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Rare disease (RD) patients present complex therapeutic needs. When there are therapeutic options available, orphan drugs (OD) represent only a limited proportion of prescribed treatments. This study aims at investigating the real-world use of treatments considered not replaceable and essential for the care of RD patients, besides their reimbursement status, using data from a RD population-based registry.The study is based on data derived from the Veneto region RD registry. For the period 2019-2020, we have analyzed the prescriptions of treatments defined as essential and not replaceable, besides their reimbursement status, included in therapeutic plans issued by RD expert Centres for patients resident in the Veneto region (north-east of Italy, 4.9 million inh.). The correspondent pharmaceutical costs have been estimated as well.In the study period there have been 22.186 prescriptions, included in 9,197 therapeutic plans issued for RD patients resident in the monitored area. The plans present a high level of complexity in terms of number and type of prescribed treatments, with 11% of the plans containing 5 or more prescriptions. 3,041 medicinal products have been prescribed in the study period, of whom 41% are drugs. Although these prescriptions are distributed among all the groups of RD patients, only a limited proportion of products (n=10) is responsible of the 50% of all the costs attributable to these treatments. Overall, the annual cost attributable to essential treatments not directly reimbursed by the national health system is quantifiable in 1 million euros per million inhabitants.This real-world study offers a snapshot of the complexity of treatments defined as essential, besides their reimbursement status, in therapeutic plans issued by RD expert Centres for a consistent group of RD patients monitored by a population-based registry. It highlights the complexity of the therapeutic approaches put in place for the care of RD patients, including drugs and a variety of other treatments. Population-based registries collecting data on prescribed treatments can contribute to understand the therapeutic needs of RD patients, treatments' accessibility and the impact of prescriptions on the global pharmaceutical costs.

Objectives: Aim of the study was to describe the use and pharmacoutilization profiles of recommended drugs for HF patients, hospital re-admission rates, mortality rates and determine healthcare res...

PURPOSE: The present work aimed at conducting a real-world data analysis on the management costs and survival analysis comparing data from non–small-cell lung cancer (NSCLC) cases diagnosed in the Veneto region before (2015) and after (2017) the implementation of a regional diagnostic and therapeutic pathway including all new diagnostic and therapeutic strategies. METHOD: This study considered 254 incidental cases of NSCLC in 2015 and 228 in 2017 within the territory of the Padua province (Italy), as recorded by the Veneto Cancer Registry. Tobit regression analysis was performed to verify if total and each item costs (2 years after NSCLC diagnosis) are associated with index year, adjusting by year of diagnosis, sex, age, and stage at diagnosis. Logistic regression models were run to study overall mortality at 2 years, adjusting by the same covariates. RESULTS: The 2017 cohort had a lower mortality odd (odds ratio, 0.93; P = .02) and a significant increase in the average overall costs ( P = .009) than the 2015 cohort. The Tobit regression analysis by cost item showed a very significant increase in the average cost of drugs (coefficient = 5,953, P = .008) for the 2017 cohort, as well as a decrease in the average cost of hospice care (coefficient = –1,822.6, P = .022). CONCLUSION: Our study showed a survival improvement for patients with NSCLC as well as an economic burden growth. Physicians should therefore be encouraged to follow new clinical care pathways, while the steadily rising related costs underscore the need for policymakers and health professionals to pursue.

Background and Aims Diabetes conveys an increased risk of infectious diseases and related mortality. We investigated risk of ascertained SARS-CoV-2 infection in diabetes subjects from the Veneto Region, Northeastern Italy, as well as the risk of being admitted to hospital or intensive care unit (ICU), or mortality for COVID-19. Methods and Results – Diabetic subjects were identified by linkage of multiple health archives. The rest of the population served as reference. Information on ascertained infection by SARS-CoV-2, admission to hospital, admission to ICU and mortality in the period from February 21 to July 31, 2020 were retrieved from the regional registry of COVID-19. Subjects with ascertained diabetes were 269,830 (55.2% men; median age 72 years). Reference subjects were 4,681,239 (men 48.6%, median age 46 years). Ratios of age- and gender-standardized rates (RR) [95% CI] for ascertained infection, admission to hospital, admission to ICU and disease-related death in diabetic subjects were 1.31 [1.19-1.45], 2.11 [1.83-2.44], 2.45 [1.96-3.07], 1.87 [1.68-2.09], all p

Background The European Medicines Agency has recently approved rivaroxaban for the prevention of atherothrombotic events in patients with coronary artery disease (CAD) and peripheral arterial disease (PAD). Purpose The present study aimed at analysing the prevalence of CAD and PAD among the health-assisted population and to estimate the pharmaceutical expenditure. Material and methods Data have been obtained by retrospectively analysing a regional healthcare database between 1 January 2017 and 31 December 2017. We included adult patients (≥18 years) with a diagnosis of CAD (at least one prescription of nitrates and/or CAD diagnosis at hospital discharge) and a diagnosis of PAD (at least one prescription of antiplatelet drugs with concomitant prescription of anti-hypertensive agents or lipid-lowering agents and/or PAD diagnosis at hospital discharge). Patients were characterised over the period 2009–2016 for comorbidities (diabetes mellitus, heart failure, chronic kidney disease, cardiovascular event), during 2016 for drug use. Treatment adherence was calculated using the Medication Possession Ratio. Results More than 4.9 million health-assisted participants, 8.8% (430,254, mean age 74.2 years) received a diagnosis of CAD or PAD (41.2% PAD, 30.4% CAD, 28.4% both). Forty-two per cent (180,551) of patients had at least one comorbidity. Most frequently prescribed cardiovascular agents were anti-hypertensives (85%), antiplatelet drugs (58.7%), lipid-lowering drugs (53%) and anticoagulants (17.2%). Among patients treated with antiplatelet drugs, 36.2% were adherent to treatment. Considering the actual price of rivaroxaban, the pharmaceutical expenditure for one-year treatment in the considered region resulted at €266 million (€38 million is the current pharmaceutical expenditure for new anticoagulant therapies for 2018). Applying this analysis for patient sub-groups, the estimation was €191 million for patients with CAD or PAD, €75 million for patients with both and €112 million for those with at least one comorbidity. Considering patients adherent to antiplatelet treatment, the estimation was €58 million. Conclusion Nearly one out of ten health-assisted subjects had CAD and PAD diagnosis, in accordance with that already published for the national population. Pharmaceutical costs’ estimations represent the maximal costs as they did not consider the possible reduction in drug prices negotiated by the National Medicines Agency, considering the significant market increase and redeemability limitations. References and/or acknowledgements No conflict of interest.

Aims We assessed the influence of patient and General Practitioner (GP) characteristics on the adherence to process of care indicators for non-insulin-treated type 2 diabetes management in the Veneto Region (northeastern Italy). Methods Among non-insulin-treated diabetic patients aged 18–84 years identified by multiple information sources, we assessed the measurement of glycated hemoglobin, microalbumin, and lipids through the year 2013. Patients’ variables included gender, age, citizenship, and the attendance to Diabetes Clinics, while GP characteristics were gender, age and an attitude score derived from a questionnaire. The influence of patient and GP variables were investigated through multilevel regression with the execution of two HbA1c tests in 2013 as the outcome. Results Out of 139,935 study subjects, more than 70% had at least one HbA1c test in 2013; this percentage decreased to about 40% for two HbA1c examinations. 67% of patients had an assessment of lipid profile, while 45% underwent a microalbumin test. These percentages were lower for immigrant patients and increased with age until the 65–74 years age class. Patients attending Diabetes Clinics were usually better monitored than those who did not. In this latter group, female gender (risk ratio 1.08, 1.02–1.14), younger age (risk ratio 1.15, 1.06–1.25) and high attitude score of GPs (risk ratio 1.20, 1.13–1.27) were associated with a better management. Conclusions Both patient and GP variables influence the adherence to process of care indicators. The implementation of effective strategies of disease management at the primary care level may improve the control of glycemic and cardiovascular risk factors.

Direct-acting antiviral agents (DAAs) are safe and effective in patients with hepatitis C. Conflicting data were reported on the risk of hepatocellular carcinoma (HCC) during/after therapy with DAAs. The aim of this study was to evaluate the incidence of newly diagnosed HCC and associated risk factors in patients with advanced hepatitis C treated with DAAs.The study is based on the NAVIGATORE platform, a prospectively recording database of all patients with hepatitis C receiving DAAs in the Veneto region of Italy. The inclusion criteria were: fibrosis stage ≥F3. The exclusion criteria were: Child-Turcotte-Pugh (CTP)-C, liver transplantation before DAAs, history or presence of HCC, follow-up4 weeks after starting DAAs. A total of 3,917 out of 4,234 consecutive patients were included, with a mean follow-up of 536.2 ± 197.6 days.Overall, HCC was diagnosed in 55 patients. During the first year, HCC incidence was 0.46% (95% CI 0.12-1.17) in F3, 1.49% (1.03-2.08) in CTP-A and 3.61% (1.86-6.31) in CTP-B cirrhotics; in the second year, HCC incidences were 0%, 0.2%, and 0.69%, respectively. By multivariate analysis, HCC was significantly associated with an aspartate aminotransferase to platelet ratio ≥2.5 (hazard ratio [HR] 2.03; 95% CI 1.14-3.61; p = 0.016) and hepatitis B virus infection (HR 3.99; 1.24-12.91; p = 0.021). Failure to achieve a sustained virological response was strongly associated with development of HCC (HR 9.09; 5.2-16.1; p = 0.0001). A total of 29% of patients with HCC had an aggressive tumor, often seen in the early phase of treatment.These data, obtained in a large, prospective, population-based study, indicate that in patients with advanced hepatitis C receiving DAAs, the risk of "de novo" hepatocarcinoma during the first year is not higher, and might be lower, than that of untreated patients. The risk further declines thereafter. Early hepatocarcinoma appearance may reflect pre-existing, microscopic, undetectable tumors.Hepatocellular carcinoma is one of the complications of hepatitis C related cirrhosis. Treating patients with advanced hepatitis C with the new interferon-free direct-acting antiviral agents has been associated with improvement in liver function and survival, while more conflicting data have been reported regarding the risk of hepatocellular carcinoma. We report the results of a prospective population study on the incidence of newly diagnosed hepatocellular carcinoma in patients with advanced hepatitis C treated with direct-acting antiviral agents, clearly indicating that the residual hepatocellular carcinoma risk is reduced and declines progressively with time after a sustained virological response. Development of a liver tumor during/after therapy was associated with known risk factors and with virological failure.

Background: Over the past few years, major changes have taken place in the treatment of gastroduodenal peptic ulcer. Aim: To evaluate risk factors associated with the incidence of peptic ulcer in inpatients. Methods: From 2001 to 2004, the number of prescriptions of H 2 -antagonists and proton pump inhibitors (PPIs) in each department of Verona University Hospital was monitored. Over the same period we prospectively recorded the number of upper endoscopies per department for inpatients with a diagnosis of peptic ulcer. Results: We analyzed 4943 inpatients. A significantly decreasing trend in H 2 -antagonist prescriptions (r = - 0,88; P < 0.001) and an increasing trend in PPI prescriptions (r = 0.97; P < 0.001) were observed. The endoscopic incidence of duodenal ulcers decreased linearly from 2001 to 2004 as follows: 6.5% (94/1439) in 2001, 5.6% (82/1473) in 2002, 4.5% (63/1411) in 2003, and 3.1% (22/ 702) (P

INTRODUCTION:A novel, sensor-based, factory-calibrated Flash Monitoring System (FMS) has recently proved to be an effective alternative to conventional self-monitoring of blood glucose (SMBG) in patients affected by type 1 and type 2 diabetes. The 14-days adhesive sensor, that continuously measures glucose levels in the interstitial fluid, can transfer glucose levels data to a handheld reader or a smartphone equipped with a specific medical app. The uptake of the new technology has been limited so far, because of its high costs. A cost analysis has been conducted to identify the optimal target population of introducing FSM in Veneto.METHODS:The model was designed with a 1-year time horizon for patients with diabetes using intensive insulin in Veneto region. The costs of the new technology was estimated using inputs from the two main randomized controlled trials (the IMPACT study and the REPLACE study) published in the international literature, Regional evidence-based guidelines and administrative database. Resource utilization included strips, lancets, needles, sensors, distribution and patients training. Regional unit costs were adopted.RESULTS:FSM has not shown so far relevant and statically significant benefits in terms of severe adverse events’ reduction. Estimated yearly costs for a FSM user included glucose monitoring, technology training and distribution costs, for a total of EUR1277 per patient. The new technology has been shown to be affordable in diabetic patients with i) 4yearsCONCLUSIONS:The Veneto Region should carefully consider prescribing extension to other diabetic patients categories, since the high cost of the new technology. A strict prescribing monitoring is strongly recommended with the aim of ensuring appropriateness and avoiding overspending.

Drug prescription or administration errors are the most common causes of adverse events for hospital patients. Computer-based systems can be effective in reducing these errors. The aim of this study was to assess whether computer-based systems are easily exploitable even in critical conditions. The oral and handwritten system for drug management was completely replaced by a computer-based system in our bone marrow transplant unit, chosen because: (i) the intensive treatments could test the efficiency of the system; (ii) the very frequent occurrence of complications and emergencies could test the flexibility of the system; and (iii) the pre-existing system could be used as comparison. From May to November 2002, 41 patients were repeatedly admitted to undergo high-dose chemotherapy, autologous/allogeneic stem cell transplantation or because of severe complications. In 27 consecutive admissions, 2264 drug prescriptions (average, 29 drugs/patient admission) and 36 786 drug administrations (39.8/patient/d) were carried out. Despite the large number of procedures, the computerized system effectively replaced the oral and handwritten transmission of information among medical staff, pharmacists and nurses, and lowered the error risks. In addition, it contributed to medical updating through warnings on potential problems in case of multiple drug prescriptions, and gave the pharmacy a valuable tool to monitor drug use.

INTRODUCTION:The Veneto Region established a Technical Panel for Continence (TPC) with the purpose of producing guidance for aids based on a Health Technology Assessment (HTA) approach. TPC is a multidisciplinary experts group that involves local clinicians, pharmacists, health economist and patients associations. Among its tasks, TPC can issue recommendations in the field of appropriate use, purchasing and distribution for aids. Currently the TPC is investigating aids for ostomy patients in order to provide the first regional guidance on appropriateness and disease management for ostomy.METHODS:The Regional Health Technology Assessment Unit (CRUF) conducted a literature review of the evidence on aids for ostomy. Grey literature, and National and Regional laws and regulations were also included in the analysis. TPC discussed the collected evidence by consensus. Final recommendations have been sent to the Regional Technical Committee on Medical Devices (CTRDM) for eventual remarks, before final approval.RESULTS:The literature review did not retrieve any relevant international studies on the topic, except for the Canadian clinical guidelines on ostomy. The upcoming regional guidance will suggest recommendations on: (i) appropriate patient disease management based on a multidisciplinary team evaluation; (ii) characteristics and selection criteria for ostomy aids and related accessories; (iii) prescribing medical specialists, authorization and distribution features; and (iv) specific indicators for appropriateness monitoring.CONCLUSIONS:The regional guidance on aids and accessories for ostomy aim at ensuring the appropriateness throughout the Regional Health Service. The strict monitoring of agreed indicators is essential for appropriateness compliance and consequently the sustainability of regional medical devices expenditure.

INTRODUCTION:Chronic Pain (CP) is the uncontrolled pain that affects patients for a long time. CP can be caused by many conditions, sometimes still poorly understood, and its levels can vary from moderate to intense. The management of resistant CP requires a stepwise approach and spinal cord stimulation (SCS) could be considered an extreme strategy. With the aim of ensuring the economic sustainability, the Veneto Region usually establishes rigorous access criteria to high-cost medical devices through its Regional Technical Committee on Medical Devices (CTRDM) and a Health Technology Assessment (HTA) procedure.METHODS:The Regional Health Technology Assessment Unit (CRUF) conducted through Pubmed a literature review of randomized controlled trials, systematic reviews, meta-analysis on SCS published from March 2006 to February 2016. International and national clinical guidelines were included in the analysis as well. The regional multidisciplinary Working Group on CP, which involved local clinicians, pharmacists, clinical engineer and health economist, discussed the collected evidence by consensus. Final recommendations on the appropriate use were submitted to the CTRDM for final approval.RESULTS:The regional guidance describes the type of pain that can be treated with spinal neurostimulators and the criteria which determine the success of the test procedure. A comparative analysis of spinal neurostimulators available on the market and related patients eligibility criteria have been also included. Moreover, the guidelines stated a list of compulsory requirements in order to become a regional center authorized in performing spinal neurostimulation procedure. Finally, the document describes some indicators for appropriateness monitoring. The CTRDM approved the final version in October 2016.CONCLUSIONS:The regional guidance on SCS aims at ensuring the appropriate use of neurostimulators in patients affected by resistant CP. The strict monitoring of agreed indicators is essential for appropriateness and consequently the sustainability of medical devices expenditure throughout the Regional Health Service.

Objective To investigate the organisation and decision-making processes of regional and local therapeutic committees in Italy, as a case-study of decentralised health care systems. Methods A structured questionnaire was designed, validated, and self-administered to respondents. Committee members, prioritisation, assessment process and criteria, and transparency of committees were investigated. Results The respondents represent 100% of the 17 regional committees out of 21 regions (in 4 regions there is not any regional formulary), 88% of the 16 hospital networks and 42% of the 183 public hospitals. The assessment process appears fragmented and may take a long time: drugs inclusion into hospital formularies requires two steps in most regions (regional and local assessment). Most of the therapeutic committees are closed to industry and patients associations involvement. Prioritisation in the assessment is mostly driven by disease severity, clinical evidence, and the absence of therapeutic alternatives. Only 13 out of the 17 regional committees have a public application form for drugs inclusion into regional formulary. Regional and local committees (i) often re-assess the clinical evidence already evaluated at central level and (ii) mostly rely on comparative drug unit prices per DDD and drug budget impact. The level of transparency is quite low. Conclusions The Italian case-study provides useful insights into an appropriate management of multi-tier drugs assessment, which is particularly complex in decentralised health care systems, but exists also in centralised systems where drugs are assessed by local therapeutic committees. A clear definition of regulatory competences at different levels, a higher collaboration between central, regional and local actors, and increased transparency are necessary to pursue consistency between central policies on price and reimbursement and budget accountability at the regional and local levels.

A case-control multicenter study was set up in 68 general and specialistic wards in Italian regional hospitals in order to assess whether underuse of heparin prophylaxis may account for at least a fraction of the thromboembolic events still occurring in surgical patients. 100 cases with clinically relevant thromboembolic or hemorrhagic events occurring during hospitalization for major surgery and 200 controls were identified. Controls were selected among patients not presenting any of the events under study during the same period of observation and were matched with cases for age, sex, and type of surgery. The results of the study suggest that heparin use in routine conditions of care closely reflects the “consensus” knowledge, patients at higher risk (specifically orthopedic surgical patients, those with varicose veins or with preoperative bed rest longer than 3 days) being treated more frequently with heparin. Absence of heparin prophylaxis does not appear to represent a specific risk factor for the occurrence of index events (OR 0.73, 95% CI = 0.42-1.26). Despite the higher rates of heparin exposure, the presence of varicose veins is associated with a statistically significant increase in the risk of postoperative complications (OR 2.23, 95% CI = 1.07-4.65). This study indicates that among known pre- and peri-operative risk factors only varicose veins may be unprotected by the current prophylaxis practice.

This contribution describes the present regulatory status in the EU of biosimilars, the generic versions of the first generation of therapeutic proteins. It points out why and where recombinant protein molecules and low-molecular-weight drugs differ in their behaviour and why biosimilars should be handled differently than generic low-molecular-weight drugs. This information is important for practitioners (pharmacists and physicians) while selecting the best supplier of a therapeutic protein.

This contribution describes the present regulatory status in the EU of biosimilars, the generic versions of the first generation of therapeutic proteins. It points out why and where recombinant prot

Drug prescription or administration errors are the most common causes of adverse events for hospital patients. Computer-based systems can be effective in reducing these errors. The aim of this study was to assess whether computer-based systems are easily exploitable even in critical conditions. The oral and handwritten system for drug management was completely replaced by a computer-based system in our bone marrow transplant unit, chosen because: (i) the intensive treatments could test the efficiency of the system; (ii) the very frequent occurrence of complications and emergencies could test the flexibility of the system; and (iii) the pre-existing system could be used as comparison. From May to November 2002, 41 patients were repeatedly admitted to undergo high-dose chemotherapy, autologous/allogeneic stem cell transplantation or because of severe complications. In 27 consecutive admissions, 2264 drug prescriptions (average, 29 drugs/patient admission) and 36 786 drug administrations (39.8/patient/d) were carried out. Despite the large number of procedures, the computerized system effectively replaced the oral and handwritten transmission of information among medical staff, pharmacists and nurses, and lowered the error risks. In addition, it contributed to medical updating through warnings on potential problems in case of multiple drug prescriptions, and gave the pharmacy a valuable tool to monitor drug use.

The hospital formulary is not only a list of drugs, but also a policy that involves several complex processes and activities. Formularies are developed based on cost-effectiveness evaluation, but maintaining cost-effectiveness requires educational strategies. The clinical pharmacist plays an active role in formulary processes. These processes are discussed in this paper. There is a need for studies on the cost-effectiveness of clinical pharmacy services in support of the hospital formulary.