Search

STN1013001 is an innovative latanoprost cationic emulsion for open-angle glaucoma/ocular hypertension (OAG/OHT) and ocular surface disease (OSD). A 5-year, 7 health states, 1-year cycle early Markov model-supported cost-utility analysis (CUA) of STN1013001 vs. other latanoprost formulations (Latanoprost) followed the Italian National Health Service (INHS) perspective. One-way, probabilistic and scenario sensitivity analyses tested the uncertainty of the baseline results. Value of information analysis (VOIA) investigated the potential cost-effectiveness of collecting further evidence. Over 5 years, the Markov model-supported CUA predicts STN1013001 to be potentially highly cost-effective vs. Latanoprost (+€57.60 cost at €2020 values; +0.089 Quality-Adjusted Life Years). The Incremental Cost-Utility Ratio (€647.65) falls well below the lower limit of the acceptability range proposed for Italy (€25,000-€40,000). Sensitivity analyses confirmed the robustness of the baseline findings. VOIA highlighted that further information might only be cost-effective for OAG/OHT utilities and OSD-related disutility. STN1013001 is potentially highly cost-effective and strongly dominant vs. Latanoprost for OAG/OHT+OSD patients from the INHS perspective. These findings should be re-assessed using the data from the ongoing Phase III trial (NCT04133311) comparing the efficacy and safety of STN1013001 vs. Latanoprost and with future real-world CUAs upon the availability of STN1013001 on the Italian market.

Background Multiple sclerosis (MS) accounts for 176 cases per 100,000 inhabitants (female/male ratio = 2:1) in Italy. For most of the patients (67%), the disease course is relapsing–remitting MS (RRMS). Objective To compare the costs and quality-adjusted life years (QALYs) of teriflunomide in RRMS naïve patients vs. RRMS patients previously treated (experienced) with other disease-modifying therapies in Italy. Methods A four health states Markov model-supported cost-utility analysis (CUA) covering a 7-year timespan through annual cycles was developed, following the healthcare sector and the societal viewpoints. Part of the parameters that populated the Markov model was obtained from a questionnaire administered to four primary Italian MS centres. Costs of healthcare and non-healthcare resources, expressed in euro (€) 2019, and QALYs were discounted at 3% real social discount rate. One-way, scenario and probabilistic sensitivity analyses tested the uncertainty of the baseline findings. Results Baseline CUA shows that teriflunomide in RRMS naïve patients is strongly dominant vs. experienced patients (healthcare sector perspective: − €1042.68 and + 0.480 QALYs; societal perspective: − €6782.81 and + 0.480 QALYs). Sensitivity analyses confirmed the robustness of the baseline results. Conclusion Teriflunomide in RRMS naïve vs. experienced patients is cost-effective and possibly strongly dominant from both the healthcare sector and the society viewpoints in Italy. Our findings need further confirmation from real-world studies.

Background Isavuconazole (ISA) is a frequently used antifungal agent for the treatment of invasive fungal diseases (IFDs). However, hospital reimbursement data for ISA is limited. Objectives The primary objective of this study was to analyse the different perspectives of relevant stakeholders and the (dis)incentives for the administration of ISA in Germany. To that aim, the health economic effects of using ISA from a hospital management perspective were analysed. Patients/methods Based on principal-agent theory (PAT), the perspectives of (a) the patient (principal) as well as (b) physicians, (c) pharmacists and iv. hospital managers (all agents) were analysed. For the evaluation of the cost-containment and reimbursement strategies of ISA, the German diagnosis-related group (G-DRG) system was used. Results Hospitals individually negotiating additional payments for innovative treatment procedures (zusatzentgelte [ZE]) within the G-DRG system is a key element of hospital management for the reduction of total healthcare expenditure. Our analysis demonstrated the beneficial role of ISA in healthcare resource utilisation, primarily due to a shortened overall length of hospital stay. Depending on underlying disease, coded G-DRG and ISA formulation, large differences in total reimbursement and the amount of ZE was shown. The PAT demonstrated disincentives for hospital managers to use innovative drugs. Conclusions Based on the PAT, beneficial, detrimental and indifferent perspectives of different stakeholders regarding the usage of ISA were shown. A reduction of bureaucratic hurdles is needed in Germany for the extension of effective and innovative antifungal treatment strategies with ISA.

Given the availability and efficacy of the mobilizing agent plerixafor in augmenting hematopoietic progenitor cell mobilization with granulocyte colony-stimulating factor (G-CSF), there is a strong case for comparing the cost-effectiveness of mobilization with G-CSF + cyclophosphamide versus G-CSF alone. This study investigated the cost and effectiveness (i.e., successful 4 million-CD34+ collection) of G-CSF alone versus high-dose cyclophosphamide (4 g/m2) + G-CSF mobilization (± on-demand plerixafor) in patients with multiple myeloma (MM) eligible for autograft in Italy. A decision tree-supported cost-effectiveness analysis (CEA) model in MM patients was developed from the societal perspective. The CEA model compared G-CSF alone with cyclophosphamide 4 g/m2 + G-CSF (± on-demand plerixafor) and was populated with demographic, healthcare and non-healthcare resource utilization data collected from a questionnaire administered to six Italian oncohematologists. Costs were expressed in Euro (€) 2019. The CEA model showed that G-CSF alone was strongly dominant versus cyclophosphamide + G-CSF ( ± on-demand plerixafor), with incremental savings of €1198.59 and an incremental probability of a successful 4 million-CD34+ apheresis (+0.052). Sensitivity analyses confirmed the robustness of the base-case results. In conclusion, chemotherapy-free mobilization (± on-demand plerixafor) is a “good value for money” option for MM patients eligible for autograft.

Objective To investigate costs and quality-adjusted life years of rehabilitation combined with abobotulinumtoxinA (aboBoNT-A) (rehab/aboBoNT-A) vs rehabilitation alone (rehab) in post-stroke spasticity in Italy. Design Based on both Italian National Health Service and societal perspectives, a 2-year cost-utility analysis model was performed. Subject/patients: The cost-utility analysis model considered hypothetical patients. Methods The cost-utility analysis model was populated with data concerning demographics, disease severity, healthcare and non-healthcare resource consumption. Data were collected via a questionnaire administered to 3 highly experienced Italian physiatrists (864 out of 930 post-stroke spasticity patients on rehab/aboBoNT-A in total). Costs are expressed in Euro (€) based on the year 2018. Results The cost to society (rounded to the nearest whole €) was €22,959 (rehab/aboBoNT-A) vs €11,866 (rehab). Italian National Health Service-funded cost was €7,593 (rehab/aboBoNT-A) vs €1,793 (rehab). Over a period of 2 years rehab/aboBoNT-A outperforms rehab in terms of quality-adjusted life years gained (1.620 vs 1.150). The incremental cost-utility ratio was €12,341 (Italian National Health Service viewpoint) and €23,601 (societal viewpoint). Sensitivity analyses confirmed the robustness of the baseline results. Conclusion Despite some limitations, the higher number of quality-adjusted life years gained vs rehab and the high probability of reaching a cost-utility ratio lower than the Italian informal acceptability range (€25,000-40,000) make rehab/aboBoNT-A a cost-effective healthcare programme for treating patients with post-stroke spasticity in Italy.

Background Chronic obstructive pulmonary disease (COPD) is characterised by recurring exacerbations. We estimated the costs of healthcare resources for COPD management funded by the Italian National Healthcare Service (INHS) for one year. Methods We examined the demographic, clinical, and economic variables at enrolment and follow-up visits (at 6 and 12 months) of COPD patients participating in the SAT study and referred to 20 Italian pulmonary centres with different institutional characteristics. Costs were expressed in Euro (€) 2018. A random effects log-linear panel regression model was performed to predict the average cost per patient. Results Most of the centres were public institutions (90%; public university hospital: 30%). The total average cost of COPD was €2647.38/patient and ICS/LABA/LAMA therapy contributed the most (€1541.45). The average cost was €6206.19/patient for severe COPD (+139.67% vs the cost/patient with mild or moderate COPD). The regression model showed that, others things being equal, increases in the predicted average logged cost per patient were due to liquid oxygen therapy (+468.31%), three COPD exacerbations during the follow-up (+254.54%), and ICS/LABA or ICS/LABA/LAMA associated therapy (+59.26%). Moreover, a 1.19% increment was observed for each additional score of the CAT questionnaire. Conversely, a 36.52% reduction in the predicted average logged cost was reported for hospitals managed by local healthcare authorities. Conclusions The health econometric approach is innovative in the management of COPD patients in Italy. The results of the random effects log-linear panel data regression model may help clinicians estimate INHS costs when managing COPD patients. Clinicaltrials.gov ID# NCT02689492.

Prior researches have suggested that home-based subcutaneous immunoglobulin (SCIG) is equally effective and can be less expensive than hospital-based intravenous immunoglobulin (IVIG) in treating chronic inflammatory demyelinating polyneuropathy (CIDP) patients. This economic evaluation aims at comparing costs of SCIG vs IVIG for CIDP patients in Italy. A 1-year model-based cost-minimization analysis basically populated via neurologists' opinion was undertaken from a societal perspective. Health care resources included immunoglobulin; drugs for premedication and complications (rash, headache, and hypertension) management; time of various health care professionals; pump for SCIG self-administration; infusion disposables. Non-health care resources encompassed transport and parking; losses of working and leisure time for patients and caregivers. Unit or yearly costs for resources valuation were mainly obtained from published sources. Costs were expressed in Euro (euro) 2013. An extensive one-way sensitivity analysis (OWSA) and a scenario SA tested the robustness of the base case findings. Overall costs per patient amount toeuro49,534.75 (SCIG) andeuro50,895.73 (IVIG); saving in favour of SCIG reacheseuro1360.98. For both SCIG and IVIG, the cost driver was immunoglobulin (94.06 vs 86.06 % of the overall costs, respectively). Sensitivity analyses confirmed the consistency of the baseline results. SCIG may be a cost-saving therapy for Italian CIDP patients.

Adelaide Mozzi,1 Michela Meregaglia,2 Carlo Lazzaro,3 Valentina Tornatore,4 Maurizio Belfiglio,4 Giovanni Fattore,1,2 1Department of Policy Analysis and Public Management, 2Centre for Research on Health and Social Care Management (CeRGAS), Bocconi University, 3Studio di Economia Sanitaria, Milan, 4AbbVie srl, Campoverde Di Aprilia, Latina, Italy Abstract: Weights associated with the EuroQol 5-Dimension 3-Level (EQ-5D-3L) instrument represent preferences for health states elicited from general population's samples. Weights have not been calculated for every country; however, empirical research shows that cross-country differences exist. This empirical study aims at investigating the impact of recently developed Italian weights in comparison with UK and US scores on health-related utility calculation using a sample of patients with Crohn's disease. The study is based on a survey on health-related quality of life in patients (n=552) affected by active Crohn's disease conducted in Italy from 2012 to 2013. Utilities computed through the Italian algorithm (mean: 0.76; SD: 0.20; median: 0.81) are generally higher than US (mean: 0.69; SD: 0.22; median: 0.77) and UK (mean: 0.57; SD: 0.32; median: 0.69) utilities, except for extremely severe health states where US values outweigh the Italian ones. UK preference weights generate the highest number of negative results. All the three value distributions are left-skewed due to very low scores associated with the most serious health states (ie, three or four levels equal to 3). As expected, despite the tariff set considered, more severe disease (Harvey Bradshaw Index >16) reduces the mean conditional EQ-5D-3L index (P16) reduces the mean conditional EQ-5D-3L index (P

New therapeutic options have modified the natural history and health care costs of multiple sclerosis (MS). An epidemiological 25 years-long model-based cost-utility analysis was performed following the Italian National Health Service (INHS) and societal perspectives to compare costs and quality-adjusted life years of treatment with Interferon beta-1b (IFNB-1b) from diagnosis of clinically isolated syndrome (CIS) versus treating at subsequent conversion to clinically definite MS (CDMS). Among patients treated (untreated) with IFNB-1b from CIS diagnosis, 40,420 (43,700) converted to CDMS after 25 years; the estimated cumulative probability of converting to CDMS during the first 3 years was 72.90% (84.94%) (P0.0001). Early treatment with IFNB-1b is highly cost-effective for the INHS (incremental cost-effectiveness ratio: Euros 2,574.94) and dominant from the societal viewpoint. Sensitivity analyses confirmed the base case findings. Early treatment with IFNB-1b delays conversion to CDMS in CIS patients and might be a "good value for money" health care programme.

Objectives: To evaluate and compare healthcare-related costs and results on patients’ health state of anastrozole 1mg per os per diem and tamoxifen 20 mg per os per diem for adjuvant treatment in postmenopausal women with early breast cancer.

AIM: To perform an empirical, single-centre, retrospective and secondary cost of illness (COI) study of advanced ovarian carcinoma (AOC) in Italy. METHODS: Demographic, clinical, health care and non-health care resource consumption data concerning a convenience sample of subsequent patients in 1st line of treatment (100 patients), 2nd line of treatment A (surgery + chemotherapy; 30 patients) and 2nd line of treatment B (chemotherapy only; 20 patients) were obtained from a database created in 2011 by the Obstetrics and Ginecology Unit at Campus Biomedico teaching hospital, Rome. Patients were followed-up for 2 years. Resources were valued according to the above mentioned database and literature, following the societal viewpoint. Costs are expressed in Euro (€) 2014 and reported as mean and standard deviation (SD). RESULTS: One-year COI for 1st line of treatment reaches € 44,999.7 (SD: €28,757.3), € 55,410.8 (SD: € 32,454.6) and €46,895.6 (SD: € 28,407.4) for 2nd line of treatment A and B, respectively. Regardless the line of treatment, COI is mainly driven by cost borne by patient and her family. Due to the high costs of relapse the mean COI per patient after 2 years from the diagnosis of AOC equals € 81,869.4 (SD: € 30,660.9), or 182% of the COI for the 1st line of treatment. CONCLUSIONS: Despite some limitations, our results show that increasing progression-free survival could well reduce the COI for AOC in Italy.

PURPOSE Paclitaxel albumin (nab-paclitaxel) is a nanoparticle albumin-bound paclitaxel formulation aimed at increasing therapeutic index in metastatic breast cancer. When compared to conventional paclitaxel, nab-paclitaxel has a reported longer time to progression, higher response, lower incidence of neutropenia, no need for premedication, shorter time of administration, and in pretreated metastatic breast cancer patients, extended overall survival. This study investigates the cost-effectiveness of nab-paclitaxel versus conventional paclitaxel for pretreated metastatic breast cancer patients in Italy. MATERIALS AND METHODS A Markov model with progression-free, progressed, and dead states was developed to estimate costs, outcomes, and quality adjusted life years over 5 years from the Italian National Health Service viewpoint. Patients were assumed to receive nab-paclitaxel 260 mg/m(2) three times weekly or conventional paclitaxel 175 mg/m(2) three times weekly. Data on health care resource consumption was collected from a convenience sample of five Italian centers. Resources were valued at Euro (€) 2011. Published utility weights were applied to health states to estimate the impact of response, disease progression, and adverse events on quality adjusted life years. Three sensitivity analyses tested the robustness of the base case incremental cost-effectiveness ratio (ICER). RESULTS AND CONCLUSION Compared to conventional paclitaxel, nab-paclitaxel gains an extra 0.165 quality adjusted life years (0.265 life years saved) and incurs additional costs of €2506 per patient treated. This translates to an ICER of €15,189 (95% confidence interval: €11,891-€28,415). One-way sensitivity analysis underscores that ICER for nab-paclitaxel remains stable despite varying taxanes cost. Threshold analysis shows that ICER for nab-paclitaxel exceeds €40,000 only if cost per mg of conventional paclitaxel is set to zero. Probabilistic sensitivity analysis highlights that nab-paclitaxel has a 0.99 probability to be cost-effective for a threshold value of €40,000 and is the optimal alternative from a threshold value of €16,316 onwards. Based on these findings, nab-paclitaxel can be considered highly cost-effective when compared to the acceptability range for ICER proposed by the Italian Health Economics Association (€25,000-€40,000).

To test the safety and efficacy of using the Arrow-Trerotola percutaneous thrombolytic device (PTD) for treating deep vein thrombosis (DVT) in an animal model.An established canine model of iliocaval subacute thrombosis was used. Thrombosis was caused by balloon occlusion of the infrarenal inferior vena cava (IVC) for 7 (n = 12), 10 (n = 1), or 17 (n = 1) days. Treatment was performed with use of an 8-F, over-the-wire (0.035-inch) PTD with a 15-mm-diameter basket. The procedure was performed without IVC filtration. Two acute procedures were performed and 12 procedures were intended as survival procedures with 30-day follow-up. Pulmonary arteriography, blood gases, and pulmonary artery pressure measurement were performed before and after the procedure, and at follow-up. The animals were killed after the follow-up procedure and their IVC, iliac veins, and lungs were removed and examined histologically. Heparin was used intraprocedurally but thrombolytic agents were not used. Low-molecular-weight heparin was given daily after the procedure.Thrombolysis was completely (12 of 13) or partially (one of 13) successful in all animals in the 7- and 10-day groups, but was unsuccessful in the animal in the 17-day group (n = 1). Variable amounts of segmental and subsegmental pulmonary emboli were found in all animals with small increases in pulmonary artery pressure. Two animals died within 6 days of the procedure, possibly due to pulmonary emboli. At 30-day follow-up, IVC patency was preserved in 80% (eight of 10) of animals, but significant caval narrowing due to intimal hyperplasia was noted at follow-up. All pulmonary emboli had resolved angiographically at follow-up, but evidence of recanalized or resolving pulmonary thromboemboli was found in seven of the 12 surviving animals. No acute vascular injury (eg, perforation) occurred.The modified PTD used in this study is effective in treating subacute (7 days old) venous thrombosis, but temporary filtration will probably be necessary to keep pulmonary emboli to a minimum during the procedure. The 30-day patency is encouraging. The results in this animal model indicate that the Arrow-Trerotola PTD may be useful in the percutaneous treatment of DVT in humans.

Objective To investigate the costs of therapy with Mirena, a Levonorgesterol releasing intrauterine device, compared with endometrial ablation and hysterectomy in the management of menorrhagia.

Purpose To assess the safety and efficacy of using the Arrow-Trerotola percutaneous thrombolytic device (PTD) as the sole means of mechanical thrombolysis in hemodialysis access grafts, including in situ treatment of the arterial plug. Patients And Methods Fifty consecutive patients (22 women, 28 men; mean age, 58 years; mean graft age, 29 months), in whom mechanical thrombolysis of a thrombosed hemodialysis access graft using the PTD was planned, were included in the study. In all patients, the PTD was used to treat the arterial plug in situ at the arterial anastomosis, instead of using a Fogarty catheter to reposition the plug, as indicated in the PTD product labeling. Prospective data collection included demographic information, technical details of the procedure, immediate outcomes, and complications. Patients were followed for 3 months using definitions and data forms that were identical to those used in the original clinical trial of the PTD. A sample of procedures drawn from the PTD clinical trial database ( n = 54) served as control. Results Immediate technical patency was 100%. Complications included arterial embolization (6% versus 2% control; P = NS; all successfully treated with backbleeding); venous rupture (6% versus 2% control; P = NS); and sepsis ( n = 1), probably due to occult graft infection. Adjunctive therapy with an Adherent Clot catheter was needed in two procedures (4%). Three month patency using life-table analysis was 42% (versus 39% control; P = NS). The number of subsequent interventions (surgical/percutaneous) to the arterial limb of the graft did not differ from the PTD trial, and no native arterial stenoses were detected during the follow-up period. Conclusions The PTD is safe and effective when used as the sole means of mechanical thrombolysis of hemodialysis grafts. Treating the arterial plug in situ with the PTD eliminates the need for a Fogarty or Adherent Clot catheter in 96% of procedures. A slight increase in arterial embolic complications was observed but these were easily treated with backbleeding.

Aims To determine the long-term costs of extending device longevity in four patient populations requiring a single-chamber implantable cardioverter-defibrillator (ICD) or requiring cardiac resynchronization therapy with defibrillation (CRT-D) device over a 15-year time window. Methods and results We considered patient populations with an accepted indication for a single-chamber ICD for prevention of sudden cardiac death in the context of preserved (Population A) or impaired (Population B) left ventricular function; or with indication for a CRT-D device in the context of heart failure in New York Heart Association class II (Population C) or III (Population D). Expected patient survival and a cost analysis, including the cost of complications, was undertaken from a hospital perspective. Extended device longevity of 5 vs. 9 years for ICDs (Populations A and B); 4 vs. 7 years for CRT-Ds (Populations C and D) were considered. Over a 15-year time horizon, total, yearly, and per diem savings, per patient, from extending ICD longevity to 9 years were €10 926.91, €728.46, and €1.99 for Population A, and €7661.32, €510.75, and €1.40 for Population B. Total, yearly, and per diem savings from extending CRT-D longevity to 7 years were €13 630.38, €908.69, and €2.49 for Population C, and €10 968.29, €731.22, and €2.00 for Population D. Avoidance of a generator replacement amounted up to 46.6–62.5% of the saving. Conclusion Extending device longevity has an important effect on the long-term cost of device therapy, both for ICD and CRT-D. This has important implications for device choice.

Complete ependymoma resection ensures a better prognosis for children with this tumor, but the complete excision of infratentorial ependymomas involves serious risks. Second-look surgery for tumor remnants may be less harmful and enable complete removal. There is a potential, although still unclear, role for neoadjuvant chemotherapy in preparation for further surgery.Since 1994, the authors have adopted two successive protocols for intracranial ependymoma, both including a phase of adjuvant chemotherapy for children with surgical tumor remnants with a plan for potential second-look surgery before radiotherapy.In the first protocol, 9 of 63 children underwent further surgery, and 6 became tumor free with no additional sequelae. Their prognosis for progression-free survival and freedom from local relapse was comparable to that of children who were operated on only once. In the second protocol, efforts were made to achieve complete resection and 29 of 110 patients underwent reoperations: 9 after the first surgery, 17 after chemotherapy, and 3 soon after radiotherapy. Fourteen of the 29 patients became tumor free, 1 of them with worsening neurological symptoms. The outcome of the 66 patients who became tumor free after 1 operation was compared with that of the 14 who became tumor free after reoperation. The 3-year progression-free survival of the 66 patients compared with the 14 other patients was 71.4% ± 6.9% and 90% ± 9.5%, respectively; the 3-year freedom from local relapse was 84.7% ± 5.9% and 90% ± 9.5%, respectively; and the 3-year overall survival was 85.9% ± 5.4% and 87.5% ± 11.7%, respectively.Second-look surgery proved feasible with no major morbidity, and results improved with time. Local tumor control was comparable in patients undergoing 1 or more resections.