital perspective of treating patients with rivaroxaban vs other anticoagulant agents across these five populations.
Methods:
An economic model was developed using treatment regimens from the ROCKET-AF, EINSTEIN-DVT and PE, and RECORD1-3 randomized clinical trials. The distribution of hospital admissions used in the model across the different populations was derived from the 2010 Healthcare Cost and Utilization Project database. The model compared total costs of anticoagulant treatment, monitoring, inpatient stay, and administration for patients receiving rivaroxaban vs other anticoagulant agents. The length of inpatient stay (LOS) was determined from the literature.
Results:
Across all populations, rivaroxaban was associated with an overall mean cost savings of $1520 per patient. The largest cost savings associated with rivaroxaban was observed in patients with DVT or PE ($6205 and $2742 per patient, respectively). The main driver of the cost savings resulted from the reduction in LOS associated with rivaroxaban, contributing to ～90% of the total savings. Furthermore, the overall mean anticoagulant treatment cost was lower for rivaroxaban vs the reference groups.
Limitations:
The distribution of patients across indications used in the model may not be generalizable to all hospitals, where practice patterns may vary, and average LOS cost may not reflect the actual reimbursements that hospitals received.
Conclusion:
From a hospital perspective, the use of rivaroxaban may be associated with cost savings when compared to other anticoagulant treatments due to lower drug cost and shorter LOS associated with rivaroxaban. 相似文献
Cushing’s disease (CD) is a rare condition with a prevalence of roughly 39 cases per million in the general population. Healthcare costs are substantial for CD patients with either untreated or inadequately controlled disease. This study assesses the 3-year budget impact of pasireotide on a US managed care health plan following pasireotide (Signifor) availability.
Methods:
Two scenarios were evaluated to understand the differences in costs associated with the introduction of pasireotide. The first scenario evaluates the budget impact of pasireotide from the perspective of an entire health plan (total budget impact) and the second from the perspective of the pharmacy budget (pharmacy budget impact). Both scenarios evaluate the annual incremental budget impact with and without pasireotide. Scenario 1 includes costs for medical procedures, drug therapies, monitoring, surgical complications, comorbidities for patients with controlled or uncontrolled CD, and adverse events. Procedures include transsphenoidal surgery, bilateral adrenalectomy, radiotherapy and radiosurgery. Drugs include pasireotide (indicated for CD), mifepristone (indicated to control hyperglycemia secondary to hypercortisolism in patients with Cushing’s syndrome) as well as several off-label treatments (ketoconazole, cabergoline, mitotane). Scenario 2 considers costs solely from the perspective of a health plan pharmacy. Costs are in $2013.
Results:
The estimated total budget impact is $0.0115 per-member per-month (PMPM) in the first year following FDA approval, $0.0184 in the second year, and $0.0194 in the third year. Introduction of pasireotide is expected to increase the pharmacy budget by $0.0257 PMPM in the first year, $0.0363 in the second year, and $0.0360 in the third year.
Limitations:
Model inputs rely on the small body of literature available for Cushing’s disease.
Conclusions:
Cushing’s disease is severe disease with debilitating comorbidities and substantial healthcare costs when untreated or inadequately controlled. The inclusion of pasireotide in a health plan formulary appears to have only a small impact on the total health plan or pharmacy budget. 相似文献
Traditional pathology techniques alone can be insufficient to reliably distinguish between malignant melanoma, dysplastic nevi, and benign nevi in biopsies of suspicious pigmented lesions. Numerous studies have shown high rates of ambiguity when assessing such samples. A novel gene expression assay has been developed to objectively differentiate malignant melanoma from benign nevi.
Objective:
The purpose of this study was to quantify the economic impact of the gene expression assay on a US commercial health plan.
Methods:
The clinical paradigm of care was modeled for a hypothetical cohort of patients with suspicious pigmented lesions that are difficult-to-diagnose. Costs were assigned to each unit of care provided based on 2013 Medicare fee-for-service rates. Patients were followed for 10 years and were modeled to progress according to the natural history of their disease. The total cost of care was calculated for two scenarios: a Reference Scenario, representing current clinical practice, and a Test Scenario, in which each lesion was tested with the gene expression assay and diagnosed. Total cost of care was compared between the two scenarios to determine overall budget impact. Sensitivity analyses were performed to test the robustness of the model.
Results:
The gene expression assay reduces costs by $1268 per patient tested over 10 years, a decrease of 8.3%, after accounting for the cost of the assay. For a health plan with 10 million members, this would translate to over $8 million in savings. The largest portion of this saving comes from reducing the number of missed melanomas, which would otherwise progress to advanced disease. In sensitivity analyses, no single model input changed within a reasonable range of values caused the model to show that the assay was not cost-saving.
Conclusion:
In addition to improving the diagnosis of melanoma, this gene expression assay would likely reduce costs for health plans that choose to cover it. 相似文献
In patients with significant mitral regurgitation (MR) at high risk of mortality and morbidity from mitral valve surgery, transcatheter mitral valve repair with the MitraClip System is associated with a reduction in MR and improved quality-of-life and functional status compared with baseline. The objective was to evaluate the cost-effectiveness of MitraClip therapy compared with standard of care in patients with significant MR at high risk for mitral valve surgery from a Canadian payer perspective.
Methods:
A decision analytic model was developed to estimate the lifetime costs, life years, quality-adjusted life years (QALYs), and incremental cost per life year and QALY gained for patients receiving MitraClip therapy compared with standard of care. Treatment-specific overall survival, risk of clinical events, quality-of-life, and resource utilization were obtained from the Endovascular Valve Edge-to-Edge REpair High Risk Study (EVEREST II HRS). Health utility and unit costs (CAD $2013) were taken from the published literature. Sensitivity analyses were conducted to explore the impact of alternative assumptions and parameter uncertainty on results.
Results:
The base case incremental cost per QALY gained was $23,433. Results were most sensitive to alternative assumptions regarding overall survival, time horizon, and risk of hospitalization for congestive heart failure (CHF). Probabilistic sensitivity analysis showed MitraClip therapy to have a 92% chance of being cost-effective compared with standard of care at a willingness-to-pay threshold of $50,000 per QALY gained.
Study limitations:
Key limitations include the small number of patients included in the EVEREST II HRS which informed the analysis, the limited data available to inform clinical events and disease progression in the concurrent comparator group, and the lack of a comparator group from a randomized control trial.
Conclusion:
MitraClip therapy is likely a cost-effective option for the treatment of patients at high risk for mitral valve surgery with significant MR. 相似文献
To identify cost estimates related to myocardial infarction (MI) or stroke in patients with type 2 diabetes mellitus (T2DM) for use in economic models.
Methods:
A systematic literature review was conducted. Electronic databases and conference abstracts were screened against inclusion criteria, which included studies performed in patients who had T2DM before experiencing an MI or stroke. Primary cost studies and economic models were included. Costs were converted to 2012 pounds sterling.
Results:
Fifty-four studies were identified: 13 primary cost studies and 41 economic evaluations using secondary sources for complication costs. Primary studies provided costs from 10 countries. Estimates for a fatal event ranged from £2482–£5222 for MI and from £4900–£6694 for stroke. Costs for the year a non-fatal event occurred ranged from £5071–£29,249 for MI and from £5171–£38,732 for stroke. Annual follow-up costs ranged from £945–£1616 for an MI and from £4704–£12,926 for a stroke. Economic evaluations from 12 countries were identified, and costs of complications showed similar variability to the primary studies.
Discussion:
The costs identified within primary studies varied between and within countries. Many studies used costs estimated in studies not specific to patients with T2DM. Data gaps included a detailed breakdown of resource use, which affected the ability to compare data across countries.
Conclusions:
In the development of economic models for patients with T2DM, the use of accurate estimates of costs associated with MI and stroke is important. When country-specific costs are not available, clear justification for the choice of estimates should be provided. 相似文献
The goal of this study is to determine the cost-effectiveness of MIRISK VP, a next generation coronary heart disease risk assessment score, in correctly reclassifying and appropriately treating asymptomatic, intermediate risk patients.
Study design:
A Markov model was employed with simulated subjects based on the Multi-Ethnic Study of Atherosclerosis (MESA). This study evaluated three treatment strategies: (i) practice at MESA enrollment, (ii) current guidelines, and (iii) MIRISK VP in MESA.
Methods:
The model assessed patient healthcare costs and outcomes, expressed in terms of life years and quality-adjusted life years (QALYs), over the lifetime of the cohort from the provider and payer perspective. A total of 50,000 hypothetical individuals were used in the model. A sensitivity analysis was conducted (based on the various input parameters) for the entire cohort and also for individuals aged 65 and older.
Results:
Guiding treatment with MIRISK VP leads to the highest net monetary benefits when compared to the ‘Practice at MESA Enrollment’ or to the ‘Current Guidelines’ strategies. MIRISK VP resulted in a lower mortality rate from any CHD event and a modest increase in QALY of 0.12–0.17 years compared to the other two approaches.
Limitations:
This study has limitations of not comparing performance against strategies other than the FRS, the results are simulated as with all models, the model does not incorporate indirect healthcare costs, and the impact of patient or physician behaviors on outcomes were not taken into account.
Conclusions:
MIRISK VP has the potential to improve patient outcomes compared to the alternative strategies. It is marginally more costly than both the ‘Practice at MESA Enrollment’ and the ‘Current Guidelines’ strategies, but it provides increased effectiveness, which leads to positive net monetary benefits over either strategy. 相似文献
To estimate the clinical and economic trade-offs involved in using a molecular assay (92-gene assay, CancerTYPE ID) to aid in identifying the primary site of difficult-to-diagnose metastatic cancers and to explore whether the 92-gene assay can be used to standardize the diagnostic process and costs for clinicians, patients, and payers.
Methods:
Four decision-analytic models were developed to project the lifetime clinical and economic impact of incorporating the 92-gene assay compared with standard care alone. For each model, total and incremental costs, life-years, quality-adjusted life-years (QALYs), incremental cost–effectiveness ratios (ICERs), and the proportion of patients treated correctly versus incorrectly were projected from the payer perspective. Model inputs were based on published literature, analyses of SEER (Surveillance Epidemiology and End Results) data, publicly available data, and interviews with clinical experts.
Results:
In all four models, the 92-gene assay increased the proportion of patients treated correctly, decreased the proportion of patients treated with empiric therapy, and increased quality-adjusted survival. In the primary model, the ICER was $50,273/QALY; thus, the 92-gene assay is therefore cost effective when considering a societal willingness-to-pay threshold of $100,000/QALY. These findings were robust across sensitivity analyses.
Conclusions:
Use of the 92-gene assay for diagnosing metastatic tumors of uncertain origin is associated with reduced misdiagnoses, increased survival, and improved quality of life. Incorporating the assay into current practice is a cost-effective approach to standardizing diagnostic methods while improving patient care. Limitations of this analysis are the lack of data availability and resulting modeling simplifications, although sensitivity analyses showed these to not be key drivers of results. 相似文献
To determine the cost-effectiveness of home-based point-of-care self-monitoring compared to clinic-based care for patients managed on long-term warfarin medication. Current evidence is inconsistent; results should reduce uncertainty and inform service delivery.
Methods:
A Markov model compared self-testing and self-management, using point-of-care devices to usual care in patients with atrial fibrillation and mechanical heart valves. The primary clinical end-points were stroke and mortality avoided; costs and utilities were associated with these events. The costs of warfarin monitoring were included in the model.
Results:
Over 10 years, self-monitoring saved £1187 per person compared to usual care. Patients who self-monitored had notably fewer strokes and deaths. The results were sensitive to life-years gained and cost of the device. If the NHS purchased the device, financial break-even was achieved at the end of the second year; if the patient bought the device the NHS saved money every year. If 10% of the current 950,000 patients switched to point-of-care devices for 10 years, the NHS could save over £112million.
Limitations:
Clinical studies had a relatively short duration and only data on composite end-points were reported.
Conclusions:
With training, self-testing and self-management are safe, reliable, and cost-effective for a sizable proportion of patients receiving long-term warfarin. Compared to clinic-based services, self-monitoring offers the NHS the potential to make cost savings and release bed-days by reducing the number of strokes experienced by these high-risk patients. 相似文献
Methods: A nested case-control sample (IBS-C and CIC cases) and matched controls (1:2) for each case group were selected from Olmsted County, MN, individuals responding to a community-based survey of gastrointestinal symptoms (2008) who received healthcare from a participating Rochester Epidemiology Project (REP) provider. Using REP healthcare utilization data, unadjusted and adjusted standardized costs were compared for the 2- and 10-year periods prior to the survey for 115 IBS-C patients and 230 controls and 365 CIC patients and 730 controls. Two time periods were chosen as these conditions are episodic, but long-term.
Results: Outpatient costs for IBS-C ($6,800) and CIC ($6,284) patients over a 2-year period prior to the survey were significantly higher than controls ($4,242 and $5,254, respectively) after adjusting for co-morbidities, age, and sex. IBS-C outpatient costs ($25,448) and emergency room costs ($6,892) were significantly higher than controls ($21,024 and $3,962, respectively) for the 10-year period prior. Unadjusted data analyses of cases compared to controls demonstrated significantly higher imaging costs for IBS-C cases and procedure costs for CIC cases over the 10-year period.
Limitations: Data were collected from a random community sample primarily receiving care from a limited number of providers in that area.
Conclusions: Patients with IBS-C and CIC had significantly higher outpatient costs for the 2-year period compared with controls. IBS-C patients also had higher ER costs than the general population. 相似文献
To evaluate the health system costs among patients with hemophilia A and B with and without inhibitors over 5 years.
Methods:
This was a retrospective, observational study utilizing medical and pharmacy electronic medical records and administrative encounters/claims data tracking US patients between 2006–2011. Patients with diagnosis codes for hemophilia A and B were identified. Patients with inhibitors were characterized by utilization of bypassing agents activated prothrombin complex concentrate or factor VIIa on two or more distinct dates. Severity was classified as mild, moderate, or severe based on laboratory tests of clotting factor.
Results:
There were 160 hemophilia A patients and 54 hemophilia B patients identified. From this group, seven were designated as patients with inhibitors (five with hemophilia A and two with hemophilia B). Hemophilia A patients without inhibitors reported 65 (41.9%) as being severe, 19 (12.3%) as moderate, and 71 (45.8%) as mild. Hemophilia B patients without inhibitors reported nine (17.3%) had severe, 13 (25.0%) had moderate, and 30 (57.7%) had mild hemophilia. All patients with inhibitors had been hospitalized in the previous 5 years compared to 64 (41.3%) with hemophilia A without inhibitors and 22 (42.3%) with hemophilia B without inhibitors. The median aggregate cost per year (including factor and health resource use) was $325,780 for patients with inhibitors compared to $98,334 for hemophilia A patients without inhibitors and $23,265 for hemophilia B patients without inhibitors.
Conclusions:
The results suggest that, while the frequency of inhibitors within the hemophilia cohort was low, there was a higher frequency of hospitalizations, and the associated median aggregate costs per year were 3-fold higher than those patients without inhibitors. In contrast, hemophilia B patients experience less severe disease and account for lower aggregate yearly costs compared to either patients with hemophilia A or patients with inhibitors. 相似文献
Economic evaluations are increasingly utilized to inform decisions in healthcare; however, decisions remain uncertain when they are not based on adequate evidence. Value of information (VOI) analysis has been proposed as a systematic approach to measure decision uncertainty and assess whether there is sufficient evidence to support new technologies.
Scope:
The objective of this paper is to review the principles and applications of VOI analysis in healthcare. Relevant databases were systematically searched to identify VOI articles. The findings from the selected articles were summarized and narratively presented.
Findings:
Various VOI methods have been developed and applied to inform decision-making, optimally designing research studies and setting research priorities. However, the application of this approach in healthcare remains limited due to technical and policy challenges.
Conclusion:
There is a need to create more awareness about VOI analysis, simplify its current methods, and align them with the needs of decision-making organizations. 相似文献
Invasive pneumococcal disease (IPD) and pneumococcal pneumonia cause substantial morbidity and mortality worldwide. This retrospective study was conducted to estimate the disease burden from pneumococcal disease in older adults in Taiwan from a health insurer’s perspective.
Methods:
Data for the years 2002–2009 from patients aged ≥50 years with insurance records indicating pneumococcal meningitis, pneumococcal bacteremia, or hospitalized or outpatient pneumonia were obtained from the National Health Insurance Research Database in Taiwan. Admission data for inpatients, visit data for outpatients, and associated costs were extracted from the database to estimate the incidence, case fatality rates, and direct and indirect costs of pneumococcal disease episodes. These data were applied to the estimated population of Taiwan in 2010 to provide an estimated disease burden for a single year from the payer perspective.
Results:
The average incidence per 100,000 person years was 2.4 for IPD, 278.8 for hospitalized pneumococcal pneumonia, and 1376.4 for outpatient pneumococcal pneumonia. The average case fatality rate was 12.3% for IPD and 10.0% for hospitalized pneumonia. Hospitalized pneumonia accounted for over 90% of direct medical costs. The incidence of hospitalized pneumococcal pneumonia per 100,000 person years was 84.4 for adults of 50–64 years, 313.1 for adults of 65–74 years, 820.3 for adults of 75–84 years, and 1650.9 for adults of 85+ year of age. In 2010, it was estimated there were over 113,000 episodes of pneumococcal disease, causing almost 2000 deaths, with direct medical costs of more than NT$3.4 billion annually.
Conclusions:
Pneumococcal disease is a significant cause of mortality and excess healthcare expense among the elderly in Taiwan. Disease burden in older adults increases with advancing age. 相似文献
The economic implications from the US Medicare perspective of adopting alternative treatment strategies for acute bacterial skin and skin structure infections (ABSSSIs) are substantial. The objective of this study is to describe a modeling framework that explores the impact of decisions related to both the location of care and switching to different antibiotics at discharge.
Methods:
A discrete event simulation (DES) was developed to model the treatment pathway of each patient through various locations (emergency department [ED], inpatient, and outpatient) and the treatments prescribed (empiric antibiotic, switching to a different antibiotic at discharge, or a second antibiotic). Costs are reported in 2012 USD.
Results:
The mean number of days on antibiotic in a cohort assigned to a full course of vancomycin was 11.2 days, with 64% of the treatment course being administered in the outpatient setting. Mean total costs per patient were $8671, with inpatient care accounting for 58% of the costs accrued. The majority of outpatient costs were associated with parenteral administration rather than drug acquisition or monitoring. Scenarios modifying the treatment pathway to increase the proportion of patients receiving the first dose in the ED, and then managing them in the outpatient setting or prescribing an oral antibiotic at discharge to avoid the cost associated with administering parenteral therapy, therefore have a major impact and lower the typical cost per patient by 11–20%. Since vancomycin is commonly used as empiric therapy in clinical practice, based on these analyses, a shift in treatment practice could result in substantial savings from the Medicare perspective.
Conclusions:
The choice of antibiotic and location of care influence the costs and resource use associated with the management of ABSSSIs. The DES framework presented here can provide insight into the potential economic implications of decisions that modify the treatment pathway. 相似文献
Myelofibrosis is a non-frequent chronic myeloproliferative Philadelphia-negative chromosome neoplasm. It is a heavy incapacitating orphan disease and associated with high morbidity and mortality. In this context, indirect and non-medical costs are expected to be high. The main objective of this project is to estimate the economic burden of this disease in Spain.
Methods:
Thirty-three patients with a diagnosis of myelofibrosis for at least 1 year participated in a questionnaire in three Spanish centers. The study consisted of analyzing in various aspects the cost and impact of the disease; indeed, daily life time limitations with a need of informal care, symtomatology. Additionally, information concerning the clinical management of the disease was collected through a focus group of eight experts.
Results:
The mean age was 65 years. 15 of 33 patients were at their productive stage. Six had difficulties at work and eight have received informal care. Bone and muscular pain were the main symptoms of patients (72%). The estimated global indirect and non-medical costs of the disease were 86,315€ per patient (20% working and 80% informal care), which reached 104,153€ at productive stage patients (45%) and 168,459€ for more symptomatic patients.
Conclusions:
The economic burden of indirect and non-medical costs of myelofibrosis are important (15,142€/annual) as a result, and should be considered in economic evaluation, as well as in preventive plans for patients and caregivers, despite the fact that studies with larger numbers of patients should be done. 相似文献
Patients with persistent or longstanding atrial fibrillation have modest success achieving sinus rhythm with catheter ablation or rhythm control medications. Their high risk of stroke, bleed, and heart failure leads to significant morbidity and health care costs. The convergent procedure has been shown to be successful in this population, with 80% of patients in sinus rhythm after 1 year. This study evaluated the cost-effectiveness of the convergent procedure, catheter ablation, and medical management for non-paroxysmal AF patients.
Methods:
A Markov micro-simulation model was used to estimate costs and effectiveness from a payer perspective. Parameter estimates were from the literature. Three patient cohorts were simulated, representing lower, medium, and higher risks of stroke, bleed, heart failure, and hospitalization. Effects were estimated by quality-adjusted life-years (QALYs). Single-variable sensitivity analysis was performed.
Results:
After 5 years, convergent procedure patients averaged 1.10 procedures, with 75% of survivors in sinus rhythm; catheter ablation patients had 1.65 procedures, with 49% in sinus rhythm. Compared to medical management, catheter ablation and the convergent procedure were cost-effective for the lower risk (ICER <$35,000) and medium risk (ICER <$15,000) cohorts. The procedures dominated medical management for the higher risk cohort (lower cost and higher QALYs). The convergent procedure dominated catheter ablation for all risk cohorts. Results were subject to simplifying assumptions and limited by uncertain factors such as long-term maintenance of sinus rhythm after successful procedure and incremental AF-associated event rates for AF patients relative to patients in sinus rhythm. In the absence of clinical trial data, convergent procedure efficacy was estimated with observational evidence. Limitations were addressed with sensitivity analyses and a moderate 5 year time horizon.
Conclusion:
The convergent procedure results in superior maintenance of post-ablation sinus rhythm with fewer repeat ablation procedures compared to catheter ablation, leading to lower cost and higher QALYs after 5 years. 相似文献
To evaluate the cost-effectiveness of vildagliptin plus metformin vs generic sulphonylurea plus metformin in patients with type 2 diabetes mellitus, not controlled with metformin, from a Portuguese healthcare system perspective.
Methods:
A cost-effectiveness model was constructed using risk equations from the UK Prospective Diabetes Study Outcomes Model with a 10,000-patient cohort and a lifetime horizon. The model predicted microvascular and macrovascular complications and mortality in yearly cycles. Patients entered the model as metformin monotherapy failures and switched to alternative treatments (metformin plus basal-bolus insulin and subsequently metformin plus intensive insulin) when glycated hemoglobin A1c >7.5% was reached. Baseline patient characteristics and clinical variables were derived from a Portuguese epidemiological study. Cost estimates were based on direct medical costs only. One-way and probabilistic sensitivity analyses were conducted to test the robustness of the model.
Results:
There were fewer non-fatal diabetes-related adverse events (AEs) in patients treated with metformin plus vildagliptin compared with patients treated with metformin plus sulphonylurea (6752 vs 6815). Addition of vildagliptin compared with sulphonylurea led to increased drug acquisition costs but reduced costs of AEs, managing morbidities, and monitoring patients. Treatment with metformin plus vildagliptin yielded a mean per-patient gain of 0.1279 quality-adjusted life years (QALYs) and a mean per-patient increase in total cost of €1161, giving an incremental cost-effectiveness ratio (ICER) of €9072 per QALY. Univariate analyses showed that ICER values were robust and ranged from €4195 to €16,052 per QALY when different parameters were varied.
Limitations:
The model excluded several diabetes-related morbidities, such as peripheral neuropathy and ulceration, and did not model second events. Patients were presumed to enter the model with no diabetes-related complications.
Conclusion:
Treatment with metformin plus vildagliptin compared with metformin plus sulphonylurea is expected to result in a lower incidence of diabetes-related AEs and to be a cost-effective treatment strategy. 相似文献
To provide evidence on recent trends in: (1) market exclusivity periods (MEPs, the time between launch of a brand-name drug and its first generic competitor) for new molecular entities (NMEs); (2) the likelihood and timing of patent challenges under Paragraph IV of the Hatch-Waxman Act; and (3) generic drug penetration.
Methods:
IMS Health National Sales Perspectives data were used to calculate MEPs for the 257 NMEs experiencing initial generic entry between January 1995 and September 2012 and the number of generic competitors for 12 months afterwards, by level of annual sales prior to generic entry and time period. The likelihood and timing of Paragraph IV challenge were calculated using data from Abbreviated New Drug Approval (ANDA) approval letters, the FDA website, and public information searches to identify drugs experiencing Paragraph IV filings, and the first filing date.
Results:
For drugs experiencing initial generic entry in 2011–2012, the MEP was 12.6 years for drugs with sales greater than $100 million (in 2008 dollars) in the year prior to generic entry, 12.9 years overall. After generic entry, the brand rapidly lost sales, with average brand unit share of 16% at 1 year; 11% for NMEs with pre-generic entry sales of at least $250 million (in 2008 dollars). Over 80% of NMEs experiencing 2011–2012 initial generic entry had faced at least one Paragraph IV challenge from a generic manufacturer. These challenges were filed relatively early in the brand-name drug life cycle: within 7 years after brand launch, on average.
Limitations:
Analyses, including Paragraph IV calculations, were restricted to NMEs where generic entry had occurred.
Conclusion:
Pharmaceutical competition continues to evolve; while the average MEP below 13 years for 2011–2012 remains consistent with prior research, Paragraph IV challenges are increasingly frequent and occur earlier, and generic share erosion has intensified. 相似文献