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1.

Background

Considerable interest exists among health care payers and pharmaceutical manufacturers in designing outcomes-based agreements (OBAs) for medications for which evidence on real-world effectiveness is limited at product launch.

Objectives

To build hypothetical OBA models in which both payer and manufacturer can benefit.

Methods

Models were developed for a hypothetical hypercholesterolemia OBA, in which the OBA was assumed to increase market access for a newly marketed medication. Fixed inputs were drug and outcome event costs from the literature over a 1-year OBA period. Model estimates were developed using a range of inputs for medication effectiveness, medical cost offsets, and the treated population size. Positive or negative feedback to the manufacturer was incorporated on the basis of expectations of drug performance through changes in the reimbursement level. Model simulations demonstrated that parameters had the greatest impact on payer cost and manufacturer reimbursement.

Results

Models suggested that changes in the size of the population treated and drug effectiveness had the largest influence on reimbursement and costs. Despite sharing risk for potential product underperformance, manufacturer reimbursement increased relative to having no OBA, if the OBA improved market access for the new product. Although reduction in medical costs did not fully offset the cost of the medication, the payer could still save on net costs per patient relative to having no OBA by tying reimbursement to drug effectiveness.

Conclusions

Pharmaceutical manufacturers and health care payers have demonstrated interest in OBAs, and under a certain set of assumptions both may benefit.  相似文献   

2.

Objective

The aim of this study was to determine the cost of chronic wound care for Medicare beneficiaries in aggregate, by wound type and by setting.

Methods

This retrospective analysis of the Medicare 5% Limited Data Set for calendar year 2014 included beneficiaries who experienced episodes of care for one or more of the following: arterial ulcers, chronic ulcers, diabetic foot ulcers, diabetic infections, pressure ulcers, skin disorders, skin infections, surgical wounds, surgical infections, traumatic wounds, venous ulcers, or venous infections. The main outcomes were the prevalence of each wound type, Medicare expenditure for each wound type and aggregate, and expenditure by type of service.

Results

Nearly 15% of Medicare beneficiaries (8.2 million) had at least one type of wound or infection (not pneumonia). Surgical infections were the largest prevalence category (4.0%), followed by diabetic infections (3.4%). Total Medicare spending estimates for all wound types ranged from $28.1 to $96.8 billion. Including infection costs, the most expensive estimates were for surgical wounds ($11.7, $13.1, and $38.3 billion), followed by diabetic foot ulcers ($6.2, $6.9, and $18.7 billion,). The highest cost estimates in regard to site of service were for hospital outpatients ($9.9–$35.8 billion), followed by hospital inpatients ($5.0–$24.3 billion).

Conclusions

Medicare expenditures related to wound care are far greater than previously recognized, with care occurring largely in outpatient settings. The data could be used to develop more appropriate quality measures and reimbursement models, which are needed for better health outcomes and smarter spending for this growing population.  相似文献   

3.
4.

Background

Conditional reimbursement of new health technologies is increasingly considered as a useful policy instrument. It allows gathering more robust evidence regarding effectiveness and cost-effectiveness of new technologies without delaying market access. Nevertheless, the literature suggests that ending reimbursement and provision of a technology when it proves not to be effective or cost-effective in practice may be difficult.

Objectives

To investigate how policymakers and the general public in the Netherlands value removing a previously reimbursed treatment from the basic benefits package relative to not including a new treatment.

Methods

To investigate this issue, we used discrete-choice experiments. Mixed multinomial logit models were used to analyze the data. Compensating variation values and changes in probability of acceptance were calculated for withdrawal of reimbursement.

Results

The results show that, ceteris paribus, both the general public (n = 1169) and policymakers (n = 90) prefer a treatment that is presently reimbursed over one that is presently not yet reimbursed.

Conclusions

Apparently, ending reimbursement is more difficult than not starting reimbursement in the first place, both for policymakers and for the public. Loss aversion is one of the possible explanations for this result. Policymakers in health care need to be aware of this effect before engaging in conditional reimbursement schemes.  相似文献   

5.

Objectives

The objective of this study was to explore the trade-offs society and payers make when expanding treatment access to patients with chronic hepatitis C virus (HCV) infection in early stages of disease as well as to vulnerable, high-risk populations, such as people who inject drugs (PWID) and HIV-infected men who have sex with men (MSM-HIV).

Methods

A discrete time Markov model simulated HCV progression and treatment over 20 years. Population cohorts were defined by behaviors that influence the risk of HCV exposure: PWID, MSM-HIV, an overlap cohort of individuals who are both PWID and MSM-HIV, and all other adults. Six different treatment scenarios were modeled, with varying degrees of access to treatment at different fibrosis stages and to different risk cohorts. Benefits were measured as quality-adjusted life-years and a $150,000/quality-adjusted life-year valuation was used to assess social benefits.

Results

Compared with limiting treatment to METAVIR fibrosis stages F3 or F4 and excluding PWID, expanding treatment to patients in all fibrosis stages and including PWID reduces cumulative new infections by 55% over a 20-year horizon and reduces the prevalence of HCV by 93%. We find that treating all HCV-infected individuals is cost saving and net social benefits are over $500 billion greater compared with limiting treatment. Including PWID in treatment access saves 12,900 to 41,200 lives.

Conclusions

Increased access to treatment brings substantial value to society and over the long-term reduces costs for payers, as the benefits accrued from long-term reduction in prevalent and incident cases, mortality, and medical costs outweigh the cost of treatment.  相似文献   

6.

Objectives

To estimate the impact of increased glycated hemoglobin (A1C) monitoring and treatment intensification for patients with type 2 diabetes (T2D) on quality measures and reimbursement within the Medicare Advantage Star (MA Star) program.

Methods

The primary endpoint was the share of patients with T2D with adequate A1C control (A1C ≤ 9%). We conducted a simulation of how increased A1C monitoring and treatment intensification affected this end point using data from the National Health and Nutrition Examination Survey and clinical trials. Using the estimated changes in measured A1C levels, we calculated corresponding changes in the plan-level A1C quality measure, overall star rating, and reimbursement.

Results

At baseline, 24.4% of patients with T2D in the average plan had poor A1C control. The share of plans receiving the highest A1C rating increased from 27% at baseline to 49.5% (increased monitoring), 36.2% (intensification), and 57.1% (joint implementation of both interventions). However, overall star ratings increased for only 3.6%, 1.3%, and 4.8% of plans, respectively, by intervention. Projected per-member per-year rebate increases under the MA Star program were $7.71 (monitoring), $2.66 (intensification), and $10.55 (joint implementation).

Conclusions

The simulation showed that increased monitoring and treatment intensification would improve A1C levels; however, the resulting average increases in reimbursement would be small.  相似文献   

7.

Objectives

To determine whether implementation of interferon-free treatment for hepatitis C virus (HCV) reached groups less likely to benefit from earlier therapies, including patients with genotype 1 virus or contraindications to interferon treatment, and groups that faced treatment disparities: African Americans, patients with HIV co-infection, and those with drug use disorder.

Methods

Electronic medical records of the US Veterans Health Administration (VHA) were used to characterize patients with chronic HCV infection and the treatments they received. Initiation of treatment in 206,544 patients with chronic HCV characterized by viral genotype, demographic characteristics, and comorbid medical and mental illness was studied using a competing events Cox regression over 6 years.

Results

With the advent of interferon-free regimens, the proportion treated increased from 2.4% in 2010 to 18.1% in 2015, an absolute increase of 15.7%. Patients with genotype 1 virus, poor response to previous treatment, and liver disease had the greatest increase. Large absolute increases in the proportion treated were observed in patients with HIV co-infection (18.6%), alcohol use disorder (11.9%), and drug use disorder (12.6%) and in African American (13.7%) and Hispanic (13.5%) patients, groups that were less likely to receive interferon-containing treatment. The VHA spent $962 million on interferon-free treatments in 2015, 1.5% of its operating budget.

Conclusions

The proportion of patients with HCV treated in VHA increased sevenfold. The VHA was successful in implementing interferon treatment in previously undertreated populations, and this may become the community standard of care.  相似文献   

8.

Background

Concerns about pathology testing such as the value provided by new tests and the potential for inappropriate utilization have led to a greater need to assess costs and benefits. Economic evaluations are a formal method of analyzing costs and benefits, yet for pathology tests, questions remain about the scope and quality of the economic evidence.

Objective

To describe the extent and quality of published evidence provided by economic evaluations of pathology tests from 2010 to 2015.

Methods

Economic evaluations relating to pathology tests from 2010 to 2015 were reviewed. Eight databases were searched for published studies, and details recorded for the country, clinical focus, type of testing, and consideration of sensitivity, specificity, and false test results. The reporting quality of studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist and cost-effectiveness ratios were analyzed for publication bias.

Results

We found 356 economic evaluations of pathology tests, most of which regarded developed countries. The most common economic evaluations were cost-utility analyses and the most common clinical focus was infectious diseases. More than half of the studies considered sensitivity and specificity, but few studies considered the impact of false test results. The average Consolidated Health Economic Evaluation Reporting Standards checklist score was 17 out of 24. Cost-utility ratios were commonly less than $10,000/quality-adjusted life-year or more than $200,000/quality-adjusted life-year.

Conclusions

The number of economic evaluations of pathology tests has increased in recent years, but the rate of increase has plateaued. Furthermore, the quality of studies in the past 5 years was highly variable, and there is some question of publication bias in reporting cost-effectiveness ratios.  相似文献   

9.

Background

Phosphate binders are used to treat hyperphosphatemia among patients with chronic kidney disease (CKD).

Objectives

To conduct an economic evaluation comparing calcium-free binders sevelamer and lanthanum with calcium-based binders for patients with CKD.

Methods

Effectiveness data were obtained from a recent meta-analysis of randomized trials. Effectiveness was measured as life-years gained and translated to quality-adjusted life-years (QALYs) using utility weights from the literature. A Markov model consisting of non–dialysis-dependent (NDD)-CKD, dialysis-dependent (DD)-CKD, and death was developed to estimate the incremental costs and effects of sevelamer and lanthanum versus those of calcium-based binders. A lifetime horizon was used and both costs and effects were discounted at 1.5%. All costs are presented in 2015 Canadian dollars from the Canadian public payer perspective. Results of probabilistic sensitivity analysis were presented using cost-effectiveness acceptability curves. Sensitivity analyses were conducted for risk pooling methods, omission of dialysis costs, and persistence of drug effects on mortality.

Results

Sevelamer resulted in an incremental cost-effectiveness ratio of $106,522/QALY for NDD-CKD and $133,847/QALY for DD-CKD cohorts. Excluding dialysis costs, sevelamer was cost-effective in the NDD-CKD cohort ($5,847/QALY) and the DD-CKD cohort ($11,178/QALY). Lanthanum was dominated regardless of whether dialysis costs were included.

Conclusions

Existing evidence does not clearly support the cost-effectiveness of non–calcium-containing phosphate binders (sevelamer and lanthanum) relative to calcium-containing phosphate binders in DD-CKD patients. Our study suggests that sevelamer may be cost-effective before dialysis onset. Because of the remaining uncertainty in several clinically relevant outcomes over time in DD-CKD and NDD-CKD patients, further research is encouraged.  相似文献   

10.

Background

A recent cluster randomized trial evaluating a multicomponent intervention showed significant reductions in blood pressure in low-income hypertensive subjects in Argentina.

Objectives

To assess the cost-effectiveness of this intervention.

Methods

A total of 1432 hypertensive participants were recruited from 18 primary health care centers. The intervention included home visits led by community health workers, physician education, and text messaging. Resource use and quality of life data using the three-level EuroQol five-dimensional questionnaire were prospectively collected. The study perspective was that of the public health care system, and the time horizon was 18 months. Intention-to-treat analysis was used to analyze cost and health outcomes (systolic blood pressure [SBP] change and quality-adjusted life-years [QALYs]). A 1 time gross domestic product per capita per QALY was used as the cost-effectiveness threshold (US $14,062).

Results

Baseline characteristics were similar in the two arms. QALYs significantly increased by 0.06 (95% confidence interval [CI] 0.04–0.09) in the intervention group, and SBP net difference favored the intervention group: 5.3 mm Hg (95% CI 0.27–10.34). Mean total costs per participant were higher in the intervention arm: US $304 in the intervention group and US $154 in the control group (adjusted difference of US $140.18; 95% CI US $75.41–US $204.94). The incremental cost-effectiveness ratio was $3299 per QALY (95% credible interval 1635–6099) and US $26 per mm Hg of SBP (95% credible interval 13–46). Subgroup analysis showed that the intervention was cost-effective in all prespecified subgroups (age, sex, cardiovascular risk, and body mass index).

Conclusions

The multicomponent intervention was cost-effective for blood pressure control among low-income hypertensive patients.  相似文献   

11.

Background

The German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen) adapted the efficiency frontier (EF) approach to conform to statutory provisions on cost-effectiveness analysis of health technologies. EF serves as a framework for evaluating cost-effectiveness and indirectly for pricing and reimbursement decisions.

Objectives

To calculate an EF on the basis of single multidimensional benefit by taking patient preferences and uncertainty into account; to evaluate whether EF is useful to inform decision makers about cost-effectiveness of new therapies; and to find whether a treatment is efficient at given prices demonstrated through a case study on chronic hepatitis C.

Methods

A single multidimensional benefit was calculated by linear additive aggregation of multiple patient-relevant end points. End points were identified and weighted by patients in a previous discrete-choice experiment (DCE). Aggregation of overall benefit was ascertained using preferences and clinical data. Monte-Carlo simulation was applied. Uncertainty was addressed by price acceptability curve (PAC) and net monetary benefit (NMB).

Results

The case study illustrates that progress in benefit and efficiency of hepatitis C virus treatments could be depicted very well with the EF. On the basis of cost, effect, and preference data, the latest generations of interferon-free treatments are shown to yield a positive NMB and be efficient at current prices.

Conclusions

EF was implemented taking uncertainty into account. For the first time, a DCE was used with the EF. The study shows how DCEs in combination with EF, PAC, and NMB can contribute important information in the course of reimbursement and pricing decisions.  相似文献   

12.

Background

It has been suggested that differences in health technology assessment (HTA) processes among countries, particularly within Europe, have led to inequity in patient access to new medicines.

Objectives

To provide an up-to-date snapshot analysis of the present status of HTA and reimbursement systems in select European countries, and to investigate the implications of these processes, especially with regard to delays in market and patient access.

Methods

HTA and reimbursement processes were assessed through a review of published and gray literature, and through a series of interviews with HTA experts. To quantify the impact of differences among countries, we conducted case studies of 12 products introduced since 2009, including 10 cancer drugs.

Results

In addition to the differences in HTA and reimbursement processes among countries, the influence of particular sources of information differs among HTA bodies. The variation in the time from the authorization by the European Medicines Agency to the publication of HTA decisions was considerable, both within and among countries, with a general lack of transparency as to why some assessments take longer than others. In most countries, market access for oncology products can occur outside the HTA process, with sales often preceding HTA decisions.

Conclusions

It is challenging even for those with considerable personal experience in European HTA processes to establish what is really happening in market access for new drugs. We recommend that efforts should be directed toward improving transparency in HTA, which should, in turn, lead to more effective processes.  相似文献   

13.

Background

When proven effective, decision making regarding reimbursement of new health technology typically involves ethical, social, legal, and health economic aspects and constraints. Nevertheless, when applying standard value of information (VOI) analysis, the value of collecting additional evidence is typically estimated assuming that only cost-effectiveness outcomes guide such decisions.

Objectives

To illustrate how decision makers’ constraints can be incorporated into VOI analyses and how these may influence VOI outcomes.

Methods

A simulation study was performed to estimate the cost-effectiveness of a new hypothetical technology compared with usual care. Constraints were defined for the new technology on 1) the maximum acceptable rate of complications and 2) the maximum acceptable additional budget. The expected value of perfect information (EVPI) for the new technology was estimated in various scenarios, both with and without incorporating these constraints.

Results

For a willingness-to-pay threshold of €20,000 per quality-adjusted life-year, the probability that the new technology was cost-effective equaled 57%, with an EVPI of €1868 per patient. Applying the complication rate constraint reduced the EVPI to €1137. Similarly, the EVPI reduced to €770 when applying the budget constraint. Applying both constraints simultaneously further reduced the EVPI to €318.

Conclusions

When decision makers explicitly apply additional constraints, beyond a willingness-to-pay threshold, to reimbursement decisions, these constraints can and should be incorporated into VOI analysis as well, because they may influence VOI outcomes. This requires continuous interaction between VOI analysts and decision makers and is expected to improve both the relevance and the acceptance of VOI outcomes.  相似文献   

14.

Background

Childhood and adolescent cancers are uncommon, but they have important economic and health impacts on patients, families, and health care systems. Few studies have measured the economic burden of care for childhood and adolescent cancers.

Objectives

To estimate costs of cancer care in population-based cohorts of children and adolescents from the public payer perspective.

Methods

We identified patients with cancer, aged 91 days to 19 years, diagnosed from 1995 to 2009 using cancer registry data, and matched each to three noncancer controls. Using linked administrative health care records, we estimated total and net resource-specific costs (in 2012 Canadian dollars) during 90 days prediagnosis and 1 year postdiagnosis.

Results

Children (≤14 years old) numbered 4,396: 36% had leukemia, 21% central nervous system tumors, 10% lymphoma, and 33% other cancers. Adolescents (15–19 years old) numbered 2,329: 28.9% had lymphoma. Bone and soft tissue sarcoma, germ cell tumor, and thyroid carcinoma each comprised 12% to 13%. Mean net prediagnosis costs were $5,810 and $1,127 and mean net postdiagnosis costs were $136,413 and $62,326 for children and adolescents, respectively; the highest were for leukemia ($157,764 for children and $172,034 for adolescents). In both cohorts, costs were much higher for patients who died within 1 year of diagnosis. Inpatient hospitalization represented 69% to 74% of postdiagnosis costs.

Conclusions

Treating children with cancer is costly, more costly than treating adolescents or adults. Substantial survival gains in children mean that treatment may still be very cost-effective. Comprehensive age-specific population-based cost estimates are essential to reliably assess the cost-effectiveness of cancer care for children and adolescents, and measure health system performance.  相似文献   

15.

Background

Although evidence suggests significant clinical benefits of home noninvasive ventilation (NIV) for management of severe chronic obstructive pulmonary disease (COPD), economic analyses supporting the use of this technology are lacking.

Objectives

To evaluate the economic impact of adopting home NIV, as part of a multifaceted intervention program, for severe COPD.

Methods

An economic model was developed to calculate savings associated with the use of Advanced NIV (averaged volume assured pressure support with autoexpiratory positive airway pressure; Trilogy100, Philips Respironics, Inc., Murrysville, PA) versus either no NIV or a respiratory assist device with bilevel pressure capacity in patients with severe COPD from two distinct perspectives: the hospital and the payer. The model examined hospital savings over 90 days and payer savings over 3 years. The number of patients with severe COPD eligible for home Advanced NIV was user-defined. Clinical and cost data were obtained from a quality improvement program and published reports. Scenario analyses calculated savings for hospitals and payers covering different COPD patient cohort sizes.

Results

The hospital base case (250 patients) revealed cumulative savings of $402,981 and $449,101 over 30 and 90 days, respectively, for Advanced NIV versus both comparators. For the payer base case (100,000 patients), 3-year cumulative savings with Advanced NIV were $326 million versus no NIV and $1.04 billion versus respiratory assist device.

Conclusions

This model concluded that adoption of home Advanced NIV with averaged volume assured pressure support with autoexpiratory positive airway pressure, as part of a multifaceted intervention program, presents an opportunity for hospitals to reduce COPD readmission-related costs and for payers to reduce costs associated with managing patients with severe COPD on the basis of reduced admissions.  相似文献   

16.

Background

Several public cord blood banks are struggling financially, and the question remains as to whether additional allocations of funds to them are justified.

Objectives

To estimate the social benefits of public cord blood bank inventory net of cord blood banks’ operational costs.

Methods

We used publicly available data from the Health Resources and Service Administration on the number of annual cord blood transplants as well as the patient age distribution in 2010, and the survival estimates between 2008 and 2012 for the several diseases treated by cord blood transplantation. Data on aggregate annual costs to the cord blood industry for recruitment, processing, and storage were obtained from published work. We used estimated increases in life expectancy due to treatment using umbilical cord blood and value for life-years gained to estimate the social benefits of the public cord blood inventory annually.

Results

We found that the annual social benefits of between $500 million and $1.5 billion outweigh the current operational annual costs of running cord blood banks of $60 to $70 million by a significant margin.

Conclusions

We estimated that the annual social benefit of having a cord blood system far outweighs its costs, by more than an order of magnitude. Thus, the social benefits of maintaining the US public cord blood banking system at the present time far outweigh the costs of collecting, storing, and distributing cord blood. This suggests that there is a potential justification for government intervention to align social benefits and costs. Nevertheless, simple fixes may produce unintended consequences, and so a careful design for subsidies is needed.  相似文献   

17.

Background

The use of cost-effectiveness analysis for medical devices has proven to be challenging because of the existence of the learning effects in the device-operator interactions. The need for the relevant analytical framework for assessing the economic value of such technologies has been recognized.

Objectives

To present a modified difference-in-differences (DID) cost-effectiveness methodology that facilitates visualization of a new health technology’s learning curve.

Methods

Using the Premier Perspective database (Premier Inc., Charlotte, NC), we examined the impact of physicians adopting a bipolar sealer (BPS) to control blood loss in primary unilateral total knee arthroplasties on hospital lengths of stay and total hospitalization costs when compared with two control groups. In our DID approach, we substituted month-from-adoption for the calendar-month-of-adoption in both graphical representations and ordinary least-squares regression results to estimate the effect of the BPS.

Results

The results clearly demonstrated a learning curve associated with the adoption of the BPS technology. Although the reductions in length of stay were immediate, the first postadoption year costs increased by $1335 (extrahospital controls) to $1565 (within-hospital controls). Importantly, and also consistent with a learning curve hypothesis, these initial higher costs were offset by subsequent cost savings in the second and third years postadoption.

Conclusions

The presented modified DID approach is a suitable and versatile analytical tool for economic evaluation of a slowly diffusing medical device or health technology. It provides a better understanding of the potential learning effects associated with relevant interventions.  相似文献   

18.

Background

The time trade-off (TTO) technique is commonly used to elicit health state utilities. Nevertheless, when the health states being valued are temporary, the TTO approach may be unsuitable. A variant of TTO— chained TTO—has been suggested to be used when the health states are temporary, but little research has been done on how chained TTO should be conducted.

Objectives

To systematically review the use of chained TTO in valuing temporary health states.

Methods

A systematic literature search was conducted using the following major databases: Ovid MEDLINE(R), Embase, EBM Reviews, and PsycINFO. Abstracts (full articles if necessary) were screened by two independent reviewers, with a third reviewer resolving any disagreements.

Results

The resulting number of articles for review was low (n = 9). All the reviewed studies used face-to-face interviews, most had small sample sizes (<100), and all studies valued a small number of health states (<7), with time horizons typically ranging from 4 weeks to 1 year. All studies discussed methodological issues of using chained TTO, and some compared the results with those generated using other preference elicitation methods.

Conclusions

Chained TTO appears to be feasible, consistent, and responsive and allows the valuation of temporary health states that would improve the efficiency and accuracy of decision making in health and health care. Nevertheless, the evidence is limited due to the low number of relevant studies in the literature. Further research is needed to examine the performance and validity of chained TTO compared with conventional TTO in the valuation of temporary health states.  相似文献   

19.

Background

Economic evaluations often measure an intervention effect with mean overall survival (OS). Emerging types of cancer treatments offer the possibility of being “cured” in that patients can become long-term survivors whose risk of death is the same as that of a disease-free person. Describing cured and noncured patients with one shared mean value may provide a biased assessment of a therapy with a cured proportion.

Objective

The purpose of this article is to explain how to incorporate the heterogeneity from cured patients into health economic evaluation.

Methods

We analyzed clinical trial data from patients with advanced melanoma treated with ipilimumab (Ipi; n = 137) versus glycoprotein 100 (gp100; n = 136) with statistical methodology for mixture cure models. Both cured and noncured patients were subject to background mortality not related to cancer.

Results

When ignoring cured proportions, we found that patients treated with Ipi had an estimated mean OS that was 8 months longer than that of patients treated with gp100. Cure model analysis showed that the cured proportion drove this difference, with 21% cured on Ipi versus 6% cured on gp100. The mean OS among the noncured cohort patients was 10 and 9 months with Ipi and gp100, respectively. The mean OS among cured patients was 26 years on both arms. When ignoring cured proportions, we found that the incremental cost-effectiveness ratio (ICER) when comparing Ipi with gp100 was $324,000/quality-adjusted life-year (QALY) (95% confidence interval $254,000–$600,000). With a mixture cure model, the ICER when comparing Ipi with gp100 was $113,000/QALY (95% confidence interval $101,000–$154,000).

Conclusions

This analysis supports using cure modeling in health economic evaluation in advanced melanoma. When a proportion of patients may be long-term survivors, using cure models may reduce bias in OS estimates and provide more accurate estimates of health economic measures, including QALYs and ICERs.  相似文献   

20.

Objectives

Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient access to ultra-orphan drugs between countries. Furthermore, we describe how reimbursed and non-reimbursed orphan drugs differ with respect to pharmacoeconomic properties.

Methods

We studied patient access to eight high-priced inpatient ultra-orphan drugs in nine countries. In addition, we determined whether differences with respect to cost per patient, budget impact and cost-effectiveness existed between orphan drugs with a positive and negative reimbursement status.

Results

Reimbursement status was available for 78 orphan drugs, of which 56 (72%) were positive. Large differences were observed between countries; while two countries had a positive status for two out of nine ultra-orphan drugs, four countries had positive status for all drugs it assessed. A number of drugs were reimbursed only after price negotiations and/or through specific orphan drug policies. The average cost per patient, budget impact and incremental cost-effectiveness ratios were lower for ultra-orphan drugs with a positive reimbursement status than for those with a negative status, although only cost-effectiveness ratios were statistically significant.

Conclusions

Large differences in patient access to ultra-orphan drugs were observed between countries. Future research should examine if similar findings can be seen in other countries and with other orphan drugs, and it should also determine which other factors play a role in reimbursement status of orphan drugs.  相似文献   

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