Inclusion of cost effectiveness in licensing requirements of new drugs: the fourth hurdle.
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ABSTRACT:
SUBMITTER: Taylor RS
PROVIDER: S-EPMC524116 | biostudies-literature | 2004 Oct
REPOSITORIES: biostudies-literature
Publications
Inclusion of cost effectiveness in licensing requirements of new drugs: the fourth hurdle.
Taylor R S RS Drummond M F MF Salkeld G G Sullivan S D SD
BMJ (Clinical research ed.) 20041001 7472
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Project description:New medical technology is expensive and we are introducing it in services in an unplanned manner. A significant proportion of our resources are wasted through inefficiency in the selection and production of health care strategies. We should choose new technologies which are 'need based' and 'cost effective'.
| S-EPMC5530383 | biostudies-other
Project description:BackgroundThe INVICTUS trial assessed the efficacy and safety of ripretinib compared with placebo in the management of advanced gastrointestinal stromal tumors.MethodWe used a Markov model with three health states: progression-free disease, progression disease and death. We parameterized the model from time-to-event data (progression-free survival, overall survival) of ripretinib and placebo arms in the INVICTUS trial and extrapolated to a patient's lifetime horizon. Estimates of health state utilities and costs were based on clinical trial data and the published literature. The outcomes of this model were measured in quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). Uncertainty was tested via univariate and probabilistic sensitivity analyses.ResultsThe base-case model projected improved outcomes (by 0.29 QALYs) and additional costs (by $70,251) and yielded an ICER of $244,010/QALY gained for ripretinib versus placebo. The results were most sensitive to progression rates, the price of ripretinib, and health state utilities. The ICER was most sensitive to overall survival. When overall survival in the placebo group was lower, the ICER dropped to $127,399/QALY. The ICER dropped to $150,000/QALY when the monthly cost of ripretinib decreased to $14,057. Probabilistic sensitivity analyses revealed that ripretinib was the cost-effective therapy in 41.1% of simulations at the willingness-to-pay (WTP) threshold of $150,000.ConclusionAs the fourth- or further-line therapy in advanced gastrointestinal stromal tumors, ripretinib is not cost-effective in the US. Ripretinib would achieve its cost-effectiveness with a price discount of 56% given the present effectiveness.
| S-EPMC8685288 | biostudies-literature
Project description:BackgroundThis study aimed to describe the clinical and cost-effectiveness evidence supporting reimbursement decisions of new cancer drugs and analyze the influence of trial characteristics and the cost per quality-adjusted life years (QALYs) on the likelihood of reimbursement in Sweden.Patients and methodsData were extracted from all appraisal dossiers for new cancer drugs seeking reimbursement in Sweden and claiming added therapeutical value between the years 2010 and 2020. The data were analyzed using descriptive statistics, and logistic regression models were also used with the cost per QALY, study design, comparator, and evidence on final outcomes in the clinical trials as predictors of reimbursement.ResultsAll 60 included appraisals were based on trial evidence that assessed at least one final outcome (overall survival [OS] or quality of life [QoL]), although rarely as a primary outcome. Of the appraisals with a final decision (n = 58), 79% were approved for reimbursement. Among the reimbursed drugs, only half had trial evidence demonstrating improved OS or QoL. Only one drug had trial evidence supporting improvements in both OS and QoL. The average cost per QALY for reimbursed cancer drugs was estimated to be 748 560 SEK (€73 583). A higher cost per QALY was found to decrease the likelihood of reimbursement by 9.4% for every 100 000 SEK (€9830) higher cost per QALY (P = 0.03). For cost-effectiveness models without direct evidence of improvements in final outcomes, a larger QALY gain was observed compared with those with evidence mainly relying on intermediate and surrogate outcomes.ConclusionsThere are substantial uncertainties in the clinical and cost-effectiveness evidence underlying reimbursement decisions of new cancer drugs. Decision makers should be cautious of the limited evidence on patient-centered outcomes and the implications of allocating resources to expensive treatments with uncertain value for money.
| S-EPMC9588887 | biostudies-literature
Project description:ImportanceAlthough the American Academy of Pediatrics has recommended treatment with antiobesity drugs for adolescents, the cost-effectiveness of antiobesity drugs for this population is still unknown.ObjectiveTo quantify cost-effectiveness of different antiobesity drugs available for pediatric use.Design, setting, and participantsThis economic evaluation used a Markov microsimulation model with health states defined by obesity levels. Effectiveness was measured by quality-adjusted life-years (QALYs) and costs were calculated from third-party payer perspective, estimated in 2023 US dollars over a 10-year horizon. Data were obtained from the published literature.InterventionAntiobesity drugs orlistat, liraglutide, semaglutide, and phentermine-topiramate vs no treatment. Metformin hydrochloride and 2 types of bariatric surgical procedures (sleeve gastrectomy and gastric bypass) were considered in sensitivity analysis.Main outcomes and measuresIncremental cost-effectiveness ratio.ResultsAmong the 4 antiobesity drugs currently approved for pediatric use, phentermine-topiramate was the most cost-effective with an incremental cost-effectiveness ratio of $93 620 per QALY relative to no treatment in this simulated cohort of 10 000 adolescents aged 12 to 17 years (mode, 15 years) with severe obesity (62% female). While semaglutide offered more QALYs than phentermine-topiramate, its higher cost resulted in an incremental cost-effectiveness ratio ($1 079 480/QALY) that exceeded the commonly used willingness-to-pay threshold of $100 000 to $150 000/QALY. Orlistat and liraglutide cost more and were less effective than phentermine-topiramate and semaglutide, respectively. Sleeve gastrectomy and gastric bypass were more effective than phentermine-topiramate but were also more costly, rendering them not cost-effective compared with phentermine-topiramate at the willingness-to-pay threshold of $100 000 to $150 000/QALY.Conclusions and relevanceIn this economic evaluation of weight loss drugs for adolescents with severe obesity, we found phentermine-topiramate to be a cost-effective treatment at a willingness-to-pay threshold of $100 000 to $150 000/QALY. Further research is needed to determine long-term drug efficacy and how long adolescents continue treatment.
| S-EPMC10570871 | biostudies-literature
Project description:PurposeTo examine the utility of electronic health records from a routine care setting in assessing comparative effectiveness of fourth-line anti-hypertensive drugs to treat resistant hypertension.MethodsWe conducted a cohort study using the Clinical Practice Research Datalink: a repository of electronic health records from UK primary care. We identified patients newly prescribed fourth-line anti-hypertensive drugs (aldosterone antagonist , beta-blocker, or alpha-blocker). Using propensity score-adjusted Cox proportional hazards models, we compared the incidence of the primary outcome (composite of all-cause mortality, stroke, and myocardial infarction) between patients on different fourth-line drugs. AA was the reference drug in all comparisons. Secondary outcomes were individual components of the primary outcome, blood pressure changes, and heart failure. We used a negative control outcome, Herpes Zoster, to detect unmeasured confounding.ResultsOverall, 8639 patients were included. In propensity score-adjusted analyses, the hazard ratio for the primary outcome was 0.81 (95% CI, 0.55-1.19) for beta-blockers and 0.68 (95% CI, 0.46-0.96) for alpha-blockers versus AA. Findings for individual cardiovascular outcomes trended in a more plausible direction, albeit imprecise. A trend for a protective effect for Herpes Zoster across both comparisons was seen.ConclusionsA higher rate of all-cause death in the AA group was likely due to unmeasured confounding in our analysis of the composite primary outcome, supported by our negative outcome analysis. Results for cardiovascular outcomes were plausible, but imprecise due to small cohort sizes and a low number of observed outcomes.
| S-EPMC6771826 | biostudies-literature
Project description:BackgroundOrphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan drugs, leading to questions about their cost-effectiveness.ObjectiveTo compare the value of orphan and non-orphan drugs approved by the FDA from 1999 through 2015.DesignWe searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions).ResultsWe identified estimates for 49 orphan drug-indication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009).ConclusionsOur study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending.
| S-EPMC7458970 | biostudies-literature
Project description:BackgroundConsidering the heavy economic burden of osteoporotic fractures, the limits of healthcare resources, and the recent availability of new anti-osteoporosis drugs, there is continuing interest in economic evaluation studies of osteoporosis management strategies.ObjectivesThis study aims to (1) systematically review recent economic evaluations of drugs for osteoporosis and (2) to apply an osteoporosis-specific guideline to critically appraise them.MethodsA literature search was undertaken using PubMed, EMBASE, National Health Service Economic Evaluation database, and the Cost-Effectiveness Analysis Registry to identify original articles containing economic evaluations of anti-osteoporosis drugs, published between 1 July, 2013 and 31 December, 2019. A recent European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases-International Osteoporosis Foundation (ESCEO-IOF) guideline for the conduct and reporting of economic evaluations in osteoporosis was used to assess the quality of included articles.ResultsThe database search retrieved 3860 records, of which 27 studies fulfilled the inclusion criteria. These studies were conducted in 15 countries; 12 active drugs were assessed, including various traditional pharmacological treatments such as bisphosphonates, raloxifene, strontium ranelate, denosumab, and teriparatide, and new agents such as abaloparatide, romosozumab, and gastro-resistant risedronate. Eight out of 12 studies that compared traditional oral bisphosphonates to other active interventions (denosumab, zoledronic acid, gastro-resistant risedronate, and teriparatide) suggested that the other active agents were generally cost-effective or dominant. Additionally, the cost-effectiveness of sequential therapy has recently been assessed and indications are that it can lead to extra health benefits (larger gains in quality-adjusted life-year). The key drivers of cost effectiveness included baseline fracture risk, drug effect on the risk of fractures, drug cost, and medication adherence/persistence. The current average score for quality assessment was 17 out of 25 (range 2-15); room for improvement was observed for most studies, which could potentially be explained by the fact that most studies were published prior to the osteoporosis-specific guideline. Greater adherence to guideline recommendations was expected for future studies. The quality of reporting was also suboptimal, especially with regard to treatment side effects, treatment effect after discontinuation, and medication adherence.ConclusionsThis updated review provides an overview of recently published cost-effectiveness analyses. In comparison with a previous review, recent economic evaluations of anti-osteoporosis drugs were conducted in more countries and included more active drugs and sequential therapy as interventions/comparators. The updated economic evidence could help decision makers prioritize health interventions and the unmet/unreported quality issues indicated by the osteoporosis-specific guideline could be useful in improving the transparency, quality, and comparability of future economic evaluations in osteoporosis.
| S-EPMC7867562 | biostudies-literature
Project description:BackgroundAt least 10% of the 56,000 annual new HIV infections in the United States are caused by individuals with acute HIV infection (AHI). It unknown whether the health benefits and costs of routine nucleic acid amplification testing (NAAT) are justified, given the availability of newer fourth-generation immunoassay tests.MethodsUsing a dynamic HIV transmission model instantiated with U.S. epidemiologic, demographic, and behavioral data, I estimated the number of acute infections identified, HIV infections prevented, quality-adjusted life years (QALYs) gained, and the cost-effectiveness of alternative screening strategies. I varied the target population (everyone aged 15-64, injection drug users [IDUs] and men who have sex with men [MSM], or MSM only), screening frequency (annually, or every six months), and test(s) utilized (fourth-generation immunoassay only, or immunoassay followed by pooled NAAT).ResultsAnnual immunoassay testing of MSM reduces incidence by 9.5% and costs <$10,000 per QALY gained. Adding pooled NAAT identifies 410 AHI per year, prevents 9.6% of new cases, costs $92,000 per QALY gained, and remains <$100,000 per QALY gained in settings where undiagnosed HIV prevalence exceeds 4%. Screening IDUs and MSM annually with fourth-generation immunoassay reduces incidence by 13% with cost-effectiveness <$10,000 per QALY gained. Increasing the screening frequency to every six months reduces incidence by 11% (MSM only) or 16% (MSM and IDUs) and costs <$20,000 per QALY gained.ConclusionsPooled NAAT testing every 12 months of MSM and IDUs in the United States prevents a modest number of infections, but may be cost-effective given sufficiently high HIV prevalence levels. However, testing via fourth-generation immunoassay every six months prevents a greater number of infections, is more economically efficient, and may obviate the benefits of acute HIV screening via NAAT.
| S-EPMC3218000 | biostudies-literature
Project description:The UK NHS Women's National Breast Screening programme aims to detect breast cancer early. The reference standard approach requires mammograms to be independently double-read by qualified radiology staff. If two readers disagree, arbitration by an independent reader is undertaken. Whilst this process maximises accuracy and minimises recall rates, the procedure is labour-intensive, adding pressure to a system currently facing a workforce crisis. Artificial intelligence technology offers an alternative to human readers. While artificial intelligence has been shown to be non-inferior versus human second readers, the minimum requirements needed (effectiveness, set-up costs, maintenance, etc) for such technology to be cost-effective in the NHS have not been evaluated. We developed a simulation model replicating NHS screening services to evaluate the potential value of the technology. Our results indicate that if non-inferiority is maintained, the use of artificial intelligence technology as a second reader is a viable and potentially cost-effective use of NHS resources.
| S-EPMC10542368 | biostudies-literature
Project description:BackgroundFalls may lead to hip fractures, which have a detrimental effect on the prognosis of patients as well as a considerable impact on healthcare expenditures. Since a secondary hip fracture (SHF) may lead to even higher costs than primary fractures, the development of innovative services is crucial to limit falls and curb costs in high-risk patients. An early economic evaluation assessed which patients with a second hip fracture could benefit most from an exoskeleton preventing falls and whether its development is feasible.MethodsThe life-course of hip fractured patients presenting with dementia or cardiovascular diseases was simulated using a Markov model relying on the United Kingdom administrative data and complemented by published literature. A group of experts provided the exoskeleton parameters. Secondary analyses included a threshold analysis to identify the exoskeleton requirements (e.g. minimum impact of the exoskeleton on patients' quality of life) leading to a reimbursable incremental cost-effectiveness ratio. Similarly, the uncertainty around these requirements was modelled by varying their standard errors and represented alongside population Expected Value of Perfect Information (EVPI).ResultsOur base-case found the exoskeleton cost-effective when providing a statistically significant reduction in SHF risk. The secondary analyses identified 286 cost-effective combinations of the exoskeleton requirements. The uncertainty around these requirements was explored producing further 22,880 scenarios, which showed that this significant reduction in SHF risk was not necessary to support the exoskeleton adoption in clinical practice. Conversely, a significant improvement in women quality of life was crucial to obtain an acceptable population EVPI regardless of the cost of the exoskeleton.ConclusionsOur study identified the exoskeleton requisites to be cost-effective and the value of future research. Decision-makers could use our analyses to assess not only whether the exoskeleton could be cost-effective but also how much further research and development of the exoskeleton is worth to be pursued.
| S-EPMC7565816 | biostudies-literature