A Budget Impact Analysis of the VENTANA PD-L1 SP142 Immunohistochemistry Assay Versus the Dako PD-L1 IHC 22C3 Assay in Patients With Advanced or Metastatic Triple-Negative Breast Cancer Treated With Atezolizumab in Combination With Nab-Paclitaxel

OBJECTIVE: To estimate the budget impact determined by the adoption of two different diagnostic strategies, SP142 assay or 22C3 assay, in the identification (in terms of PD-L1 status) of patients with mTNBC eligible for treatment with atezolizumab in combination with nab-paclitaxel.METHODS: The budget impact analysis (BIA) was conducted using a budget impact model (BIM) considering the Italian National Health Service’s (iNHS) perspective. The analysis assessed only the direct medical cost (tissue biopsy, PD-L1 assay, specialist visit, pharmacological treatment with atezolizumab in combination with nab-paclitaxel) of patients with PD-L1 positive mTNBC, and management of the adverse events associated with the pharmacological treatment administered. The BIM also considered the clinical benefits (progression free survival, PFS) resulting from the drug therapy administered on the basis of the results of the post-hoc analysis of the IMpassion130 clinical trial. The BIA was conducted over a 1-year time horizon. The median cost per patient in the progression-free state was also calculated. The costs were calculated using the net ex-factory prices (cancer drugs) and regional or national tariffs (tissue biopsy, PD-L1 assay, specialist visit and adverse events management). A sensitivity analysis was conducted to evaluate the base case result.RESULTS: The SP142 assay diagnostic pathway would result in a reduction of the iNHS expenditure of approximately 5.6 million euros (-12%). Almost all of the reduction in iNHS expenditure would be determined by the lower number of patients treated (SP142: 689 patients vs 22C3: 786 patients) with immunotherapy (-€ 5,530,871). Compared with 22C3 assay, the SP142 assay shows a cost per PFS month reduction of € 736 (€ 7,010 vs € 7,746).CONCLUSIONS: The use of the SP142 assay proved to be cost-effective compared to the 22C3 assay; the SP142 assay can support the choice of the most appropriate cancer drug, maximizing the effectiveness and minimizing the waste of healthcare resources.

Cost-Effectiveness Analysis of Alirocumab in High Cardiovascular-Risk Patients in Italy

OBJECTIVE: Dyslipidemia, in particular elevated total and low-density lipoprotein cholesterol (LDL-C), results in atherosclerosis and increases the risk of cardiovascular (CV) events. Despite treatment with statins, many patients fail to reduce their LDL-C enough to optimally minimize their risk. Novel therapy alirocumab, on top of background statin therapy, resulted efficacious in lowering CV risk by reducing LDL-C levels. Aim of the present paper is to evaluate the cost-effectiveness of alirocumab in high cardiovascular-risk patients in ItalyMETHODS: A 1-year cycles Markov model was developed to evaluate the cost-effectiveness of statins at maximum dose tolerated plus ezetimibe (MDTS+E) with or without alirocumab. Target population consisted of patients with high baseline risk of CV events. Patients entered the model in stable disease and could experience a non- fatal CV event (acute coronary syndrome, elective revascularization or ischemic stroke) or die. Results from the ODYSSEY trial were used to evaluate CV risk reduction due to alirocumab add-on. Pharmaceutical, CV events, and LDL-C levels’ detection costs are considered in the analysis from the perspective of Italian National Health Service.RESULTS: Simulated cohort was 75 years old on average, 66% male, 42% diabetes mellitus and baseline LDL-C level equal to 121mg/dl. Furthermore, 96% of subjects were hospitalized in the last 12 months. Alirocumab used as an add-on to MDTS+E was more costly (€ 45,358 vs € 13,208) but more effective (8.01LY vs 6.33LY) than MDTS+E, leading to an incremental cost effectiveness ratio of € 19,158 per LY. At a willingness to pay threshold of € 30,000 per LY, alirocumab had 96% probability to be cost effective vs. MDTS+E alone. Results were relatively more favorable in the patient subset with recent CV event (<12 months from index).CONCLUSION: The results indicate that alirocumab in addition to MDTS+E is cost-effective versus MDTS+E alone in a representative cohort of high CV risk patients in Italy.

Osteoarthritis in Italy: Impact on Health-Related Quality of Life and Health Care Resources

OBJECTIVE: To determine how osteoarthritis (OA) severity correlates with self-reported outcomes relevant from the patient’s perspective in the Italian clinical setting.METHODS: Data were drawn from the 2017-18 Adelphi OA Disease Specific ProgrammeTM (DSP). Data were collected in the Italian clinical practice settings by primary care physicians, rheumatologists, orthopedists, and their patients with OA, during their regular office visits. Physicians completed information about OA-related visits to healthcare professionals (HCPs), tests/scans conducted, emergency room (ER) visits, surgeries, and OA-related treatment. Physicians also rated patients’ functioning on a 0 to 10 scale (0 = fully functional; 10 = completely impaired). Outcomes included Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score, functional limitations, treatment needs, EuroQoL-5 Dimension (EQ-5D) visual analogue scale (VAS), the work productivity and activity impairment. Descriptive statistics (numbers and percent for categorical variables; means with standard deviations [SD] for continuous variables) were used to evaluate the different variables as appropriate.RESULTS: The study population comprised 900 patients from Italy with knee (40.9%), back (38.7%), hip (27.9%), and/or shoulder (20.3%) OA. Mean age was 66.6 years with a prevalence of female (63%) patients. Patients had mild (26%), moderate (54%), severe (20%) disease severity. Patients with severe disease reported higher functional limitations, greater use of treatments, reduced quality of life, and impaired work productivity and activity. The burdens were higher among elderly and obese patients and in patients with highest pain severity score.CONCLUSIONS: The results from this cross-sectional study show the impact of OA disease severity on all dimensions of health-related quality of life (HRQoL), as well as in OA-related health care resource use.

Health Technology Assessment on the use of the Wearable Cardioverter Defibrillator in Patients with Myocardial Infarction and with ICD Explant

The objective of the present work is to conduct a Health Technology Assessment (HTA) on the use of the Wearable Cardioverter Defibrillator (WCD) in patients at risk of Sudden Cardiac Arrest (SCA) following Myocardial Infarction (MI) or with an explanted Implantable Cardioverter Defibrillator (ICD).

Clinical and Economic Rationale for the Early use of SGLT2 Inhibitors in Patients with Type 2 Diabetes

Type 2 diabetes (T2D) is a chronic disease associated with a high epidemiological and economic burden. It is associated with a high risk of developing both macrovascular and microvascular complications and cardiovascular diseases represent the main cause of mortality and morbidity in T2D patients. The economic impact of diabetes is primarily due to the cost and duration of treatment and secondary complications of diabetes and associated costs. Sodium-glucose co-transporter-2 (SGLT2) inhibitors are an effective therapy for providing a long-term improvement of glucose control, thus contributing to the long-term prevention of diabetic (particularly microvascular) complications. Furthermore, SGLT-2 inhibitors seem to lead to significant reductions in hospital admissions due to heart failure and progression of renal disease, regardless of baseline atherosclerotic risk category or history of heart failure. Evidence from randomized controlled trials, observational and pharmacoeconomic studies suggest that SGLT2 inhibitors should be considered not only in patients with established cardiovascular disease and incipient nephropathy but also in earlier stages of T2D in order to prevent the first onset of cardiovascular and renal complications and contain the cost of illness.

Analysis of Market Access Agreements in Turkey

BACKGROUND: Reimbursement agencies are increasingly adopting innovative reimbursement approaches for new and expensive technologies. Social Security Institution (SSI), Turkey`s reimbursement agency, established the Alternative Reimbursement Commission (ARC) on February 10, 2016. This study aimed to understand the implementations of market access agreements in recent years in Turkey.METHODS: Decisions of the Health Services Pricing Commission published in the official gazette , and information from the Turkey Pharmaceuticals and Medical Devices Agency, the Abroad Drug List, Health Implementation Communique published by SSI with additional lists, such as the Annex-4A List of Reimbursed Medicines and the Annex-4C Abroad Drug Price List, were used. The data was transferred to Windows Office Excel files, and a descriptive analysis was conducted and evaluated by two market access experts.RESULTS: There were 57 drugs included in the coverage of reimbursement with alternative reimbursement since the application was started in Turkey. 35 of them were added to Annex-4A and 22 of them to Annex-4C. Furthermore, 45.6% of the drugs had an Anatomic Therapeutic Chemical Classification (ATC) code of l-antineoplastic and immunomodulating agents, 28% were orphan drugs, 44 of them had a confidential discount rate, and financial-based agreement models were preferred for nearly all agreements.CONCLUSIONS: Turkey has been implementing market access agreements since mid-2016. In the past 3 years, 57 drugs have been covered for reimbursement under the new implementation. Further analysis should be conducted to understand the decision-making process involved.

Extended Half-life rFVIII for the Treatment of Hemophilia A: Drugs Consumption and Patients’ Perspective

Prophylaxis is recognized as the most effective treatment regimen for patients with severe hemophilia A. Prophylaxis with standard half-life (SHL) FVIII products requires frequent intravenous administrations, at least two-three times per week. Frequency of injections is reported as one of the major obstacles to adherence to treatment and caused impairment in quality of life (QoL) and possible clinical implications. The extended half-life (EHL) rFVIII products recently reimbursed by Italian National Health Service (NHS), give the possibility of prophylactic dosage regimens characterized by reduced administration frequency with the potential to increase adherence to therapy and to improve QoL and clinical outcomes. Based on the approved dosing regimens the minimum and maximum number of administrations per year and the annual consumption for the marketed EHL rFVIII products were estimated. Compared to Adynovi® and Elocta®, Jivi® is the drug associated with the lowest number of administrations per year while versus Esperoct® is associated to a slightly higher maximum number of administrations per year. Furthermore, Jivi® has an annual mean consumption per kg lower than Adynovi®, Elocta® and Esperoct® (-24%, -27% and -22%, respectively). The contemporary reduction of number of injections per year and the lower annual mean consumption (IU) represent important benefits for the patient. From the economic point of view, at the prices published in Italian Official Journal, Jivi® weighted average annual expenditure per patient is 26% lower than Adynovi® and 29% lower than Elocta® and a saving is possible even considering a 10% increase in Jivi®’s price per IU versus comparators. Among the EHL rFVIII concentrates Jivi® appears to be more suitable to cover patients’ needs due to the possibility to adopt different dosage regimens (up to every 7 days) and is associated with the lowest average annual consumption per patient allowing a more predictable budget forecast and overall reducing the NHS expenditure.

The Economic Burden of Different Multiple Sclerosis Courses: Analysis from Italian Administrative and Clinical Databases

INTRODUCTION: Poor specific economic information are available for the different Multiple Sclerosis (MS) courses: relapsing remitting (RRMS), secondary progressive (SPMS) and primary progressive (PPMS). This study aims to fill this gap.METHODS: A cost of illness study was conducted. Clinical information of patients treated in a major MS Center located in Lombardy, in the period 2004-2010, were linked with administrative data of Lombardy Healthcare System. We assessed the mean cost per patient-year and its association with different MS characteristics.RESULTS: The study identified 869 patients (83.9% RRMS, 8.5% SPMS, 7.2% PPMS). RRMS reported the highest cost per patient-year with a mean of € 5,623 in Expanded Disability Status Scale (EDSS) 0-3, € 8,675 in EDSS 3.5-6.5, and € 7,451 in EDSS 7-9. The PPMS patients reported the lower annual mean cost per patient in all EDSS categories. The mul-tivariate analysis reported a significant association between cost per patient-year and EDSS categories, relapse and use of Disease Modifying Therapies but not to MS courses, age and sex.CONCLUSION: This study provides a complete picture of MS courses direct costs at the different disability levels. The results can help to better understand the burden of each MS courses and the cost-effectiveness of different interventions.

Funding Innovation Thanks to Anti-TNF-α Biosimilars Uptake: The Economic Impact in Italy

INTRODUCTION: Anti-TNF-α biosimilars (ATB) hold the promise of reducing costs leading in improving access to bio-logical therapies. There is limited insight into how the savings generated by biosimilars may translate into patient benefit in other disease areas.AIMS: To assess the economic savings for Italian National Health System (NHS) due to the expansion of ATB market, together with a reduction in their price and to illustrate how this potential savings can be used by NHS to fund orphan drugs.METHODS: Trend of IMS Health monthly sell-in units (August 2016-December 2019) were used to estimate the current biologic and biosimilar market for rheumatic and inflammatory bowel disease in Italy and its evolution up to 2022. The scenario for 2019-2020 was compared with the future evolution (2021-2022) assuming an increasing uptake of biosimilars in the Italian market. Finally, it was estimated how these savings can potentially fund the treatment of orphan drugs, without increasing the Italian NHS budget.RESULTS: Italian biologic and biosimilar market remains stable in the next years (about 4 million units both in the current scenario and in the future evolution market) with a slight decreasing of less than 2%. However, according to our assump-tions, ATB market is expected to increase of about 33% in the next two years, covering 67% of the total Italian market, mostly due to biosimilar etanercept. Total savings due to biosimilars increases from € 96 million in 2019 to € 161 million in 2022 corresponding to a mean annual savings of about € 130 million. Such savings would permit funding 17.4% of the actual orphan drugs market corresponding to 2,600-4,800 new patients.CONCLUSIONS: The introduction of biosimilars in a range of rheumatic, dermatological and inflammatory bowel disease can be an opportunity to increase patient access to innovative treatments. Potential savings due to biosimilars uptake could lead to a re-allocation of economic resources to fund innovative therapies.

Corrigendum: An Integrated Management Model of Patients With Atrial Fibrillation: The Experience of the Local Health Unit Tuscany North-West

There was an error in the Figure 5 at page 11 in this Supplement by Casolo et al. [Farmeconomia. Health economics and therapeutic pathways 2019; 20(Suppl 1): 3-16; https://doi.org/10.7175/fe.v20i1S.1454]. The online version has been corrected on April 21, 2020.