Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007

Background

Orphan drugs are indicated for the treatment of rare diseases which, in the EU, are defined as those with a prevalence of <5 per 10 000 inhabitants. Characteristically, these diseases negatively affect health-related quality of life and may be life threatening. The EU has passed legislation to encourage pharmaceutical companies to invest in research programmes into rare diseases, with the aim of developing new, safe and effective orphan drugs.

Objectives

To describe the status of orphan drugs in five countries in the EU (France, Germany, the UK, Italy and Spain), estimate the mean annual cost per patient and indication of these orphan drugs, and determine the associated cost of these drugs in comparison with overall spending on drugs in each country (year 2007 values).

Methods

The analysis was limited solely to costs of orphan drugs with sales data available for 2007. The mean annual cost per patient was estimated using recommended regimens for maintenance dose and duration from the summary of product characteristics. Likewise, the ratio between annual costs per patient for treatment of each disease and its prevalence was calculated. Sales data were available for at least one of the countries studied for 38 of the 44 orphan drugs authorized by the European Medicines Agency. Only 21 products had data available for all five countries studied.

Results

Germany was the country with access to the largest number of orphan drugs (36), followed by the UK (34), Spain (28), France (27) and Italy (25). The mean annual cost per patient and indication of the 38 orphan drugs on the market ranged widely from €331 to €337 501. It appears that orphan drugs indicated to treat diseases with a prevalence of <2 per 10 000 inhabitants have higher annual per-patient costs than those indicated to treat diseases with a higher prevalence. The percentage of total drug spending accounted for by orphan drugs in 2007 was 1.7% in France, 2.1% in Germany, 1.0% in the UK, 1.5% in Italy and 2.0% in Spain, with an average overall percentage of 1.7% for these five countries.

Conclusions

In 2007, spending on orphan drugs in five European countries was acceptable in terms of the percentage of these countries’ overall drug expenditure. Mean annual costs per patient of orphan drugs varied widely, with costs being related to the prevalence of the disease for which the product is indicated.     

Cost and efficiency of public sector sexually transmitted infection clinics in Andhra Pradesh, India

Background

Control of sexually transmitted infections (STIs) is an important part of the effort to reduce the risk of HIV/AIDS. STI clinics in the government hospitals in India provide services predominantly to the poor. Data on the cost and efficiency of providing STI services in India are not available to help guide efficient use of public resources for these services.

Methods

Standardised methods were used to obtain detailed cost and output data for the 2003–2004 fiscal year from written records and interviews in 14 government STI clinics in the Indian state of Andhra Pradesh. The economic cost per patient receiving STI treatment was calculated, and the variations of total and unit costs across the STI clinics analysed. Multivariate regression technique was used to estimate incremental unit costs. The optimal number of STIs that could be handled by the clinics was estimated.

Results

18807 STIs were diagnosed and treated at the 14 STI clinics in fiscal year 2003–2004 (range 323–2784, median 1199). The economic cost of treating each STI varied 5-fold from Indian Rupees (INR) 225.5 (US$ 4.91) to INR 1201.5 (US$ 26.15) between 13 clinics, with one other clinic having a very high cost of INR 2478.5 (US$ 53.94). The average cost per STI treated for all 14 clinics combined was INR 729.5 (US$ 15.88). Personnel salaries made up 76.2% of the total cost. The number of STIs treated per doctor full-time equivalent and cost-efficiency for each STI treated had a significant direct non-linear relation (p < 0.001, R² = 0.81; power function). With a multiple regression model, apart from the fixed costs, the incremental cost for each STI detected and cost of treatment was INR 55.57 (US$ 1.21) and for each follow-up visit was INR 3.75 (US$ 0.08). Based on estimates of optimal STI cases that could be handled without compromising quality by each doctor full-time equivalent available, it was projected that at 8 of the 14 clinics substantially more STI cases could be handled, which could increase the total STI cases treated at the 14 clinics combined by 38% at an additional cost of only 3.5% for service provision.

Conclusion

There is un-utilised capacity in the public sector STI clinics in this Indian state. Efforts to facilitate utilisation of this capacity would be useful, as this would enable more poor patients with STIs to be served at minimal additional cost, and would also reduce the cost per STI treated leading to more efficient use of public resources.     

Capacity utilization and the cost of primary care visits: Implications for the costs of scaling up health interventions

Objective

A great deal of international attention has been focussed recently on how much additional funding is required to scale up health interventions to meet global targets such as the Millennium Development Goals (MDGs). Most of the cost estimates that have been made in response have assumed that unit costs of delivering services will not change as coverage increases or as more and more interventions are delivered together. This is most unlikely. The main objective of this paper is to measure the impact of patient load on the cost per visit at primary health care facilities and the extent to which this would influence estimates of the costs and financial requirements to scale up interventions.

Methods

Multivariate regression analysis was used to explore the determinants of variability in unit costs using data for 44 countries with a total of 984 observations.

Findings

Controlling for other possible determinants, we find that the cost of an outpatient visit is very sensitive to the number of patients seen by providers each day at primary care facilities. Each 1% increase in patient through-put results, on average, in a 27% reduction in the cost per visit (p < 0.0001), which can lead to a difference of up to $30 in the observed costs of an outpatient visit at primary facilities in the same setting, other factors held constant.

Conclusion

Variability in capacity utilization, therefore, need to be taken into account in cost estimates, and the paper develops a method by which this can be done.     

Cost of individual peer counselling for the promotion of exclusive breastfeeding in Uganda

Background

Exclusive breastfeeding (EBF) for 6 months is the recommended form of infant feeding. Support of mothers through individual peer counselling has been proved to be effective in increasing exclusive breastfeeding prevalence. We present a costing study of an individual peer support intervention in Uganda, whose objective was to raise exclusive breastfeeding rates at 3 months of age.

Methods

We costed the peer support intervention, which was offered to 406 breastfeeding mothers in Uganda. The average number of counselling visits was about 6 per woman. Annual financial and economic costs were collected in 2005-2008. Estimates were made of total project costs, average costs per mother counselled and average costs per peer counselling visit. Alternative intervention packages were explored in the sensitivity analysis. We also estimated the resources required to fund the scale up to district level, of a breastfeeding intervention programme within a public health sector model.

Results

Annual project costs were estimated to be US$56,308. The largest cost component was peer supporter supervision, which accounted for over 50% of total project costs. The cost per mother counselled was US$139 and the cost per visit was US$26. The cost per week of EBF was estimated to be US$15 at 12 weeks post partum. We estimated that implementing an alternative package modelled on routine public health sector programmes can potentially reduce costs by over 60%. Based on the calculated average costs and annual births, scaling up modelled costs to district level would cost the public sector an additional US$1,813,000.

Conclusion

Exclusive breastfeeding promotion in sub-Saharan Africa is feasible and can be implemented at a sustainable cost. The results of this study can be incorporated in cost effectiveness analyses of exclusive breastfeeding promotion programmes in sub-Saharan Africa.     

Nutrition therapy cost analysis in the US

Background

Bloodstream infections (BSI) occur in up to 350 000 inpatient admissions each year in the US, with BSI rates among patients receiving parenteral nutrition (PN) varying from 1.3% to 39%. BSI-attributable costs were estimated to approximate $US12000 per episode in 2000. While previous studies have compared the cost of different PN preparation methods, this analysis evaluates both the direct costs of PN and the treatment costs for BSI associated with different PN delivery methods to determine whether compounded or manufactured pre-mixed PN has lower overall costs.

Objective

The purpose of this study was to compare costs in the US associated with compounded PN versus pre-mixed multi-chamber bag (MCB) PN based on underlying infection risk.

Methods

Using claims information from the Premier Perspective? database, multivariate logistic regression was used to estimate the risk of infection. A total of 44 358 hospitalized patients aged ≥18 years who received PN between 1 January 2005 and 31 December 2007 were included in the analyses. A total of 3256 patients received MCB PN and 41 102 received compounded PN. The PN-associated costs and length of stay were analysed using multivariate ordinary least squares regression models constructed to measure the impact of infectious events on total hospital costs after controlling for baseline and clinical patient characteristics.

Results

There were 7.3 additional hospital days attributable to BSI. After adjustment for baseline variables, the probability of developing a BSI was 30% higher in patients receiving compounded PN than in those receiving MCB PN (16.1% vs 11.3%; odds ratio= 1.56; 95% CI 1.37, 1.79; p<0.0001), demonstrating 2172 potentially avoidable infections. The observed daily mean PN acquisition cost for patients receiving MCB PN was $US164 (including all additives and fees) compared with $US239 for patients receiving compounded PN (all differences p < 0.001). With a mean cost attributable to BSI of $US16 141, the total per-patient savings (including avoided BSI and PN costs) was $US1545.

Conclusion

In this analysis of real-world PN use, MCB PN is associated with lower costs than compounded PN with regards to both PN acquisition and potential avoidance of BSI. Our base case indicates that $US1545 per PN patient may be saved; even if as few as 50% of PN patients are candidates for standardized pre-mix formulations, a potential savings of $US773 per patient may be realized.     

The economic burden of inpatient paediatric care in Kenya: household and provider costs for treatment of pneumonia, malaria and meningitis

Background

Knowledge of treatment cost is essential in assessing cost effectiveness in healthcare. Evidence of the potential impact of implementing available interventions against childhood illnesses in developing countries challenges us to define the costs of treating these diseases. The purpose of this study is to describe the total costs associated with treatment of pneumonia, malaria and meningitis in children less than five years in seven Kenyan hospitals.

Methods

Patient resource use data were obtained from largely prospective evaluation of medical records and household expenditure during illness was collected from interviews with caretakers. The estimates for costs per bed day were based on published data. A sensitivity analysis was conducted using WHO-CHOICE values for costs per bed day.

Results

Treatment costs for 572 children (pneumonia = 205, malaria = 211, meningitis = 102 and mixed diagnoses = 54) and household expenditure for 390 households were analysed. From the provider perspective the mean cost per admission at the national hospital was US $95.58 for malaria, US $177.14 for pneumonia and US $284.64 for meningitis. In the public regional or district hospitals the mean cost per child treated ranged from US $47.19 to US $81.84 for malaria and US $54.06 to US $99.26 for pneumonia. The corresponding treatment costs in the mission hospitals were between US $43.23 to US $88.18 for malaria and US $ 43.36 to US $142.22 for pneumonia. Meningitis was treated for US $ 189.41 at the regional hospital and US $ 201.59 at one mission hospital. The total treatment cost estimates were sensitive to changes in the source of bed day costs. The median treatment related household payments within quintiles defined by total household expenditure differed by type of facility visited. Public hospitals recovered up to 40% of provider costs through user charges while mission facilities recovered 44% to 100% of costs.

Conclusion

Treatments cost for inpatient malaria, pneumonia and meningitis vary by facility type, with mission and tertiary referral facilities being more expensive compared to primary referral. Households of sick children contribute significantly towards provider cost through payment of user fees. These findings could be used in cost effectiveness analysis of health interventions.     

Economic modeling of the combined effects of HIV-disease, cholesterol and lipoatrophy based on ACTG 5142 trial data

Background

Insomnia is perhaps the most common sleep disorder in the general population, and is characterised by a range of complaints around difficulties in initiating and maintaining sleep, together with impaired waking function. There is little quantitative information on treatment pathways, costs and outcomes. The aims of this New Zealand study were to determine from which healthcare practitioners patients with insomnia sought treatment, treatment pathways followed, the net costs of treatment and the quality of life improvements obtained.

Methods

The study was retrospective and prevalence based, and was both cost effectiveness (CEA) and a cost utility (CUA) analysis. Micro costing techniques were used and a societal analytic perspective was adopted. A deterministic decision tree model was used to estimate base case values, and a stochastic version, with Monte Carlo simulation, was used to perform sensitivity analysis. A probability and cost were attached to each event which enabled the costs for the treatment pathways and average treatment cost to be calculated. The inputs to the model were prevalence, event probabilities, resource utilisations, and unit costs. Direct costs and QALYs gained were evaluated.

Results

The total net benefit of treating a person with insomnia was $482 (the total base case cost of $145 less health costs avoided of $628). When these results were applied to the total at-risk population in New Zealand additional treatment costs incurred were $6.6 million, costs avoided $28.4 million and net benefits were $21.8 million. The incremental net benefit when insomnia was "successfully" treated was $3,072 per QALY gained.

Conclusions

The study has brought to light a number of problems relating to the treatment of insomnia in New Zealand. There is both inadequate access to publicly funded treatment and insufficient publicly available information from which a consumer is able to make an informed decision on the treatment and provider options. This study suggests that successful treatment of insomnia leads to direct cost savings and improved quality of life.     

The cost of antiretroviral therapy in Haiti

Background

Tobacco smoking is a risk factor for age-related macular degeneration, but studies of ex-smokers suggest quitting can reduce the risk.

Methods

We fitted a function predicting the decline in risk of macular degeneration after quitting to data from 7 studies involving 1,488 patients. We assessed the cost-effectiveness of smoking cessation in terms of its impact on macular degeneration-related outcomes for 1,000 randomly selected U.S. smokers. We used a computer simulation model to predict the incidence of macular degeneration and blindness, the number of quality-adjusted life-years (QALYs), and direct costs (in 2004 U.S. dollars) until age 85 years. Cost-effectiveness ratios were based on the cost of the Massachusetts Tobacco Control Program. Costs and QALYs were discounted at 3% per year.

Results

If 1,000 smokers quit, our model predicted 48 fewer cases of macular degeneration, 12 fewer cases of blindness, and a gain of 1,600 QALYs. Macular degeneration-related costs would decrease by $2.5 million if the costs of caregivers for people with vision loss were included, or by $1.1 million if caregiver costs were excluded. At a cost of $1,400 per quitter, smoking cessation was cost-saving when caregiver costs were included, and cost about $200 per QALY gained when caregiver costs were excluded. Sensitivity analyses had a negligible impact. The cost per quitter would have to exceed $77,000 for the cost per QALY for smoking cessation to reach $50,000, a threshold above which interventions are sometimes viewed as not cost-effective.

Conclusion

Smoking cessation is unequivocally cost-effective in terms of its impact on age-related macular degeneration outcomes alone.     

The costs of a sexually transmitted infection outreach and treatment programme targeting most at risk youth in Tajikistan

Background

Communicable diseases are the leading causes of illness, deaths, and disability in sub-Saharan Africa. To address these threats, countries within the World Health Organization (WHO) African region adopted a regional strategy called Integrated Disease Surveillance and Response (IDSR). This strategy calls for streamlining resources, tools, and approaches to better detect and respond to the region's priority communicable disease. The purpose of this study was to analyze the incremental costs of establishing and subsequently operating activities for detection and response to the priority diseases under the IDSR.

Methods

We collected cost data for IDSR activities at central, regional, district, and primary health care center levels from Burkina Faso, Eritrea, and Mali, countries where IDSR is being fully implemented. These cost data included personnel, transportation items, office consumable goods, media campaigns, laboratory and response materials and supplies, and annual depreciation of buildings, equipment, and vehicles.

Results

Over the period studied (2002–2005), the average cost to implement the IDSR program in Eritrea was $0.16 per capita, $0.04 in Burkina Faso and $0.02 in Mali. In each country, the mean annual cost of IDSR was dependent on the health structure level, ranging from $35,899 to $69,920 at the region level, $10,790 to $13,941 at the district level, and $1,181 to $1,240 at the primary health care center level. The proportions spent on each IDSR activity varied due to demand for special items (e.g., equipment, supplies, drugs and vaccines), service availability, distance, and the epidemiological profile of the country.

Conclusion

This study demonstrates that the IDSR strategy can be considered a low cost public health system although the benefits have yet to be quantified. These data can also be used in future studies of the cost-effectiveness of IDSR.     

Cost-utility of Intravenous Immunoglobulin (IVIG) compared with corticosteroids for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in Canada

Objectives

Intravenous immunoglobulin (IVIG) has demonstrated improvement in chronic inflammatory demyelinating polyneuropathy (CIDP) patients in placebo controlled trials. However, IVIG is also much more expensive than alternative treatments such as corticosteroids. The objective of the paper is to evaluate, from a Canadian perspective, the cost-effectiveness of IVIG compared to corticosteroid treatment of CIDP.

Methods

A markov model was used to evaluate the costs and QALYs for IVIG and corticosteroids over 5 years of treatment for CIDP. Patients initially responding to IVIG could remain a responder or relapse every 12 week model cycle. Non-responding IVIG patients were assumed to be switched to corticosteroids. Patients on corticosteroids were at risk of a number of adverse events (fracture, diabetes, glaucoma, cataract, serious infection) in each cycle.

Results

Over the 5 year time horizon, the model estimated the incremental costs and QALYs of IVIG treatment compared to corticosteroid treatment to be $124,065 and 0.177 respectively. The incremental cost per QALY gained of IVIG was estimated to be $687,287. The cost per QALY of IVIG was sensitive to the assumptions regarding frequency and dosing of maintenance IVIG.

Conclusions

Based on common willingness to pay thresholds, IVIG would not be perceived as a cost effective treatment for CIDP.     

Family practice nurses supporting self-management in older patients with mild osteoarthritis: a randomized trial

Background

Context: Acute cough is a very common symptom presentation among children in primary care and is usually due to respiratory infection, yet its cost is unknown. An estimate of the cost to healthcare providers and parents would aid budgetary decision-making, and provide an insight into the need for interventions to reduce the burden. Purpose: To estimate the cost per child per episode, and the annual population cost in the UK, of acute cough in pre-school children presenting to primary care.

Methods

Design: Incidence and prevalence-based cost-of-illness study from the perspectives of the UK NHS and of parents and caregivers. Setting: 11 general practices in Bristol, UK. Subjects: 121 children without known asthma aged 3 to 59 months presenting for the first time with an acute (≤ 28 days) cough.

Results

Mean cost per episode to the NHS: £27.43 (95% CI: £24.38 – £30.49). Mean cost per episode to parents and carers: £14.77 (£4.90 – £24.65). Annual cost to the NHS in the UK: at least £31.5 m (95% CI: £28.0 m – £35.0 m).

Conclusion

The cost burden on the healthcare provider of acute cough in pre-school children is substantial; the majority of this cost arises from consultations with general practitioners. Parents experience some personal cost through travel and expenditure on over-the-counter preparations, and may suffer significantly if loss of earnings is experienced. There is scope for evaluating interventions designed to reduce this burden.     

Policymaking based on CERs: changes in costs are not the same as changes in benefits

Objective

The study aims to investigate whether laparoscopic cholecystectomy (LC) is a cost-effective strategy for managing gallbladder-stone disease compared to the conventional open cholecystectomy(OC) in a Thai setting.

Design and Setting

Using a societal perspective a cost-utility analysis was employed to measure programme cost and effectiveness of each management strategy. The costs borne by the hospital and patients were collected from Chiang Rai regional hospital while the clinical outcomes were summarised from a published systematic review of international and national literature. Incremental cost per Quality Adjusted Life Year (QALY) derived from a decision tree model.

Results

The results reveal that at base-case scenario the incremental cost per QALY of moving from OC to LC is 134,000 Baht under government perspective and 89,000 Baht under a societal perspective. However, the probabilities that LC outweighed OC are not greater than 95% until the ceiling ratio reaches 190,000 and 270,000 Baht per QALY using societal and government perspective respectively.

Conclusion

The economic evaluation results of management options for gallstone disease in Thailand differ from comparable previous studies conducted in developed countries which indicated that LC was a cost-saving strategy. Differences were due mainly to hospital costs of post operative inpatient care and value of lost working time. The LC option would be considered a cost-effective option for Thailand at a threshold of three times per capita gross domestic product recommended by the committee on the Millennium Development Goals.     

Cost-analysis of different management policies for patients with mild hepatitis A virus infection in Kazakhstan

Objective

For patients with mild hepatitis A virus (HAV) infection, this study compared estimates of total costs associated with managing cases under a policy of mandatory hospitalization in the Republic of Kazakhstan and estimates of total costs associated with managing cases in outpatient settings. Costs were estimated both from the perspective of the Ministry of Health and from a broader societal perspective.

Methods

Data were collected by using a standardized structured questionnaire. For cases of mild HAV infection, medical records were obtained from 200 patients managed by hospitalization and from 251 patients managed in an outpatient setting. Personal interviews were also conducted to collect information on productivity losses and out-of-pocket expenses.

Results

Nationally, we estimated about 21,600 cases of mild HAV infection annually. The mean annual treatment costs in hospital for mild HAV infection was estimated at US$3.39 million (2001 US$) (95% confidence interval [CI] = [US$3.26 million – US$3.52 million]). The total annual mild HAV infection cost to the society, including direct medical and nonmedical costs and productivity losses due to 721,440 lost work days, was estimated at US$6.26 million (95% CI [US$6.05 million – US$6.47 million]). In sensitivity analyses, the total annual cost of mild HAV infection ranged from US$4.37 million to US$24.66 million. The survey results showed that a relatively minor change in the current policy of mandatory hospitalization could result in an estimated total annual savings of US$4.62 million (2001 US$) in Kazakhstan.

Conclusion

Adoption of an outpatient management policy for cases of mild HAV infection would generate substantial cost savings to the Ministry of Health and society.     

The economic burden of treating neonates in Intensive Care Units (ICUs) in Greece

Background

Measurement of individuals' costs and outcomes in randomized trials allows uncertainty about cost effectiveness to be quantified. Uncertainty is expressed as probabilities that an intervention is cost effective, and confidence intervals of incremental cost effectiveness ratios. Randomizing clusters instead of individuals tends to increase uncertainty but such data are often analysed incorrectly in published studies.

Methods

We used data from a cluster randomized trial to demonstrate five appropriate analytic methods: 1) joint modeling of costs and effects with two-stage non-parametric bootstrap sampling of clusters then individuals, 2) joint modeling of costs and effects with Bayesian hierarchical models and 3) linear regression of net benefits at different willingness to pay levels using a) least squares regression with Huber-White robust adjustment of errors, b) a least squares hierarchical model and c) a Bayesian hierarchical model.

Results

All five methods produced similar results, with greater uncertainty than if cluster randomization was not accounted for.

Conclusion

Cost effectiveness analyses alongside cluster randomized trials need to account for study design. Several theoretically coherent methods can be implemented with common statistical software.     

Hospital costs of central line-associated bloodstream infections and cost-effectiveness of closed vs. open infusion containers. The case of Intensive Care Units in Italy

Background

Little is known about the cost recovery of primary health care facilities in Bangladesh. This study estimated the cost recovery of a primary health care facility run by Building Resources Across Community (BRAC), a large NGO in Bangladesh, for the period of July 2004 - June 2005. This health facility is one of the seven upgraded BRAC facilities providing emergency obstetric care and is typical of the government and private primary health care facilities in Bangladesh. Given the current maternal and child mortality in Bangladesh and the challenges to addressing health-related Millennium Development Goal (MDG) targets the financial sustainability of such facilities is crucial.

Methods

The study was designed as a case study covering a single facility. The methodology was based on the 'ingredient approach' using the allocation techniques by inpatient and outpatient services. Cost recovery of the facility was estimated from the provider's perspective. The value of capital items was annualized using 5% discount rate and its market price of 2004 (replacement value). Sensitivity analysis was done using 3% discount rate.

Results

The cost recovery ratio of the BRAC primary care facility was 59%, and if excluding all capital costs, it increased to 72%. Of the total costs, 32% was for personnel while drugs absorbed 18%. Capital items were17% of total costs while operational cost absorbed 12%. Three-quarters of the total cost was variable costs. Inpatient services contributed 74% of total revenue in exchange of 10% of total utilization. An average cost per patient was US$ 10 while it was US$ 67 for inpatient and US$ 4 for outpatient.

Conclusion

The cost recovery of this NGO primary care facility is important for increasing its financial sustainability and decreasing donor dependency, and achieving universal health coverage in a developing country setting. However, for improving the cost recovery of the health facility, it needs to increase utilization, efficient planning, resource allocation and their optimum use. It also requires controlling variable costs and preventing any wastage of resources.     

Adding a quadrivalent human papillomavirus vaccine to the UK cervical cancer screening programme: A cost-effectiveness analysis

Background

Intravitreal ranibizumab prevents vision loss and improves visual acuity in patients with neovascular age-related macular degeneration, but it is expensive, and efficacy beyond 2 years is uncertain.

Methods

We assessed the cost-effectiveness of ranibizumab compared with no ranibizumab over 10 years, using randomized trial efficacy data for the first 2 years, post-trial efficacy assumptions, and ranibizumab acquisition costs ranging from the wholesale price ($1,950 per dose) to the price of bevazicumab ($50), a similar molecule which may be equally efficacious. We used a computer simulation model to estimate the probability of blindness, the number of quality-adjusted life-years (QALYs), direct costs (in 2004 U.S. dollars), and cost-effectiveness ratios for a 67-year old woman. Costs and QALYs were discounted at 3% per year.

Results

The probability of blindness over 10 years was reduced from 56% to 34% if ranibizumab was efficacious for only 2 years, 27% if efficacy was maintained for a further 2 years only (base-case scenario), and 17% if visual acuity at 4 years was then sustained. It was cost-saving under all price assumptions, when caregiver costs were included. When caregiver costs were excluded, the cost per QALY for the base-case ranged from $5,600, assuming the bevazicumab price, to $91,900 assuming the wholesale ranibizumab price. The cost per QALY was < $50,000 when the cost of ranibizumab was less than $1000.

Conclusion

From a societal perspective, ranibizumab was cost-saving. From a health care funder's perspective, ranibizumab was an efficient treatment when it cost less than $1000 per dose.     

Costs and consequences of clopidogrel versus aspirin for secondary prevention of ischaemic events in (high-risk) atherosclerotic patients in Sweden

Background

Antiplatelet therapy plays a central role in the prevention of atherothrombotic events. Both acetylsalicylic acid (aspirin) and Clopidogrel have been shown to reduce the risk of recurrent cardiovascular events in various subgroups of patients with vascular disease.

Objective

To estimate the cost effectiveness of clopidogrel versus aspirin in Sweden for the prevention of atherothrombotic events based on CAPRIE trial data. The focus of this study is on two high-risk subpopulations: (i) patients with pre-existing symptomatic atherosclerotic disease; and (ii) patients with polyvascular disease.

Methods

A Markov model combining clinical, epidemiological and cost data was used to assess the economic value of clopidogrel compared with aspirin during a patient’s lifetime. A societal perspective was used, with costs stated in Swedish kronor (SEK), year 2007 values. For the first 2 years, the clinical input for the model was based on the relevant subpopulations in the CAPRIE trial. Thereafter, transition probabilities were extrapolated, taking account of increased risks related to age and to a history of events. Cost effectiveness of 2 years of therapy is presented as cost per life-year gained (LYG) and as cost per QALY. Univariate and multivariate sensitivity analyses were performed to investigate robustness of results.

Results

For patients resembling the total CAPRIE population, who were treated with clopidogrel, the expected cost per LYG was SEK217 806 and the cost per QALY was estimated at SEK169 154. For the high-risk CAPRIE subpopulations, costs per QALY were lowest for patients with pre-existing symptomatic atherosclerotic disease (SEK38 153). Using a ‘willingness-to-pay’ perspective indicated that treatment with Clopidogrel instead of aspirin in high-risk patients is associated with a high probability for cost effectiveness; 81% using a threshold of SEK100 000 per QALY and 98% using a threshold of SEK500 000 per QALY. Overall, the results appeared to be robust over the sensitivity analyses performed.

Conclusion

When considering the cost-effectiveness categorization as proposed by the Swedish National Board of Health and Welfare, clopidogrel appears to be associated with costs per QALY that range from intermediate in the total CAPRIE population to low in high-risk atherosclerotic patients.     

Cost effectiveness of chemoprevention for prostate cancer with dutasteride in a high-risk population based on results from the REDUCE Clinical trial

Background

The REDUCE trial examined whether chemoprevention with the dual 5-alpha reductase inhibitor, dutasteride, reduced risk of prostate cancer (PCa) detection on biopsy.

Objective

We examined the cost effectiveness of dutasteride compared with placebo in preventing PCa in men at increased risk as seen in REDUCE, from a US payer perspective.

Methods

A Markov model was developed to compare costs and outcomes of chemoprevention with dutasteride 0.5 mg/day or placebo with usual care in men aged 50–75 years, with serum prostate-specific antigen (PSA) of 2.5–10 ng/mL (men aged <60 years) or 3.0–10 ng/mL (men aged ≥60 years), and with a single negative prostate biopsy in the prior 6 months. The model simulated the REDUCE cohort annually through different health states over 4-, 10-year and lifetime time horizons. Risks of PCa for men receiving placebo and dutasteride were obtained from REDUCE. Rates of acute urinary retention events and benign prostate hyperplasia-related surgeries also came from REDUCE. Costs and utilities were obtained from published literature. All costs are reported in $US, year 2009 values.

Results

The model indicated that, over 10 years, dutasteride patients would experience fewer PCas (251 vs 312 per 1000 patients) at increased cost ($US15341 vs $US12316) than placebo patients. Although life-years were not substantially affected, the model calculated an increase in QALYs of 0.14 for dutasteride patients. Chemoprevention with dutasteride appeared to be cost effective, with an incremental cost per QALY of $US21 781 and cost per PCa avoided of $US50 254. The 4-year and lifetime incremental costs per QALY were $US18 409 and $US22498, respectively.

Conclusions

Despite increased cost due to taking a drug for prevention, dutasteride 0.5 mg/day may be cost effective in men at increased risk for PCa.     

Drug costs developments after patent expiry of enalapril, fluoxetine and ranitidine

Background

In order to increase price competition, government regulations focus on controlling drug costs. Drug costs after patent expiry are an area of particular interest because the substitution of branded medication with generics represents an opportunity for lowering drug costs. However, drug costs may not decrease after patent expiry, because of a lack of price competition and different national pricing systems.

Aim

The aim of this study was to investigate the trends in the use of generics after patent expiry for enalapril, fluoxetine and ranitidine and the subsequent changes, if any, in the costs of these medications.

Methods

A drug-utilisation study was performed using data from a large sample of Dutch pharmacies. Both volumes (measured as defined daily doses [DDD] per 1000 population) as well as drug costs (calculated per DDD) prior to and after patent expiry were calculated. Costs per DDD were compared using trend-line analysis. In addition, the relative market shares of the different trade channels (branded, parallel imported and generic) were compared before and after patent expiry.

Results

The costs per DDD decreased for all three drugs and, as expected, these costs decrease more rapidly after patent expiry. Significant differences in the trend lines were found for enalapril and fluoxetine.

Conclusions

Despite relatively high reimbursement prices for generics in the Netherlands, this example from the Dutch pharmaceutical market demonstrates the benefit of generic substitution for containing pharmaceutical costs, which contrasts with concerns raised by the Dutch government.     

Disease progression and costs of care in Alzheimer’s disease patients treated with donepezil: a longitudinal naturalistic cohort

Background/Aims

Improved data and methods are needed for modeling disease progression in Alzheimer’s disease (AD) for economic evaluation of treatments. The aim is to estimate prediction models for long-term AD progression and subsequently economic outcomes.

Methods

Three-year follow-up data on 435 patients treated with the cholinesterase inhibitor donepezil in clinical practise were analyzed. Regression models were estimated for long-term prediction of decline in cognitive function (ADAS-cog) and activities in daily living (ADL) ability, risk of institutionalization and costs of care.

Results

The cognitive deterioration was estimated at between 1.6 and 4 ADAS-cog points per every 6?months, increasing with disease severity. Cognitive function was an important predictor of ADL-ability, which itself was the most important predictor of the risk of institutionalization and costs of care. Combining all models in a cross-validation process generated accurate predictions of costs of care at each 6?months follow-up.

Conclusion

The proposed methods for representing AD progression and economic outcomes can be used in micro-simulation models for the economic evaluation of new treatments.