Adapting a global cost-effectiveness model to local country requirements: posaconazole case study.

BACKGROUND: Many countries have various requirements for local economic analyses to assess the value of a new health technology and/or to secure reimbursement. This study presents a case study of an economic model developed to assess the cost-effectiveness of posaconazole vs standard azole therapy (fluconazole/itraconazole) to prevent invasive fungal infections (IFIs), which was adapted by at least 11 countries. METHODS: Modeling techniques were used to assess the cost-effectiveness of posaconazole vs fluconazole/itraconazole as IFI prophylaxis in patients with acute myelogenous leukemia or myelodysplastic syndromes and chemotherapy-induced neutropenia. For the core model, the probabilities of experiencing an IFI, IFI-related death, and death from other causes were estimated from clinical trial data. Long-term mortality, drug costs, and IFI treatment costs were obtained from secondary sources. Locally changed parameters were probabilities of long-term death and survival, currency, drug costs, health utility, IFI treatment costs, and discount rate. RESULTS: Locally adapted cost-effective modeling studies indicate that prophylaxis with posaconazole, compared with fluconazole/itraconazole, prolongs survival, and, in most countries, is cost-saving. In all countries, the model predicted that prophylaxis with posaconazole would be associated with an increase in life-years, with increases ranging from 0.016-0.1 life-year saved. In all countries, use of the model led to posaconazole being approved by the appropriate reimbursement authority. LIMITATIONS: The study did not have power to detect differences between posaconazole and fluconazole or itraconazole separately. The risk of death after 100 days was assumed to be equal for those who did and did not develop an IFI, and equal probabilities of IFI-related and other death during the trial period were used for both groups. CONCLUSIONS: A core economic model was successfully adapted locally by several countries. The model showed that posaconazole was cost-saving or cost-effective vs fluconazole/itraconazole and led to positive reimbursement listings.

Cost of invasive fungal infections in the era of new diagnostics and expanded treatment options.

STUDY OBJECTIVE: To determine the true institutional cost of treating invasive fungal infections in light of recent advances in diagnostic techniques and antifungal therapies for both treatment and prophylaxis of these infections. DESIGN: Economic analysis. SETTING: Academic medical center. PATIENTS: A total of 200 patients discharged from the hospital during 2004-2005 with a diagnosis of proven, probable, or possible aspergillosis, cryptococcosis, invasive candidiasis, or zygomycosis (cases). Patients were matched in a 1:1 fashion with patients having similar underlying disease states but no invasive fungal infections (controls). MEASUREMENTS AND MAIN RESULTS: Data on demographic and clinical characteristics were collected from patients' medical records. In addition, information concerning each patient's hospitalization was recorded. Resource utilization data for a patient's entire hospitalization were collected from the hospital's charge databases and converted to costs. These data were compared between the cases and the controls. After adjusting for race-ethnicity, sex, age, and comorbid illnesses, mean total hospital cost for cases was $32,196 more than for controls (p<0.0001). Nonpharmacy costs accounted for the majority (63%) of this difference, and an additional $3996 was attributed to systemic antifungal drugs. The mean length of hospital stay was longer for cases than controls (25.8 vs 18.4 days). CONCLUSION: Treatment of patients with invasive fungal infections was associated with a significantly higher inpatient hospital cost compared with controls. However, due to new diagnostic techniques and effective antifungal therapy, the relative cost of these infections appears to be at least stable compared with the previous decade. These findings can help assess the utility of cost-avoidance strategies such as antifungal prophylaxis and application of appropriate treatment.

Cost effectiveness of golimumab for the treatment of active psoriatic arthritis.

BACKGROUND: Golimumab is a novel TNF-α inhibitor licensed to treat patients with active PsA. Although its clinical efficacy has been proven in clinical trials, its cost effectiveness is yet to be established. OBJECTIVES: To estimate the cost effectiveness of golimumab among patients with active PsA from the UK NHS perspective. METHODS: A decision analytic model was used to simulate progression of a hypothetical cohort of active PsA patients on golimumab and other TNF-α inhibitors as well as palliative care. The clinical evidence was derived from clinical trials of TNF-α inhibitors and compared using mixed treatment models. The primary outcome measure was quality-adjusted life years (QALYs) estimated based on change in Health Assessment Questionnaire (HAQ) and Psoriasis Area Severity Index (PASI) from baseline. The annual acquisition cost of golimumab was assumed to be identical to annual cost of other subcutaneous TNF-α inhibitors. The resource use costs and outcomes were discounted at 3.5% over a period of 40 years. The uncertainty surrounding important variables was further explored using probabilistic sensitivity analyses (PSA). RESULTS: TNF-α inhibitors were significantly superior to palliative care but comparable to each other on Psoriatic Arthritis Response Criteria (PsARC), HAQ and PASI response. The incremental cost effectiveness ratio (ICERs) for golimumab compared to palliative care was £16,811 for PsA patients and £16,245 for a subgroup of PsA patients with significant psoriasis. At an acceptability threshold of £30,000 per QALY, the probability of golimumab being cost effective is 89%. CONCLUSION: Once monthly, golimumab is a cost-effective treatment alternative for patients with active PsA. With its patient-focussed attributes, golimumab is likely to offer additional choice in PsA treatment.

Cost-effectiveness of evidence-based pharmacotherapy or cognitive behavior therapy compared with community referral for major depression in predominantly low-income minority women.

BACKGROUND: Few clinical trials have evaluated interventions for major depressive disorder in samples of low-income minority women, and little is known about the cost-effectiveness of depression interventions for this population. OBJECTIVE: To evaluate the cost-effectiveness of pharmacotherapy or cognitive behavior therapy (CBT) compared with community referral for major depression in low-income minority women. DESIGN, SETTING, AND PARTICIPANTS: A randomized clinical trial was conducted in 267 women with current major depression. INTERVENTIONS: Participants were randomly assigned to pharmacotherapy (paroxetine hydrochloride or bupropion hydrochloride) (n = 88), CBT (n = 90), or community referral (n = 89). MAIN OUTCOME MEASURES: The main outcomes were intervention and health care costs, depression-free days, and quality-adjusted life years based on Hamilton Depression Rating Scale scores and Medical Outcomes Study 36-Item Short-Form Health Survey summary scores for 12 months. Cost-effectiveness ratios were estimated to compare incremental patient outcomes with incremental costs for pharmacotherapy relative to community referral and for CBT relative to community referral. RESULTS: Compared with the community referral group, the pharmacotherapy group had significantly lower adjusted mean Hamilton Depression Rating Scale scores from the 3rd month through the 10th month (P = .04 to P<.001) of the study, and the CBT group had significantly lower adjusted mean scores from the 5th month through the 10th month (P = .03 to P = .049). There were significantly more depression-free days in the pharmacotherapy group (mean, 39.7; 95% confidence interval, 12.9-66.5) and the CBT group (mean, 25.80; 95% confidence interval, 0.04-51.50) than in the community referral group. The cost per additional depression-free day was USD 24.65 for pharmacotherapy and USD 27.04 for CBT compared with community referral. CONCLUSIONS: Effective treatment for depression in low-income minority women reduces depressive symptoms but increases costs compared with community referral. The pharmacotherapy and CBT interventions were cost-effective relative to community referral for the health care system.

A review of the economic burden of ADHD.

Attention-deficit hyperactivity disorder (ADHD) is a common disorder that is associated with broad functional impairment among both children and adults. The purpose of this paper is to review and summarize available literature on the economic costs of ADHD, as well as potential economic benefits of treating this condition. A literature search was performed using MEDLINE to identify all published articles on the economic implications of ADHD, and authors were contacted to locate conference abstracts and articles in press that were not yet indexed. In total, 22 relevant items were located including published original studies, economic review articles, conference presentations, and reports available on the Internet. All costs were updated and presented in terms of year 2004 US dollars. A growing body of literature, primarily published in the United States, has demonstrated that ADHD places a substantial economic burden on patients, families, and third-party payers. Results of the medical cost studies consistently indicated that children with ADHD had higher annual medical costs than either matched controls (difference ranged from 503 dollars to 1,343 dollars) or non-matched controls (difference ranged from 207 dollars to 1,560 dollars) without ADHD. Two studies of adult samples found similar results, with significantly higher annual medical costs among adults with ADHD (ranging from 4,929 dollars to 5,651 dollars) than among matched controls (ranging from 1,473 dollars to 2,771 dollars). A limited number of studies have examined other economic implications of ADHD including costs to families; costs of criminality among individuals with ADHD; costs related to common psychiatric and medical comorbidities of ADHD; indirect costs associated with work loss among adults with ADHD; and costs of accidents among individuals with ADHD. Treatment cost-effectiveness studies have primarily focused on methylphenidate, which is a cost-effective treatment option with cost-effectiveness ratios ranging from 15,509 dollars to 27,766 dollars per quality-adjusted life year (QALY) gained. As new treatments are introduced it will be important to evaluate their cost-effectiveness to provide an indication of their potential value to clinicians, patients, families, and third-party payers.

The health-related quality of life and economic burden of constipation.

Constipation is a prevalent condition that disproportionately affects women and older adults and leads to self-medication and/or medical consultation. It occurs as a result of functional idiopathic causes or secondarily as a result of a variety of factors including dietary and exercise patterns, adverse effects of medication and disease processes. Constipation is often perceived to be a benign, easily treated condition with short-term treatment being relatively straightforward; however, chronic constipation is associated with mild complications that, left untreated, can develop into more serious bowel complaints (faecal impaction, incontinence and bowel perforations) with further implications for healthcare costs and the patient's health-related quality of life (HR-QOL). This review summarises the evidence of the HR-QOL impact and economic burden of constipation on patients. Relatively few studies have systematically explored the HR-QOL and economic impact of constipation; however, the existing evidence suggests that HR-QOL is lower in patients with constipation than in non-constipated individuals, and treatments for constipation improve HR-QOL. Additionally, constipation represents an economic burden for the patient and healthcare provider. Resource utilisation associated with the diagnosis and management of constipation is a significant cost driver, whereas constipation prevention programmes have demonstrated cost savings.

The effect on social welfare of a switch of second-generation antihistamines from prescription to over-the-counter status: a microeconomic analysis.

BACKGROUND: The US Food and Drug Administration (FDA) recently held a meeting to determine whether the status of second-generation antihistamines (SGAs) should be switched from prescription (Rx) to over-the-counter (OTC) status. OBJECTIVE: This article provides a conceptual microeconomic framework for addressing issues regarding the impact of such a switch on social welfare. METHODS: A review of the economic literature on Rx-to-OTC switches was conducted. Relevant articles published in English between 1990 and 2001 were identified through searches of ABI Info, EconLit, PsychInfo, MEDLINE, CANCERLIT, AIDSLINE, and HealthStar, as well as a general Internet search for statements in the press or on the FDA Web site. The search terms used were Rx, prescription, OTC, over-the-counter, second-generation antihistamines, nonsedating antihistamines, first-generation antihistamines, and sedating antihistamines. Microeconomic models focusing on consumer surplus were employed to determine the potential price response and social-welfare implications of a switch of SGAs to OTC status. RESULTS: Unlike the agents involved in previous Rx-to-OTC switches, SGAs are still under patent protection. Economic theory suggests that a firm that is protected by a patent will price aggressively. The market for OTC SGAs is likely to be more elastic due to a lack of insurance coverage for OTC products; hence, drug manufacturers would be likely to charge a lower price if SGAs were sold OTC. However, a lower price does not necessarily guarantee an improvement in social welfare; the net impact is determined by whether the increase in consumer surplus outweighs the deadweight loss (losses of consumer and producer surplus not transferred to other parties). Additionally, the assumption of a price reduction would be called into question if there were inequalities in marginal costs between the Rx and OTC markets. In this situation, the postswitch price might increase or not be reduced significantly. CONCLUSIONS: It is uncertain whether granting OTC status to SGAs would be cost saving to society, particularly as these drugs are patent protected. The social-welfare implications of such a switch would depend heavily on pricing strategies and consumer behavior. Further analyses are needed to determine how both factors influence social welfare; only then can the costs and benefits of a switch be understood completely.