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
Knee cartilage damage is a common cause of referral for orthopedic surgery. Treatment aims to reduce pain and symptoms by repairing cartilage. Microfracture, the current standard of care, yields good short-term clinical outcomes; however, treatment might fail after 2–3 years. A Chitosan-Beta glycerolphosphate-based medical device (BST-CarGel) is used as an adjunct to microfracture and demonstrates improvements in quantity and quality of repaired tissue, potentially reducing the risk of treatment failure. This study aimed to establish the economic value of BST-CarGel vs microfracture alone in knee cartilage repair from the societal perspective, using Germany as the reference market.
Methods:
A decision tree with a 20-year time-horizon was constructed, in which undesirable clinical events were inferred following initial surgery. These events consisted of pain management, surgery, and total knee replacement. Clinical outcomes were taken from the pivotal clinical trial, supplemented by other literature. Data and assumptions were validated by a Delphi panel. All relevant resource use and costs for procedures and events were considered.
Results:
In a group of patients with all lesion sizes, the model inferred that BST-CarGel yields a positive return on investment at year 4 (with 20-year cumulative cost savings of €6448). Reducing the incremental risk of treatment failure gap between the device and microfracture by 25–50% does not alter this conclusion. Cost savings are greatest for patients with large lesions; results for patients with small lesions are more modest.
Limitations:
Clinical evidence for microfracture and other interventions varies in quality. Comparative long-term data are lacking. The comparison is limited to microfracture and looks only at costs without considering quality-of-life.
Conclusion:
BST-CarGel potentially represents a cost-saving alternative for patients with knee cartilage injury by reducing the risk of clinical events through regeneration of chondral tissue with hyaline characteristics. Since the burden of this condition is high, both to the patient and society, an effective and economically viable alternative is of importance. 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
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
Telaprevir (TVR,T) and boceprevir (BOC,B) are direct-acting antivirals (DAAs) used for the treatment of chronic genotype 1 hepatitis C virus (HCV) infection. This analysis evaluated the cost-effectiveness of TVR combined with pegylated interferon (Peg-IFN) alfa-2a plus ribavirin (RBV) compared with Peg-IFN alfa-2a and RBV (PR) alone or BOC plus Peg-IFN alfa-2b and RBV in treatment-experienced patients.
Methods:
A Markov cohort model of chronic genotype 1 HCV disease progression reflected the pathway of experienced patients retreated with DAA therapy. The population was stratified by previous response to treatment (i.e., previous relapsers, partial responders, and null responders). Sustained virologic response (SVR) rates were derived from a mixed-treatment comparison that included results from separate Phase III trials of TVR and BOC. Incremental cost per life year (LY) gained and quality-adjusted-life-year (QALY) gained were computed at lifetime, adopting the NHS perspective. Costs and health outcomes were discounted at 3.5%. Uncertainty was assessed using deterministic and probabilistic sensitivity analyses. Sub-group analyses were carried out by interleukin (IL)-28B genotype.
Results:
Higher costs and improved outcomes were associated with T/PR relative to PR alone for all experienced patients (ICER of £6079). T/PR was cost-effective for each sub-group population with high SVR advantage in relapsers (ICER of £2658 vs £7593 and £20,875 for partial and null responders). T/PR remained cost-effective regardless of IL-28B sub-type. Compared to B/PR, T/PR prolonged QALYs by 0.57 and reduced lifetime costs by £13,960 for relapsers. For partial responders T/PR was less costly but less efficacious than B/PR, equating to an ICER of £128,117 per QALY gained.
Limitations:
No head-to-head trial provides direct evidence of better efficacy of T/PR vs B/PR.
Conclusion:
T/PR is cost-effective compared with PR alone in experienced patients regardless of treatment history and IL-28B genotype. Compared to B/PR, T/PR is always cost-saving but only more effective in relapsers. 相似文献
Most of the existing studies investigating the impact of schizophrenia on utility have focused on the different stages of the disease. The objective of this study was to describe and quantify the impact of treatment-related side-effects on utility in patients with schizophrenia, using data from an observational study.
Methods:
This study used data from the European Schizophrenia Cohort (EuroSC), a multi-center 2-year cohort study conducted in France, England, and Germany. The EQ-5D questionnaire was completed every 6 months, as well as the Subjective Side Effect Rating Scale, assessing patient distress over extrapyramidal symptoms (EPS), weight gain, sedation, and sexual dysfunction, used to assess whether or not the patient experienced the side-effect. At first a bivariate analysis was conducted to describe utility values with and without side-effects. Then, a random effects regression analysis was performed on utility, where random effects were controlled for repeated measures on the same subjects, with potential confounding factors. Finally, findings were compared with those of previous publications.
Results:
This sample consisted of 1208 patients with schizophrenia. At the baseline visit, the most reported side-effect was EPS (almost 60% of patients), followed by sedation and weight gain (～50% of patients for each), and sexual dysfunction (almost 30% of patients). Significant association with severity of symptoms, functioning abilities, and utility were found. Patients reporting none of the studied side-effects had an average EQ-5D index score of 0.81, found to be higher than scores of patients reporting EPS (0.70), sexual dysfunction (0.67), sedation (0.70), or weight gain (0.72). The random effects model reported a utility decrement of 0.042 for EPS, 0.022 for weight gain, 0.022 for sexual dysfunction, and 0.019 for sedation. Although the external validation was difficult due to the different methods or definitions of the side-effects, as well as the paucity of data for weight gain, sedation and sexual dysfunction, the results were generally consistent with previous studies.
Conclusion:
This study aimed at quantifying the direct impact of main side-effects associated with antipsychotics on patients’ utility. Results suggested a significant direct impact of side-effects, with EPS being the most impactful. 相似文献
This paper aims to provide new evidence on the occurrence of these effects in the Italian economy, where changes in labour legislation from the mid-Nineties onwards, associated with an ‘institutional’ wage moderation period, have brought about a significant process of job creation, but also an appreciable slowdown in labour productivity.
This issue is investigated using a microeconomic approach, taking a rich source of microdata for firms and estimating a dynamic model for labour productivity on a pseudo-panel of firms for the period 2003-2008.
The results support the hypothesis of a negative impact of external labour flexibility on labour productivity growth at firm level, such effect proving stronger for small and medium than for large enterprises and of varying magnitude for the different atypical contracts. 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
Materials and methods: An analysis of administrative claims data among patients diagnosed with non-small cell lung cancer from 2007–2015 was conducted. Future costs were projected through 2040 based on these data using autoregressive models.
Results: Analysis of claims data found the average total cost of care during first- and second-line therapy was $1,161.70 and $561.80 for patients, and $45,175.70 and $26,201.40 for insurers, respectively. By 2040, the average total patient out-of-pocket costs are projected to reach $3,047.67 for first-line and $2,211.33 for second-line therapy, and insurance will pay an average of $131,262.39 for first-line and $75,062.23 for second-line therapy.
Limitations: Claims data are not collected for research purposes; therefore, there may be errors in entry and coding. Additionally, claims data do not contain important clinical factors, such as stage of disease at diagnosis, tumor histology, or data on disease progression, which may have important implications on the cost of care.
Conclusions: The trajectory of the cost of lung cancer care is growing. This study estimates that the cost of care may double by 2040, with the greatest proportion of increase in patient out-of-pocket costs. Despite the average cost projections, these results suggest that a small sub-set of patients with very high costs could be at even greater risk in the future. 相似文献
Methods: Macro-indicators were obtained from the WHO Health for All Database. Inter-regional comparison among post-Semashko, former Yugoslavia, and prior-1989-free-market SEE economies was conducted.
Results: United Nations Development Program Human Development Index growth was strongest among prior-free-market SEE, followed by former Yugoslavia and post-Semashko. Policy cuts to hospital beds and nursing-staff capacities were highest in post-Semashko. Physician density increased the most in prior-free-market SEE. Length of hospital stay was reduced in most countries; frequency of outpatient visits and inpatient discharges doubled in prior-free-market SEE. Fertility rates fell for one third in Post-Semashko and prior-free-market SEE. Crude death rates slightly decreased in prior-free-market-SEE and post-Semashko, while growing in the former Yugoslavia region. Life expectancy increased by 4 years on average in all regions; prior-free-market SEE achieving the highest longevity. Childhood and maternal mortality rates decreased throughout SEE, while post-Semashko countries recorded the most progress.
Conclusions: Significant differences in healthcare resources and outcomes were observed among three historical health-policy legacies in south-eastern Europe. These different routes towards common goals created a golden opportunity for these economies to learn from each other. 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
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
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
To evaluate the cost-effectiveness of bendamustine-rituximab (B-R) compared with CHOP-R (cyclophosphamide, doxorubicin, vincristine, prednisone, rituximab) and CVP-R (cyclophosphamide, vincristine, prednisone, rituximab) as first-line treatment for patients with advanced indolent non-Hodgkin’s lymphoma (NHL).
Methods:
A patient-level simulation was adapted from the model used by the University of Sheffield School of Health and Related Research (ScHARR) in a health technology appraisal of rituximab for first-line treatment of follicular lymphoma. This approach allowed modelling of the complex treatment pathways in indolent NHL. Data from a Phase 3 randomized, open-label trial were used to compare B-R with CHOP-R. The relative efficacy of CHOP-R and CVP-R was estimated using an indirect treatment comparison similar to the original ScHARR approach. The analysis was conducted from the perspective of the National Health Service in England and Wales, using a lifetime time horizon. A number of one-way sensitivity and scenario analyses were conducted, including one using recently published data comparing CVP-R with CHOP-R.
Results:
The deterministic incremental cost-effectiveness ratio (ICER) was £5249 per quality adjusted life year (QALY) for B-R vs CHOP-R, and £8092 per QALY for B-R vs CVP-R. The alternative scenario using direct data comparing CVP-R with CHOP-R approximately halved the ICER for B-R vs CVP-R to £4733. Owing to its better toxicity profile, B-R reduced the cost of treating adverse events by over £1000 per patient vs CHOP-R.
Limitations:
The main limitations were: immaturity of overall survival data from the Phase 3 trial; reliance on quality-of-life data from previous health technology appraisals (as this was not collected in the trial); and a lack of direct evidence or a network of connected evidence comparing B-R with CVP-R.
Conclusions:
The ICERs for B-R vs CHOP-R and CVP-R were considerably below the thresholds normally regarded as cost-effective in England and Wales (£20,000–30,000 per QALY). 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
Patients with unresectable, metastatic colorectal cancer with wild type Kirsten ras mutational status are eligible for sequential treatments which include monoclonal antibodies as first line (1L), second line (2L), or third line (3L) regimens.
Objective:
To compare the economic outcomes of different sequences which include monoclonal antibodies for the treatment of unresectable metastatic colorectal cancer.
Methods:
Individual drug regimens for 1L, 2L, and 3L treatments were compiled according to the clinical studies in the Summary of Product Characteristics for monoclonal antibodies. They were combined into plausible treatment sequences. Health outcomes were approximated using additive median PFS benefit, and economic outcomes were calculated with a treatment sequencing costing tool. Limitations of the analysis include the clinical trial data sources, cost assumptions, and the additive PFS approach.
Results:
Seventeen sequences were evaluated. Results of the analysis show that sequences including 1L anti-EGFRs generally have relatively low-to-medium health outcomes at the highest comparative sequence costs compared to sequences including 2L anti-EGFRs, which have lower health outcomes at the lowest cost. Sequences including 3L anti-EGFRs (sequential bevazicumab-based 1L and 2L) have the highest health outcomes, with potential cost savings of €5972–€11,676 if replacing 2L anti-EGFRs or an additional cost of €5909–€12,708 if replacing 1L anti-EGFR regimens.
Conclusion:
Clinical sequences consisting of 1L and 2L line bevacizumab followed by 3L anti-EGFR potentially yield the greatest health outcomes associated with a reasonable trade-off in additional cost when replacing 1L anti-EGFRs and are potentially cost-saving if replacing 2L anti-EGFRs, per patient per lifetime. To maximize health outcomes, optimal sequences include anti-EGFRs as 3L regimen, with an approximately equivalent trade-off in costs between the most costly (anti-EGFR 2L) and least costly (anti-EGFR 1L) sequences. 相似文献
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 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
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 investigate the evolving use and expected impact of pay-for-performance (P4P) and risk-based provider reimbursement on patient access to innovative medical technology.
Methods:
Structured interviews with leading private payers representing over 110 million commercially-insured lives exploring current and planned use of P4P provider payment models, evidence requirements for technology assessment and new technology coverage, and the evolving relationship between the two topics.
Results:
Respondents reported rapid increases in the use of P4P and risk-sharing programs, with roughly half of commercial lives affected 3 years ago, just under two-thirds today, and an expected three-quarters in 3 years. All reported well-established systems for evaluating new technology coverage. Five of nine reported becoming more selective in the past 3 years in approving new technologies; four anticipated that in the next 3 years there will be a higher evidence requirement for new technology access. Similarly, four expected it will become more difficult for clinically appropriate but costly technologies to gain coverage. All reported planning to rely more on these types of provider payment incentives to control costs, but didn’t see them as a substitute for payer technology reviews and coverage limitations; they each have a role to play.
Limitations:
Interviews limited to nine leading payers with models in place; self-reported data.
Conclusion:
Likely implications include a more uncertain payment environment for providers, and indirectly for innovative medical technology and future investment, greater reliance on quality and financial metrics, and increased evidence requirements for favorable coverage and utilization decisions. Increasing provider financial risk may challenge the traditional technology adoption paradigm, where payers assumed a ‘gatekeeping’ role and providers a countervailing patient advocacy role with regard to access to new technology. Increased provider financial risk may result in an additional hurdle to the adoption of new technology, rather than substitution of provider- for payer-based gatekeeping. 相似文献