Showing papers in "Farmeconomia. Health economics and therapeutic pathways in 2013"

Prevention of VTE in orthopedic surgery patients

Abstract: Venous thromboembolism (VTE) is defined as the obstruction, partial or complete, of one or more veins of deep circulation. It is a condition that can lead to a deterioration in his state of health until death, manifesting as deep vein thrombosis (DVT) or pulmonary embolism (PE). The major orthopedic surgery and the surgical oncology are frequently associated with thromboembolic complications, because of conditions that are often critical in these patients. It is estimated that in Italy DVT has an incidence that varies between 50 and 150 new cases per 100,000 population, while the prevalence would be between 2.5 and 5%. In the absence of thromboprophylaxis, the orthopedic surgery lead to a high increased risk of VTE. In elective hip replacement, in the absence of prophylaxis, the incidence of DVT and of fatal PE is about 50% and 2% respectively. In elective knee arthroplasty the risk of venous thromboembolic complications is even higher. It is estimated that 56.2% of the costs of prophylaxis with Low Molecular Weight Heparin (LMWH) in patients undergoing major orthopedic surgery are attributable to the cost of drugs (about € 200), followed (with 44.8%) by the cost of administration (approximately € 159). The average total cost/day was estimated at € 8 per patient. In Italy, it has been estimated an annual cost for new cases between 215 and 260 million €. The clinical advantages of the New Oral Anticoagulants (NOA) appear to be substantially clear, the major concern with regard to their reimbursement is therefore linked to the financial impact, due to the higher cost per day of the NOA compared with LMWH. To this end, it was built a model of budget impact, in the perspective of the Italian NHS, from the data related to cases of major surgical orthopedic procedures and a meta-analysis on the pivotal RCT, which aims to measure the differential effects in terms of prevention of VTE. The results show that the financial impact of the NOA in the prophylaxis of major orthopedic surgery is not particularly relevant. In fact, the major pharmaceutical costs that, at national level, amount to € 10.8 mil. (€ 15.2 mil. in the case of prolonged prophylaxis in knee operations) would be more than offset by savings in terms of fewer treatments of VTE, which is based on the assumption of more than 4,000 cases, up to about 6,600 in hypothesis best efficacy.

Direct medical costs of COPD diagnosis and treatment, Eastern vs Western European country – examples of Serbia and Belgium

Abstract: OBJECTIVE: Comparison of COPD financial burden and underlying factors, between Eastern upper middle income and a Western European high income, healthcare settings. METHODS: The patient sample was 433 in Belgium and 322 in Serbia, age ≥ 40, with spirometry and clinically confirmed COPD diagnosis. Belgian trial followed patients prospectively during 2006, using structured survey of clinicians in charge. Serbian trial conducted in 2008, retrieved data from clinical invoice database. Time horizon was one year and perspective of third party payers was taken into account for both studies. Clinical outcomes of interest were disease exacerbation, hospital admission and death. Economic inputs referred to COPD-attributable medical services consumption value during observed period of time. RESULTS: Average annual cost was 1,812.84 € for the Serbian patients and 1,738.13 €/year for the Belgian patients (not including the value of laboratory diagnostics or imaging techniques). Severity grade and duration of hospital admissions significantly directly correlated with overall cost in both populations. Pattern of diagnostic procedures requested and ATC classes of drug consumed to treat COPD remains similar and comparable in both countries. GDP per capita ratio in respective years (10.4: 37.4), exhibits the paradox of patient being much less affordable to treat in a less developed society. CONCLUSIONS: Burden of COPD in Europe is huge and, due to contemporary life style expected to grow further. We compared cost of illness structures between two societies with different macroeconomic past in healthcare financing and management. According to our findings, direct medical costs were driven by exacerbations and hospital admissions. Significantly cheaper human labor caused higher relative relevance of drug acquisition expenses in the East and higher portion of hospital admission costs in the West. More in-depth research of indirect COPD attributable costs (e.g. lost productivity, absenteeism, premature death etc) will be needed in future. It implies serious health policy necessities to provide accessibility of care.

Role of pharmacoeconomics in developing countries

Abstract: Farmeconomia. Health economics and therapeutic pathways 2013; 14(1): 3-5ROLE OF PHARMACOECONOMICS IN DEVELOPING COUNTRIESMost developing countries lack policies that encourage the use of economic evaluations in medicine selection, be it for public funding, prioritization of aid or health insurance. In addition, relevant guidelines for reporting pharmacoeconomic analyses are not availa-ble in the majority of countries. Most deve-loping countries do have National Essential Medicine Lists to guide procurement and do -nation of medicines in the public sector [5]. However, the extent to which they employ the WHO’s evidence-based approach or other guidelines advocating the use of economic evaluation criteria in drug selection has not been established. Studies conducted by Mori et al. [6] and Gavaza et al. [7-9] in a number of countries in East, South and West Africa have documented a very low availability of pharmacoeconomic studies. Some of these studies were too poor in quality and narrow in scope to be appropriate as evidence to in-form decision-making. A similar trend has also been reported for developing countries in Asia [10-12].Another important observation is that, even when pharmacoeconomic studies are availa-ble, they are not systematically or consistently applied in decision-making [6,13]. Experien-ce shows that many pharmacoeconomic stu-dies were conducted long after the decisions had been made, sometimes simply to justify or disqualify those decisions, but more often for academic purposes. Cost-effectiveness studies of artemisinin-based combination therapies for malaria control, for example, were conducted in a number of countries af-ter the decisions to recommend the treatment in the respective malaria treatment guide-lines had already been made [14-16]. Low availability and inconsistent application of pharmacoeconomic data implies that phar-macoeconomics so far has had a limited role INTRODUCTIONThe high price of medicines and increasing expenditure on pharmaceuticals is a serious concern for governments in low-income countries where already over half of the population lacks regular access to essential medicines [1]. Currently, pharmaceuticals in low-income countries consume 30-60 percent of the recurrent healthcare budgets, yet the unmet need for medicines has been strongly associated with insufficient public spending on pharmaceuticals [2]. Poor fa-milies mostly rely upon public healthcare systems, where shortages of drugs are ram-pant [3]. In this situation the use of pharma -coeconomic principles is inevitable when an objective is to ensure that the limited re-sources are efficiently spent on drug thera-pies with large potentials to improve health. While developing countries commonly use National Essential Medicine Lists to prio-ritize the allocation of scarce public fun-ds between drug therapies, little is known about the role of pharmacoeconomics in such decisions. In this editorial we there-fore examine how pharmacoeconomics has influenced prioritization decisions between drugs in developing countries.WHAT IS PHARMACOECONOMICS?Pharmacoeconomic analysis involves the identification, measurement, valuation and comparison of costs and outcomes of alter-native drug therapies. The underlying aim is to maximize treatment outcomes within limited budgets [4]. Due to the high alterna-tive costs of scarce funds, it may be argued that pharmacoeconomics is relatively more important in developing countries than in developed countries. By alternative costs in this context we mean the health losses from making sub-optimal decisions.

Impact of dose rounding of cancer therapy on cost avoidance: a pilot study

Abstract: BACKGROUND: A significant and progressive cost rising in medical oncology due to the incorporation of novel and highly expensive drugs into clinical practice have been seen in the past ten years. Dose rounding is an option might be used in oncology settings to avoid extra cost. The purpose of this project is to determine the theoretical cost saving related to a dose rounding process for biological and chemotherapy agents in adult oncology settings and to determine the opinion of oncologists about dose rounding. MATERIALS AND METHODS: Data was obtained prospectively during April 2011. All chemotherapy and targeted therapy orders prescribed in adult oncology outpatient clinics as well as in-patient wards have been collected. We considered rounding to an amount within 15% for targeted therapy and 10% for cytotoxic drugs. Chemotherapy dosing was calculated according to body surface area. Prescriptions that include cancer therapy in doses that might be rounded according to study criteria were identified. RESULTS: Two hundred and thirty three orders of chemotherapy and targeted therapy were processed by Adult Oncology Satellite Pharmacy during the period of data collection. Forty percent of the collected prescriptions fulfilled the criteria. The potential cost savings from dose rounding per year was $192,800. Data was extrapolated from the determined monthly cost savings. The highest cost saving was for breast cancer orders $80,820 (42%), followed by colorectal cancer $47,965 (25%), while in non-Hodgkin's lymphoma cost savings was $ 45,107 (23%) and for other types of cancer that include non small cell lung cancer, prostate and ovarian cancer, in addition to head and neck cost savings was $18,867 (10%). CONCLUSIONS: Our experience confirms the significant cost savings of cancer therapy by applying dose rounding to chemotherapy and biologic drugs prescriptions. While clinical impact of the suggested percentage needs to be evaluated.

ISPOR 2013: what's new and what's old?

Abstract: database relazionali). La disponibilità dei big data, al di là dei vincoli tecnici, apre possibilità per realizzare nuove analisi ed estrarre nuova informazione nei più svariati campi, dalla sanità alle scienze sociali. L’utilizzo dei big data per produrre dati istituzionali presenta però varie criticità legate alla complessità di gestione e alla natura “ufficiale” dei prodotti forniti, oltre a problemi legati all’affidabilità, non sempre chiara, della sorgente e la necessità di fasi preliminari di filtraggio e pulizia. Tuttavia, l’impressione emersa a Dublino, dai vari dibattiti, è quella che le potenziali opportunità fornite dai big data sono tali da spingere a fronteggiare le sfide elencate. Questa sedicesima edizione dell’ISPOR conferma anche la crescita d’importanza dei dispositivi medici in termini di Health Technology Assessment, al punto da essere quasi equiparabili ai medicinali. Una differenza sostanziale rispetto a questi ultimi è pero da ricercare nella mancanza, in molti paesi fra cui il nostro, di un sistema regolatorio per i dispositivi medici: attualmente il loro mercato è stato spesso paragonato a una giungla. In epoca di P4 Medicine (Personalized, Predictive, Preventive, Participatory) emerge però chiara la necessità di incrociare questi due mondi sia dal punto di vista del loro utilizzo che della loro valutazione: per esempio l’FDA valuta e approva i companion diagnostic insieme con i farmaci biologici di cui testano la suscettibilità. Accanto a questi spunti che aprono nuovi ambiti di ricerca abbiamo trovato anche vecchi temi, primo fra tutti il tema dei QALY che con i loro pro e contro, difesi o criticati dalla comunità economico-sanitaria rimangono indiscutibilmente il caposaldo delle analisi farmacoeconomiche. Un tentativo di critica costruttiva è da individuarsi in chiusura di congresso in un acceso dibattito tra Karl Claxton, uno dei volti più noti dell’ISPOR, e Rob Baltussen; il primo fervido sostenitore dei QALY, il secondo promotore della multi-criteria decision analysis (MCDA), una Il congresso europeo dell’International Society For Pharmacoeconomics and Outcomes Research (ISPOR) è un appuntamento fisso ormai da anni per noi di AdRes, come per la maggior parte dei ricercatori di settore, delle agenzie di consulenza, delle CRO e delle aziende farmaceutiche, soprattutto nelle figure di chi si occupa di market access. Come in ogni congresso, si assiste al sottile equilibrio fra l’aspetto scientifico e quello puramente commerciale, esattamente come fra lo scambio intellettuale che ne può derivare e il semplice desiderio della pubblica relazione, dell’incontrare partner o clienti, già fidelizzati o soltanto potenziali, del business. La motivazione che ha spinto noi a partecipare, quest’anno come in passato, oltre a quella legata alla presentazione dei nostri lavori più interessanti prodotti durante l’anno precedente, è da ricercare nella possibilità fornita dall’ISPOR di scoprire le tendenze correnti per individuare con tempismo le nuove domande che la farmacoeconomia, prima, e il mercato, dopo, ci pongono, insieme ad eventuali nuove metodologie e i campi in cui applicarle più efficacemente. L’obiettivo della XVI edizione dell’ISPOR, che si è tenuto dal 2 al 6 novembre 2013 al The Convention Center di Dublino, è stato efficacemente riassunto con lo slogan “Finding the Right Pieces for the Health Care Decision-Making Puzzle”. A giudicare dalle relazioni a cui è stato dato maggior rilievo, i pezzi mancanti per risolvere questo annoso puzzle sono stati individuati nella necessità di valorizzare la prospettiva dei pazienti nelle scelte economiche e di implementare modelli realistici tramite l’utilizzo di database aggiornati su campioni rappresentativi di pazienti. La quantità di dati generata ogni giorno nel mondo ha infatti un ordine di grandezza impressionante. È stato recentemente coniato il termine big data, proprio al fine di identificare insiemi di dati strutturati o destrutturati che, per le loro dimensioni, non sono gestibili utilizzando i comuni sistemi software (come i EDITORIAL

Results of reference pricing and reimbursement discount rate schemes of Turkey

Abstract: OBJECTIVES: General Directorate of Pharmaceuticals and Pharmacy (IEGM) is responsible for setting all prices for human medicinal products. The reference pricing system is used for setting these prices. Reference countries are reviewed annually and may be subject to certain alterations. There were 5 reference countries in 2009: Spain, Italy, Germany, France and Greece. The aim of this study is to show the distribution of reference countries which were used for reference pricing. METHODS: The price list of pharmaceuticals which was published by IEGM on 15.04.2011 was used for analysis. Distribution of reference countries and prices were evaluated. RESULTS: Prices of 6,251 generic and 3,703 original products were set according to the price list. 5,283 of generics and 3,306 of originals were in the positive list for reimbursement. Reference pricing was used for 2,352 generics and 2,281 originals. Prices of the remaining were set outside of reference pricing. 32 different countries were used for reference pricing. Italy was the most popular country for reference pricing. Even if it was not a reference country, Germany was used in some of the pharmaceuticals. The average reimbursement discount rate and price were 24.43% and 249 TL, respectively. There were no colerations between price and reimbursement discount rate, or reference country and reimbursement rate. CONCLUSION: It has been shown that Italy has the highest impact on the pricing of all pharmaceuticals in Turkey. Even if it was not a reference country, Germany showed to affect pharmaceuticals more than other countries which were also not used for reference pricing. Even if reimbursement discount rates are stated by the Social Security Institution (SGK), there are different discount rates for pharmaceuticals. The analysis stated that there were correlation between price, country and discount rates. This analysis is first for the literature. Further analysis is necessary in the light of price changes and newly launched pharmaceuticals.

Valutazione di efficienza nella somministrazione dell’ormone della crescita (GH)

Abstract: Treatment with growth hormone (somatropin) is effective in six different medical conditions: growth hormone deficiency (GHD), Turner syndrome (TS), growth retardation in children born small for gestational age (SGA), Prader-Willi syndrome (PWS), growth retardation due to chronic renal insufficiency (CRI), growth retardation associated with a deficiency of the gene SHOX (Short Stature HOmeoboXContaining gene). The treatment proved to be also effective in adults who have an impaired growth hormone (acquired in adulthood or childhood). The growth hormone (GH) is generally cost effective and, therefore, is usually reimbursed by public health services. In financial terms, GH is a major cost item for health systems. According to the Report OSMED 2010, GH ranks first in Italy between systemic hormonal preparations, excluding sex hormones, distributed by public system, with an annual value of approximately € 88 million (+ 12.7% compared to 2009). Considering the increasing need to control pharmaceutical expenditure, there is a strong interest for the efficient supply of the hormone by the regional health service. From this point of view, the comparison of the specialties on the market is normally carried out on the basis of the cost per mg; this approach, also used in the main studies of cost-effectiveness, is derived from a logic of cost minimization, but it may still be distorting, not taking into account the efficiency of devices used for the administration of the hormone. The proposed analysis verifies the efficiency of different available devices, evaluating the potential waste of product, depending both on the device used and on the characteristics of the population exposed to the treatment. Only in the case of single-dose and disposable formulations is theoretically possible to have zero waste and thus an equivalence between the actual cost of the treatment and the price charged. In other cases, the inefficiency causes a deviation between the actual cost and price. In the latter cases, since the theoretical amount of the population exposed to the treatment, it is estimated that the total share of potentially unused product can vary between 208,000 mg / year to 750,000 mg / year, depending on the device used. In particular, there is an actual average cost of treatment ranging between +9.9% and +11.4% of the ex-factory price; depending on the different doses and even between the different devices, the difference between the actual price and the theoretical price varies from a minimum of +6.9% and a maximum of +18.7%.

Can market structure explain cross-country differences in health?

Abstract: There is a well documented health disparity between several European countries and the United States. This health gap remains even after controlling for socioeconomic status and risk factors. At the same time, we note that the U.S. market structure is characterized by significantly more large corporations and "super-sized" retail outlets than Europe. Because big business is hierarchical in nature and has been reported to engender urban sprawl, inferior work environments, and loss of social capital, all identified as correlates of poor health, we suggest that differences in market structure may help account for some of the unexplained differences in health across Europe and North America. Using national level data, this study explores the relationship between market structure and health. We investigate whether individuals who live in countries with proportionately more small business are healthier than those who do not. We use two measures of national health: life expectancy at birth, and age-standardized estimates of diabetes rates. Results from ordinary least squares regressions suggest that, there is a large and statistically significant association between market structure (the ratio of small to total businesses) and health, even after controlling income, public percent of health expenditure, and obesity rates. This association is robust to additional controls such as insufficient physical activity, smoking, alcohol disease, and air pollution.

Costs of treatment of haemophilia A in Italy: comparison of the use of plasma-derived and recombinant FVIII using a discrete event simulation (DES) model

Abstract: OBJECTIVE: To simulate haemophilia A (HA) real-life management and compare the cost of different treatment strategies, both with plasma-derived and recombinant factor VIII (pdFVIII and rFVIII, respectively), from the perspective of the Italian NHS. METHODS: A discrete event (micro-)simulation (DES) model was developed to reproduce every possible HA patient clinical pathway: on-demand (OD) treatment of bleeding, continuous or discontinuous prophylaxis (PRO) with FVIII, inhibitors-tolerance-induction treatment (ITI), surgery in case of severe disability. Patient characteristics, treatment indications and disease evolution were modeled basing on data available in clinical literature in order to represent the actual state of art of HA management. In addition to the baseline scenario, reproducing current HA management, alternative strategies were simulated to explore the impact on the cost borne by the Italian NHS for these patients. Only differential direct sanitary costs were considered in the simulation, with a 3.5% discount rate. RESULTS: Baseline scenario results show difference between patients treated with pdFVIII and those treated with rFVIII: mean lifetime HA patient management cost was estimated at € 1,332,373 with pdFVIII treatment, compared with € 2,013,222 for rFVIII. The saving is due mainly to the lower acquisition cost of pdFVIII. Total medical costs are strongly and positively correlated with HA severity: cost per patient increases from € 86,269 (mild HA) to € 1,509,231 (severe HA) for patients treated with pdFVIII and from € 147,900 to € 2,621,540 in patients treated with rFVIII. All analyses conducted in the study lead to the conclusion that the use of pdFVIII is much less expensive than rFVIII, but therapeutically equivalent. CONCLUSION: Management of HA patients is complex and difficult to optimize; although involving a limited number of patients, lifetime management costs for the Italian NHS are extremely high. The main advantage of this model lies in the capability of estimating the economic impact of different strategic choices and economic/regulatory constraints.

Treatment cost of metastatic colon cancer in Turkey

Abstract: OBJECTIVES: Colon cancer is the third most common in the top cancer incidence list in Europe. In Europe 212,000 patients die every year due to colon cancer. In Turkey 120,000-130,000 new cancer patients are diagnosed every year, 7.1% of whom are diagnosed to have developed colon cancer. Metastases will occur in up to 50% of the patients who are newly diagnosed. Survival appears to be further prolonged to more than 20 months with new pharmaceuticals; however, these new pharmaceuticals increase the total cost of care. The aim of this study is to estimate the cost implications of new colon cancer treatment options for Turkey. METHODS: Gazi University Hospital treatment protocols for colon cancer treatment were used. Cost of FUFA (5 FU/LV), FOLFIRI, FOLFOX, bevacizumab/FUFA, bevacizumab/FOLFIRI, bevacizumab/FOLFOX, irinotecan and irinotecan/cetixumab protocols were calculated. The cost of combination of protocols were calculated depending on a Markov analysis. The exchange rate was US$ 1 for TL 1.5. RESULTS: Depending on the life expectancy the lowest total cost was established by FUVA (US$ 5,359). It was followed by FOLFIRI then FOLFOX and FOLFOX, US$ 14,144 and US$ 16,553, respectively. The lowest cost for each week of life expectancy was established by FUVA with US$ 98. CONCLUSIONS: Only FUFA, FOLFIRI followed by FOLFIX, FOLFIRI/bevacizumab then FOLFOX then cetuximab, FOLFOX/bevacizumab then irinotecan then cetuximab/irinotecan and FOLFIRI/bevacizumab then FOLFOX then cetuximab/irinotecan were under the cost effectiveness curve. In addition no treatments ICER was under the WHO`s threshold for Turkey, except FOLFIRI then FOLFOX compared with FUVA.

International differential pricing: easy in theory but hard in practice

Abstract: of products) and base the price in their own country on those observations. For example, Slovakia takes the second lowest price in the EU and makes price revisions twice a year. The tradeoffs between the two pricing schemes is clear: evidence-based pricing requires exploring the value of a drug while in some way attempting to establish a price based on a society’s willingness-to-pay for the drug, and reference pricing is a system that tends to drive prices to a common minimum – the logic often being that such a determination is a fair value as companies are selling at these prices in other countries. This provides an opportunity for the countries to bypass socio-economic development factors altogether; research suggests that drug prices do not vary based on the macroeconomic development factors of each market, which suggests reference pricing is the more common system [1]. International reference pricing mechanisms have largely stayed within the realm of list prices and as a result, list prices tend to show a downward trend in these countries over time. The discounts and rebates offered at national, regional, or local levels by pharmaceutical manufacturers have largely stayed “invisible” and therefore, did not enter into reference calculations. However, recent trends are making some of these off-list prices more transparent. Germany has been able to implement a system where a mandatory rebate will be enforced in a way that it is there for everyone to see. Such off-list price arrangements are, however, becoming increasingly difficult to maintain as countries and HTA agencies are requiring more rigorous reporting on how net prices are determined. More and more countries are expected to go beyond list prices and begin to look at net prices as a better proxy of the real prices in their referenced markets. Considering these trends, it is becoming increasingly difficult for manufacturers to maintain differential prices across countries. The evolving nature of reHow countries pay for patented pharmaceuticals varies widely in terms of complexity of rules and processes. A pharmaceutical pricing scheme should ideally provide affordable drug access to those in need while allowing the manufacturers to receive enough profits to sustain continued technological innovation. Profit-seeking pharmaceutical companies are incentivized to set prices in a way that would maximize their revenues and sustain long term dominance of specific market segments and they understandably attempt to justify these practices as a necessity to cover their R&D investments. Health authorities, on the other hand, typically have a current budget constraint under which they have to work and have some incentive to discount the value of future innovation. While some countries have allowed “free pricing”, others have introduced concepts such as “value-based pricing” or enforced rigid price controls. As one looks across various country markets, there is a range of practices that fall somewhere along this continuum. For the current discussion, our focus is on the practice of value-based pricing – focusing on the diversity between evidence-based pricing and reference pricing approaches. We are, in particular, seeing emerging new pressures that are beginning to have an impact on some of these long-standing practices. For all practical purposes, we might remove the so-called “free pricing” countries from this thought exercise as this as a concept is a dying breed outside of the US. Similarly, we have not attempted to consider countries that are under some form of price control. In evidence-based pricing, countries may establish health technology assessment (HTA) organizations in hopes of creating a systematic and transparent framework for evaluating the prices of drugs in terms of their outcomes. At the other end of the spectrum, reference pricing countries scan prices in other health systems (and in cases where a domestic referencing is applied, scan prices in the basket EDITORIAL

Venous thrombo-embolism (VTE) prevention of patients undergoing total hip and knee replacement: budget impact analysis of apixaban in Italy

Abstract: BACKGROUND: Venous thromboembolism (VTE) is a common and burdensome cardiovascular condition, frequently leading to severe complications and requiring high-cost healthcare interventions. New oral anticoagulants (nOACs) have demonstrated to be efficacious and safe in VTE prevention of patients undergoing total hip replacement (THR) and total knee replacement (TKR), a condition that is typically associated to cardiovascular disease. The Italian Healthcare Service (SSN) has recently approved the latest nOAC, apixaban. The present article aims to evaluate its economic impact in the perspective of the Italian SSN. METHODS: We conducted a budget impact analysis to estimate clinical outcomes and economic consequences associated to the reimbursement of apixaban, in the prevention of VTE as a consequence of major orthopedic surgery, over a three-year time horizon. In our analysis we compared two alternative scenarios, with apixaban either reimbursed (Scenario B) or not reimbursed (Scenario A) by the Italian SSN, and estimated the difference of healthcare costs between the two scenarios. Only direct healthcare costs have been considered. RESULTS: According to market assumptions, it is estimated that 1.2%, 3.7%, and 6.5% of THR patients, and 1.2%, 3.8% and 6.7% of TKR patients, would be treated with apixaban over the first three years since launch. At the estimated daily cost of apixaban (€2.48/die), this would translate into a budget impact of €14.3 mln, €45.5 mln, and €81.4 mln at years 1, 2 and 3 since launch, respectively. This expenditure would be more than offset by savings, due to: i) reduction of prescriptions of alternative treatment options (other nOACs, low-molecular weight heparins, fondaparinux); ii) reduction of the economic burden attributable of CV complications of VTE. Finally, Scenario B resulted slightly favourable compared to Scenario A, leading to economic savings for about €50 thousands over three years. Sensitivity analyses confirmed findings of the base-case analysis. CONCLUSIONS: Reimbursement of apixaban does not determine a budget impact increase for Italian SSN. Its usage may be considered fully sustainable from a pharmaco-economic viewpoint.

The utility of a model-based cost-effectiveness analysis of degarelix versus leuprolide in the therapy of hormone-dependent advanced prostate cancer

Abstract: INTRODUCTION: Prostate cancer (PC) is a very common tumor among men: in Italy its prevalence in 2006 was 0.9%. Androgen deprivation therapy is a way to treat hormone-responsive PC by decreasing testosterone levels. GnRH-analogues, including GnRH-agonists and GnRH-antagonists, are effective for this purpose. AIM: This article presents a cost-effectiveness analysis based on a semi-Markov model comparing the GnRH-antagonist degarelix and GnRH-agonist leuprolide in the treatment of hormone-dependent advanced prostate cancer from the perspective of the Regional Health Service in Veneto Region (Italy). MATERIALS AND METHODS: Effectiveness data were retrieved by a 12-month phase III non-inferiority clinical trial, comparing degarelix and 7,5 mg leuprolide in 610 patients treated for hormone-dependent prostate cancer. Epidemiological data came from a national database and were referred to Veneto Region. The values of the healthcare resources were calculated using regional and national prices (€ 2012). The model considers 3 exhaustive and mutually exclusive health status: first-line treatment, further-lines treatment and death. It lasts 10 years, with 28 days per cycle. The entry in the model is hypothesized at the age of 70 (the age with most PCs in Veneto Region). Effectiveness endpoints were life years saved and quality-adjusted life years, using 3% social discount rate. The incremental cost per QALY was related to the range of acceptability proposed by the Associazione Italiana di Economia Sanitaria (€ 25,000-40,000). The budget impact was calculated on a 5-year time horizon. Univariate and probabilistic sensitivity analyses were performed on every hypothesis of the model. RESULTS: Degarelix resulted in minor costs if compared to 7.5 mg leuprolide (€ 20,511.64 vs 22,256.49). The cost-driver was chemotherapic care (32.45% degarelix vs 44.30% 7.5 mg leuprolide). Life years saved were the same for both the alternatives (5.58), while QALYs obtained were higher in degarelix vs. 7.5 mg leuprolide (4.41 vs. 4.10). QALY better data probably could results from greater delay to disease progression in castrate resistant phase with degarelix than comparator and also due to superior symptoms relief. Therefore degarelix is dominant compared to the agonist. The probability for degarelix to be cost-effective increases with the increasing of the threshold for incremental QALY, being 69.95%, 93.76%, 95.55%, and 97.42% for threshold values equal to € 0, € 25,000, € 40,000, and € 100,000, respectively. The use of degarelix in Veneto Region instead of 7.5 mg leuprolide would result, after a five-year period, in total savings for the Regional Health Service equal to € 4,783, considering the treatment of 259 patients. CONCLUSIONS: In the treatment of hormone-dependent advanced prostate cancer PC, degarelix is thought to be an economically rational investment of resources for the Regional Health Service of Veneto Region because it’s dominant, in term of cost-effectiveness, to the comparator (agonist) thanks to superior QALY and reduced costs.

A cost-minimization analysis of combination therapy in hypertension: fixed-dose vs extemporary combinations

Abstract: BACKGROUND: Cardiovascular disease management and prevention represent the leading cost driver in Italian healthcare expenditure. In order to reach the target blood pressure, a large majority of patients require simultaneous administration of multiple antihypertensive agents. OBJECTIVE: To assess the economic impact of the use of fixed dose combinations of antihypertensive agents, compared to the extemporary combination of the same principles. METHODS: A cost minimization analysis was conducted to determine the pharmaceutical daily cost of five fixed dose combinations (olmesartan 20 mg + amlodipine 5 mg, perindopril 5 mg + amlodipine 5 mg, enalapril 20 mg + lercanidipine 10 mg, felodipine 5 mg + ramipril 5 mg, and delapril 30 mg + manidipine 10 mg) compared with extemporary combination of the same principles in the perspective of the Italian NHS. Daily acquisition costs are estimated based on current Italian prices and tariffs. RESULTS: In three cases the use of fixed‑dose combination instead of extemporary combination induces a lower daily cost. Fixed combination treatment with delapril 30 mg + manidipine 10 mg induces greater cost savings for the National Health System (95,47 €/pts/year), as compared to free drugs combination therapy. CONCLUSIONS: Compared with free drug combinations, fixed‑dose combinations of antihypertensive agents are associated with lower daily National Health Service acquisition costs.

The use of dexmedetomidine in intensive care sedation

Abstract: The goals and recommendations for ICU (Intensive Care Unit) patients’ sedation and analgesia should be to have adequately sedated patients who are calm and arousal, so that they can guarantee a proper evaluation and an adequate control of pain. This way, it is also possible to perform their neurological evaluation, preserving intellectual faculties and helping them in actively participating to their care. Dexmedetomidine is a selective alpha-2 receptor agonist, member of theraputical cathegory: “other hypnotics and sedatives” (ATC: N05CM18). Dexmedetomidine is recommended for the sedation of adult ICU patients who need a sedation level not deeper than arousal in response to verbal stimulation (corresponding to Richmond Agitation-Sedation Scale 0 to -3). After the EMA approval, some European government authorities have elaborated HTA on dexmedetomidine, based on clinical evidence derived from Prodex and Midex trials. Dexmedetomidine resulted to be as effective as propofol and midazolam in maintaining the target depth of sedation in ICU patients. The mean duration of mechanical ventilation with dexmedetomidine was numerically shorter than with propofol and significantly shorter than with midazolam. The resulting favourable economic profile of dexmedetomidine supported the clinical use in ICU. Dexmedetomidine seems to provide clinical benefits due to the reduction of mechanical ventilation and ventilator weaning duration. Within the present review, an economic analysis of costs associated to the use of dexmedetomidine was therefore performed also in the Italian care setting. Thus, four different analyses were carried out based on the quantification of the total number of days in ICU, the time spent on mechanical ventilation, the weighted average number of days with mechanical ventilation or not and TISS points (Therapeutic Intervention Scoring System). Despite the incremental cost for drug therapy associated with dexmedetomidine, a reduction of the management costs for ICU has been estimated, with savings ranging between € 800 and € 1,400 per patient.

Linked routine data to enhance health-economics analysis

Abstract: Muhammad Jami Husain 1, Sinead Brophy 2, Stefan Siebert 3, Ceri J. Phillips 4 1 Keele Management School, Keele University, Staffordshire 2 College of Medicine, Swansea University, Wales 3 Institute of Infection Immunity and Inflammation, College of Medical, Veterinary and Life Sciences, University of Glasgow, Glasgow 4 College of Human and Health Sciences, Swansea University, Wales Farmeconomia. Health economics and therapeutic pathways 2013; 14(2): 47-49

European and Italian regulation on orphan medicinal products

Abstract: The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

First-line HIV treatment: evaluation of backbone choice and its budget impact

Abstract: OBJECTIVE: The gradual increase of persons living with HIV, mainly due to the reduced mortality achieved with effective antiretroviral therapies, calls for increased rationality and awareness in health resources consumption also during the early illness phases. Aim of this work is the estimation of the budget impact related to the variation in backbone prescribing trends in naive patients. METHODS: Target population is the number of patients starting antiretroviral therapy each year, according to the Italian HIV surveillance registry, excluding patients receiving non-authorized or non-recommended regimens. We modeled 3-year mortality and durability rates on a dynamic cohort, basing on international literature. A prevalent patients analysis has also been conducted, for which the model is fed by a closed cohort consisting of all the patients without experience of virologic failure. The aim of this collateral analysis is to estimate the difference in current annual expenditures if the past prescription trends for patients starting therapy would have led to the evaluated hypothetical scenarios. Current Italian market shares of triple regimens containing first-choice or alternative backbones (tenofovir/emtricitabine, abacavir/lamivudine, tenofovir/lamivudine and zidovudine/lamivudine) are compared to three hypothetical scenarios (base-case, minimum and maximum) in which increasing shares of patients eligible to abacavir/lamivudine start first line treatment with this backbone. Annual cost for each regimen comprises drugs acquisition under hospital pricing rules, monitoring exams and preventive tests, valued basing on regional reimbursement tariffs. RESULTS: According to current prescribing trends, in the next three years about 13,000 patients starting HIV therapy will receive tenofovir/emtricitabine (83% of the target population), and minor portions other regimens (9% abacavir/lamivudine, 8% zidovudine/lamivudine). Patients that would be eligible to abacavir/lamivudine are 1.5, 4.5 and 6 thousand more than those presently treated according to the three hypothetical scenarios, leading to a cumulative saving of 850 thousand, 2.4 million and 3.3 million euro, respectively. If in the past the same modification of first line prescription trend was adopted, the annual current cost saving would vary from 922 thousands to 7.3 million euro. Most of this amount is due to reduced acquisition costs and, secondarily, to lower monitoring needs. CONCLUSION: Where patient features don’t force the choice of the backbone, abacavir/lamivudine prescription may induce substantial savings, allowing the release of resources needed to manage more complicated/advanced cases.

Economic evaluation of the chronic hepatitis B treatment strategies in Italy

Abstract: BACKGROUND AND OBJECTIVE: Pharmacological approaches available in chronic hepatitis B (CHB) are based on 48-weeks finite course of peg-interferon (PEG-IFN) or continuous administration of nucleoside analogues. Recent studies gave way to early identification of non-responders to peg-interferon with a stopping rule based on virologic/serologic markers at week 12. A pharmacoeconomic simulation model was developed to support the economic evaluation of HBeAg-negative CHB treatment strategies, which either involve first line peg-interferon with stopping rule and switch to current most effective analogue entecavir or tenofovir, or nucleosides analogues in first line treatment. This model showed a good cost-effectiveness profile of the first line treatment with peg-interferon. Aim of the present study was the estimation of the impact of a wide adoption of a first line with PEG-IFN and the stopping rule on total overall cost related to CHB over the lifetime of patients in Italy. METHODS: The Markov model was developed to perform a lifetime simulation of the disease progression considering the following health-related states: active CHB, virologic response, HBsAg clearance, compensated cirrhosis with active CHB, compensated cirrhosis with virologic response, decompensated cirrhosis, hepatocellular carcinoma, liver transplant, post-liver transplant and death. The outcomes provided by the model were average survival, quality-adjusted life years (QALY) and costs, calculated from the Italian National Health Service (SSN) perspective. A “current” mix of treatments, where 80% patients received ETV or TDF in first-line, was compared with an “hypothetical” mix defined by a 100% adoption of a PEG-IFN first-line with the stopping rule, to estimate the impact on total lifetime CHB-related costs for a cohort of 100 HBeAg-negative CHB patients in Italy. RESULTS: Results obtained from the evaluation of overall total treatment cost over patients’ lifetime appeared to confirm the cost-effectiveness of peg-interferon previously assessed. Lifetime overall total cost resulted significantly lower with the “hypothetical” treatment mix as compared to the “current” mix.. In fact, estimated lifetime overall total costs for the former and latter scenario were equal to €7,239,050 and €9,060,150 respectively, thus generating a saving of approximately 20% associated to CHB treatment with first-line peg-interferon. CONCLUSION: Based on the results obtained from the economic evaluation of the CHB treatment strategies, first-line peg-interferon associated with the stopping rule and switch to nucleoside analogues consistently represents a convenient strategy on both the clinical and economic perspective.