The emergence and organizational choices of patient advocacy associations: Evidence from the Italian context.

BACKGROUND: Within many advanced healthcare systems, patient advocacy associations are gaining a more relevant role in healthcare policy decisions. These associations provide help, representation, and voice to patients and citizens. However, although their role is important, a paucity of research exists analyzing their foundation dynamics, including their organizational decision, from a managerial perspective. To fill this knowledge gap, we formulated two research questions to investigate the motivations underlying the foundation of patient advocacy associations and the institutional pressures influencing the changes that have occurred in these associations throughout their evolution. METHODS: Using a semi-structured questionnaire developed as part of a larger research project, we collected data about 31 patient advocacy associations operating within the Italian National Health Service. We employed qualitative analysis to examine the associations' birth and evolution as well as the motivations and influences driving change within the sampled organizations. RESULTS: Questionnaire responses provided information about the patient advocacy associations' histories and highlighted their close links with the Italian National Health Service. Our results indicated the motivations for the associations' establishment could be grouped into five categories: support at the system level, patient protection and support, actions to increase awareness, actions for improving patients' health and quality of life, and dissemination of knowledge. We also identified the frequency and nature of the changes within the associations and explored the institutional pressures that influenced these organizational changes. CONCLUSIONS: The results can be interpreted considering the organizational theories that focus on organizations' establishment decisions and how external pressures impact organizations' dynamics and evolution.

A budget impact model and a cost-utility analysis of reducer device (Neovasc) in patients with refractory angina.

Background: Refractory angina (RA) is a chronic condition characterized by the presence of debilitating angina symptoms due to established reversible ischemia in the presence of obstructive coronary artery disease (CAD). Treatments for this condition have undergone major developments in recent decades; however, the treatment for RA remains a challenge for medicine. In this sense, the Coronary Sinus Reducer System (CSRS) stands as the last line of therapy for ineligible patients for revascularization with reversible ischemia. The purpose of this report is to evaluate the potential burden on the National Health Service (NHS) and measure the health effects in terms of both quantity (life years) and quality-of-life aspects related to the reducer. Methods: Two different economic evaluation models were developed as part of the analysis. The budget impact was developed to estimate the potential burden on the NHS from incremental uptake of the use of the reducer in the target population. The utility cost analysis compares and evaluates the quality of life and health resource use and costs between the two alternatives, based on the research of Gallone et al. A deterministic and probabilistic sensitivity analysis was carried out to characterize the uncertainty around the parameters of the model. Results: In the budget impact analysis (BIA), the reducer is shown to be more expensive in the first 2 years of the model, due to the gradual uptake in the market and the cost of the device. Starting from the third year, assuming maintenance of effectiveness, there are savings in terms of resource absorption in direct healthcare costs arising from hospitalizations, emergency department accesses, coronarography, and visits avoided. Conclusion: The BIA and cost-effectiveness model show that the reducer device, despite an increase in resources absorbed in the first years of implementation and use, has the potential to result in increased quality of life in patients with RA. These costs are largely offset in the short term by the improved clinical outcomes achievable leading to savings from the third year onward in the BIA and a dominance ratio in the cost-utility analysis.

Feasibility of a Stop Smoking Program for Healthcare Workers in an Italian Hospital: Econometric Analysis in a Total Worker Health® Approach.

Background: Over 20% of healthcare workers (HCWs) are active smokers. Smoking is a targeted issue for workplace health promotion (WHP) programs. Objective: Our study aims to evaluate the effectiveness of the Stop Smoking Promotion (SSP) intervention, a 6-hour training course for HCWs, which took place from May 2018 to July 2019. Methods: We compared HCWs who successfully quit smoking (n = 15) to those who did not (n = 25) in terms of Sickness Absence Days (SADs). Moreover, we conducted an econometric analysis by calculating the return on investment and implementing a break-even analysis. Findings: Among the 40 enrolled workers, a success rate of 37.5% was observed after a span of over two years from the SSP intervention (with nurses and physicians showed the best success rate). Overall, participants showed a noticeable absenteeism reduction after the SSP intervention, with a reduction rate of 85.0% in a one-year period. The estimated ROI for the hospital was 1.90, and the break-even point was 7.85. In other words, the organization nearly doubled its profit from the investment, and the success of at least eight participants balanced costs and profits. Conclusion: Our pilot study confirms that WHP programs are simple and cost-saving tools which may help improve control over the smoking pandemic in healthcare settings.

Budget impact of prophylactic treatment of rVIII-SingleChain in moderate and severe hemophilia A in Italy.

INTRODUCTION: rVIII-SingleChain, a recombinant factor VIII (rFVIII), has demonstrated safety and efficacy in patients with hemophilia A in clinical trials and real-world evidence. This analysis aimed to estimate the potential budget impact of increasing the usage of rVIII-SingleChain for the prophylactic treatment of hemophilia A over 3 years in Italy. METHODS: Patients with moderate and severe hemophilia A receiving prophylaxis were included in the analysis. Epidemiological data were obtained from published literature. Mean product consumption and mean annual bleeding rate for rVIII-SingleChain, rFVIIIFc, octocog alfa and BAY 81-8973 were based on pooled real-world data from Italy, Germany and US. A budget impact model has been developed in order to compare two scenarios: a base-case scenario where current rVIII-SingleChain shares are kept constant over 3 years and an alternative scenario where rVIII-SingleChain shares increase by taking from other rFVIII products. Analysis 1 was based on the current Italian list prices and Analysis 2 considered current regional acquisition prices for both scenarios. RESULTS: Annually, adult patients treated with rVIII-SingleChain prophylaxis are expected to consume 324,589 units per patient, resulting in annual costs of €240,196 per patient. In Analysis 1, comparing the base case (constant market share of 9% rVIII-SingleChain over time) with the alternative scenario (higher rVIII-SingleChain market share and increasing from 15% in the first year to 25% in the third year), the total expenditure for prophylaxis using rFVIII products is expected to decrease by €1.4 million in Year 1, by €3.1 million in Year 2 and by €5.4 million in Year 3. In Analysis 2 based on regional prices, the results remained consistent. DISCUSSION/CONCLUSION: This analysis suggests that increasing utilization of rVIII-SingleChain in hemophilia A patients may lead to cost savings as a result of reduced consumption with uncompromised efficacy in bleed protection.

Why was the study done? Hemophilia A is a rare inherited bleeding disorder. People with severe hemophilia are more likely to bleed compared to people without hemophilia and bleeds can occur spontaneously or in response to trauma. Patients are treated with medication to reduce the chance of bleeding. However, the cost of treating patients with hemophilia can be high and place demands on the healthcare system.What did we do and find?This study looked at the cost of treating people with hemophilia in Italy and used a type of economic analysis (called budget impact modelling) to estimate the effect of increasing the use of a particular medication (rVIII-SingleChain), compared to other medications that are available. Different variations of the model were tested to compare a range of scenarios.The results of this analysis suggested that increasing the use of rVIII-SingleChain may lead to cost-savings for the Italian healthcare system, compared to using the other currently available treatments. This analysis suggests that the use of rVIII-SingleChain enables people with hemophilia A to remain protected from bleeds, whilst using less product compared to other available medications.What is the influence of this study on the wider field?This type of analysis can be useful to healthcare systems, to guide the decision-making process regarding which medications to use or when making decisions related to healthcare policy.

The added value of applying a disinvestment approach to the process of health technology assessment in Italy.

OBJECTIVES: The objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare. METHODS: Previous international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available. RESULTS: The disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan. CONCLUSIONS: Anchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.

Real-World Data and Budget Impact Analysis (BIA): Evaluation of a Targeted Next-Generation Sequencing Diagnostic Approach in Two Orthopedic Rare Diseases.

Objective: Next-generation sequencing (NGS) technology, changing the diagnostic approach, has become essential in clinical settings, and its adoption by public health laboratories is now the practice. Despite this, as technological innovations, its intake requires an evaluation of both the clinical utility and the economic investment, especially considering the rare disease scenario. This study evaluated the analytical validity and the budget impact of an NGS-Ion Torrent™ approach for the molecular germline diagnosis of two musculoskeletal rare diseases. Methods: Two cohorts of 200 and 199 patients with suspect or clinical diagnosis of multiple osteochondromas (MO) and osteogenesis imperfecta (OI) previously evaluated with a single-gene diagnostic protocol were re-analyzed using a targeted NGS assay. Analytical validity was assessed by comparing NGS and single-gene protocol. A budget impact analysis using real-world cost data-considering the healthcare perspective- was performed by applying activity-based costing (ABC). The cost considered consumables, personnel, and equipment. Additional costs not related to NGS activities were not considered. Sensitivity analysis was performed. Results: The NGS method showed a higher (for MO) and comparable (for OI) diagnostic sensitivity than the traditional techniques, apart from always reducing the time and costs of diagnosis. Overall, the cost saving per patient is € 765 for OI and € 74 for MO. Materials represented the highest cost driver of the NGS process. A time saving-proportional to the panel size-has been assessed in both cases. Conclusions: Our targeted NGS diagnostic approach decreases time to diagnosis and costs, appearing to be beneficial and recommended both for patients and from a healthcare perspective in routine diagnosis also considering very small gene panels and a low patient flow. The adequate analytical sensitivity always required the additional Sanger sequencing step of the low- and non-covered regions. A more accurate strategy evaluation is suggested in the case of ultra-rare/complex diseases, large gene-panel, or non-reference diagnostic centers.

Analysis of the multidisciplinary approach for the management of patients affected by chronic myeloid leukaemia.

Background: Chronic myelogenous leukaemia (CML) is a pathological clinical condition with a yearly incidence between 10 and 15 cases per million people, 14 in Italy. Its incidence increases with age, reaching 20-25 yearly cases per million individuals in people over 70 years. A growing importance has been given to the need of a multidisciplinary approach (MDA) for the management of patients with CML. Objective: To analyse the importance of MDA as compared with the current Italian standard of care for the management of CML patients based on the involvement of several health professional figures. Methods: A group of healthcare professionals from several healthcare structures were gathered in a first Advisory Board (AB) and divided into as many groups as the number of belonging health structures representative of the Italian provision of therapeutic approaches for CML. In a second AB, the results were validated by the same panel of experts. Results: The number of dedicated health professionals within the dedicated ward ranged from 1 to 13. Most structures rely on several professionals intervening only in case of necessity. Only one centre provides a booking service based on clinical needs to avoid waiting times. Most centres report there is basically no disagreement in the definition of the clinical pathway and there is a high adherence to national and international CML guidelines. Conclusions: The development of forms of interorganisational and interprofessional coordination to improve the diagnosis and the treatment of CML patients have been for long on the agenda of policy makers, managers and professionals for more than 20 years. This analysis represents a starting reference to consider for the evaluation of an MDA in the Italian context.

Return on Investment (ROI) and Development of a Workplace Disability Management Program in a Hospital-A Pilot Evaluation Study.

The progressive ageing of the working population and the increase in related chronic diseases tend to affect working capacity. The aim of this study was to evaluate a Workplace Disability Management Program (WDMP) within a pediatric hospital. Absenteeism due to healthcare workers' (HCWs) pre- and post- WDMP and the related costs were used for the program evaluation. The Return on Investment (ROI), the Break-Even Analysis (BEA) and the value of the average annual productivity of HCWs who took advantage of the Disability Management (DM) interventions to assess the economic impact of the program, were also used. The HCWs enrolled in the program were 131 (approximately 4% of hospital staff), of which 89.7% females and with an average age of 50.4 years (SD ± 8.99). Sick leave days of the HCWs involved decreased by 66.6% in the year following the end of WDMP compared to the previous one (p < 0.001). The total estimated cost reduction of absenteeism is 427,896€ over a year. ROI was equal to 27.66€. BEA indicated that the break-even point was reached by implementing the program on 3.27 HCWs. The program evaluation demonstrated the particular effectiveness of the implemented WDMP model, acting positively on the variables that affect productivity and the limitation to work.

Estimating the fiscal impact of three vaccination strategies in Italy.

OBJECTIVES: When assessing the economic value of vaccines, decision makers should adopt a full societal perspective. One approach for estimation of the fiscal impact of a disease is to use the human capital method to determine productivity losses. The aim of this study was to test an analytical framework developed for the estimation of the fiscal impacts of vaccination programs for influenza (FLU), pneumococcus (PC), and herpes zoster (HZ), in Italy. METHODS: We tested the framework in a two-stage analysis. First, we estimated the fiscal impact of the disease, second we performed a cost-benefit analysis of the individual benefits of vaccination against the cost of the vaccine. To estimate the fiscal impact of the diseases, the human capital approach was used. Epidemiological data were extrapolated from the literature. A Monte Carlo simulation enabled exploration of the uncertainty in the model variables. RESULTS: For FLU, assuming 2.1 million people infected, the total expected impact was EUR 999,371,520; the estimated fiscal impact was EUR 159,563,520. For PC, assuming 90,000 people infected, the total impact was EUR 148,055,040 and the estimated fiscal impact was EUR 23,639,040. For HZ, assuming 6,400 people infected, the total impact was EUR 4,777,200, with EUR 630,000 resulting from a decrease in fiscal taxation. CONCLUSIONS: In conclusion, our work shows how traditional methods aimed at estimating the cost of illness from a social perspective can be improved by additionally considering the fiscal impact, which accounts for the decrease in fiscal revenues due to illness.

NutriCatt protocol in the Enhanced Recovery After Surgery (ERAS) program for colorectal surgery: The nutritional support improves clinical and cost-effectiveness outcomes.

BACKGROUND: Postoperative complications and length of hospital stay (LOS) are major issues and affect hospital costs. Enhanced Recovery After Surgery (ERAS) protocols are effective in reducing morbidity and LOS after major surgery. We propose a nutritional protocol within ERAS programs in colorectal surgery, starting from preadmission. METHODS: We compared the ERAS + NutriCatt approach versus the ERAS standard program adopted in our center in the previous months. Complications, LOS, hospital readmission at 30 days, and late complications (at 90 days) were assessed and compared. A cost-effectiveness analysis was performed. RESULTS: A total of 114 patients were treated according to the ERAS program between April 2015 and January 2016; 105 were enrolled in the ERAS + NutriCatt protocol from February to September 2016; Patients' characteristics were similar in the two groups, except for American Society of Anesthesiologists score, which was significantly worse in the ERAS + NutriCatt cohort; preoperative diagnoses and surgical approaches were similar in the two periods. LOS was significantly inferior in the ERAS + NutriCatt protocol (4.9 ± 1.7 d; 95% confidence interval [CI] 4.60-5.28) compared with the standard ERAS program (6.1 ± 3.9 d, 95% CI 5.36-6.81) (P = 0.006), as were postoperative complications (36, 34.3% versus 55, 48.2%; P = 0.03). Complications within 90 d were 0 in ERAS + NutriCatt and 4 in the ERAS standard cohort. Cost-effectiveness analyses indicated savings in the ERAS + NutriCatt protocol. CONCLUSIONS: Nutritional care, starting from the preadmission visit, is able to reduce LOS, postoperative and late complications, and costs, in addition to ERAS standard items in colorectal surgery.

Economic evaluation of screening programs for hepatitis C virus infection: evidence from literature.

BACKGROUND: Hepatitis C is a liver infection caused by hepatitis C virus. Its main complications are cirrhosis and liver cancer. According to the World Health Organization (WHO), more than 185 million people worldwide are infected with hepatitis C virus and, of these, 350,000 die every year. Due to the high disease prevalence and the existence of effective (and expensive) medical treatments able to dramatically change the prognosis, early detection programs can potentially prevent the development of serious chronic conditions, improve health, and save resources. OBJECTIVE: To summarize the available evidence on the cost-effectiveness of screening programs for hepatitis C. METHODS: A literature search was performed on PubMed and Scopus search engines. Trip database was queried to identify reports produced by the major Health Technology Assessment (HTA) agencies. Three reviewers dealt with study selection and data extraction blindly. RESULTS: Ten papers eventually met the inclusion criteria. In studies focusing on asymptomatic cohorts of individuals at general risk the cost/quality adjusted life year of screening programs ranged between US $4,200 and $50,000/quality adjusted life year gained, while in those focusing on specific risk factors the incremental cost-effectiveness ratio ranged between $848 and $128,424/quality adjusted life year gained. Age of the target population and disease prevalence were the main cost-effectiveness drivers. CONCLUSION: Our results suggest that, especially in the long run, screening programs represent a cost-effective strategy for the management of hepatitis C.