A cost-of-illness study of Behçet syndrome in Italy.

OBJECTIVE: This study aims at evaluating the cost-of-illness (COI) of patients diagnosed with Behcet's syndrome (BS) in Italy, trying to depict the impact of different costs' components to the overall economic burden and analysing the variability of costs according to years since diagnosis and age at first symptoms. METHODS: With a cross-sectional evaluation, we surveyed a large sample of BS patients in Italy assessing several dimensions related to BS, also including fact related to the use of health resources utilization, formal and informal care, and productivity losses. Overall costs, direct health, direct non-health, and indirect costs were thus estimated per patient/year considering a Societal perspective and the impact of years since diagnosis, age at first symptoms on costs was evaluated using generalized linear model (GLM) and a two-part model, adjusting for age and distinguishing among employed and non-employed responders. RESULTS: A total of 207 patients were considered in the present study. From the perspective of the Society, mean overall costs for BS patient were estimated to be 21,624 € (0;193,617) per patient/year. Direct non-health expenses were the main costs component accounting for 58% of the overall costs, followed direct health costs, 36%, while indirect costs because of productivity losses represented 6% of the overall costs. Being employed resulted in significantly lower overall costs (p = 0.006). Results from the multivariate regression analyses suggested that the probability of incurring in overall costs equal to zero decreased as time from BS diagnosis is 1 year or more as compared to newly diagnosed patients (p < 0.001); while among those incurring in expenses, costs decreased for those experiencing first symptoms between 21 and 30 years (p = 0.027) or later (p = 0.032) as compared to those having symptoms earlier. Similar findings emerged among the subgroups of patients declaring themselves as workers, while no impact of years since diagnosis or age of first symptoms was found among non-workers. CONCLUSIONS: The present study offers a comprehensive overview of the economic consequences imposed by BS in a societal perspective, providing insights into the distribution of the different costs component related to BS, thus helping the development of targeted policies.

Exploring patient's experience and unmet needs on pregnancy and family planning in rare and complex connective tissue diseases: a narrative medicine approach.

OBJECTIVE: The aim of this work is to explore patient' unmet needs of rare and complex rheumatic tissue diseases (rCTDs) patients during pregnancy and its planning by means of the narrative-based medicine (NBM) approach. METHODS: A panel of nine rCTDs patients' representatives was identified to codesign a survey aimed at collecting the stories of rCTD patients who had one or more pregnancies/miscarriages. The results of the survey and the stories collected were analysed and discussed with a panel of patients' representatives to identify unmet needs, challenges and possible strategies to improve the care of rCTD patients. RESULTS: 129 replies were collected, and 112 stories were analysed. Several unmet needs in the management of pregnancy in rCTDs were identified, such as fragmentation of care among different centres, lack of education and awareness on rCTD pregnancies among midwifes, obstetricians and gynaecologists. The lack of receiving appropriate information and education on rCTDs pregnancy was also highlighted by patients and their families. The need for a holistic approach and the availability specialised pregnancy clinics with a multidisciplinary organisation as well as the provision of psychological support during all the phases around pregnancy was considered also a priority. CONCLUSION: The adoption of the NBM approach enabled a direct identification of unmet needs, and a list of possible actions was elaborated to improve the care of rCTD patients and their families in future initiatives.

RarERN Path: a methodology towards the optimisation of patients' care pathways in rare and complex diseases developed within the European Reference Networks.

BACKGROUND: In 2017, the European Commission has launched the European Reference Networks (ERNs), virtual networks involving healthcare providers across Europe. The aim of the ERNs is to tackle complex and rare diseases and conditions that require highly specialized treatment and a concentration of knowledge and resources. The ERN on rare and complex connective tissue and musculoskeletal diseases (ERN ReCONNET) is one of the 24 ERNs approved that aims to improve the management of Rare and Complex Connective Tissue and Musculoskeletal Diseases. OBJECTIVE: The RarERN Path methodology aims to create a single reference organisational model for patients' care pathways which, if applied in different contexts, helps to ensure an improved, cost-effective and patient-centred equal care to rare and complex diseases. METHODS: Starting from existing standard methods for the creation and elaboration of patients' care pathways, a specific methodology was created in order to take advantage of the distinctive and peculiar characteristics of the ERNs. Specifically, the development of the RarERN Path methodology involved different stakeholders: health economists, clinicians and researchers expert in rare and complex diseases, communication experts, experts in patients' involvement and narrative medicine and policy-makers. RESULTS: The RarERN Path methodology foresees six consecutive phases, each with different and specific aims. Specifically, the six phases are represented by: Phase 1-mapping of existing patients' care pathways and patients' stories; Phase 2-design of an optimised common patients' care pathway; Phase 3-consensus on an optimised common patients' care pathway; Phase 4-key performance indicators definition; Phase 5-refinement; Phase 6-pilot phase (optional). CONCLUSION: The application of RarERN Path to the different disease-specific and geographical contexts would help to ensure an improved, cost-effective and patient-centred equal care to rare and complex diseases across Europe as well as a possible tangible action towards the integration of ERNs into the different European healthcare systems.

One year in review 2020: economic and organisational aspects in rare and complex connective tissue diseases.

Rare and complex connective tissue diseases (rCTDs) encompass a considerable number of diseases and syndromes and their variability highly impacts on the clinical management, resulting in variable economic and organisational burden that might represent a challenge for healthcare systems. This paper is aimed at providing an overview of the most recent evidence regarding the economic and organisational impact of rCTDs. In particular, this work discusses the most relevant data on specific aspects related to health economics in rCTDs published in 2019.

Rare diseases under different levels of economic analysis: current activities, challenges and perspectives.

Rare diseases imply clinical and economic burden as well as a significant challenge for health systems. One relevant objective of the activities planned within the European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases (ERN ReCONNET) is to address the economic dimensions of rare diseases to identify, develop and suggest strategies to improve research and patients' access to orphan drugs (ODs) and highly specialised health technologies. This paper presents a preliminary review of the existing policies on rare diseases in the countries of the Network members. It also introduces and discusses the theme of how to perform health economic evaluations of rare diseases and of existing or new treatments for rare diseases. To obtain a preliminary overview aiming at defining the state of the art of rare diseases policies and initiatives in ERN ReCONNET countries, we collected and analysed the rare diseases national plans of all the eight countries of the ERN ReCONNET participants. The preliminary overview that has been performed showed that in all the ERN ReCONNET countries are in place national plans for rare diseases; however, heterogeneity exists in the reimbursement of ODs, direct provision by the healthcare system, involvement of patients' associations in decision making and implementation of clinical practice guidelines.

Comparative health technology assessment of robotic-assisted, direct manual laparoscopic and open surgery: a prospective study.

BACKGROUND: Despite many publications reporting on the increased hospital cost of robotic-assisted surgery (RAS) compared to direct manual laparoscopic surgery (DMLS) and open surgery (OS), the reported health economic studies lack details on clinical outcome, precluding valid health technology assessment (HTA). METHODS: The present prospective study reports total cost analysis on 699 patients undergoing general surgical, gynecological and thoracic operations between 2011 and 2014 in the Italian Public Health Service, during which period eight major teaching hospitals treated the patients. The study compared total healthcare costs of RAS, DMLS and OS based on prospectively collected data on patient outcome in addition to healthcare costs incurred by the three approaches. RESULTS: The cost of RAS operations was significantly higher than that of OS and DMLS for both gynecological and thoracic operations (p < 0.001). The study showed no significant difference in total costs between OS and DMLS. Total costs of general surgery RAS were significantly higher than those of OS (p < 0.001), but not against DMLS general surgery. Indirect costs were significantly lower in RAS compared to both DMLS general surgery and OS gynecological surgery due to the shorter length of hospital stay of RAS approach (p < 0.001). Additionally, in all specialties compared to OS, patients treated by RAS experienced a quicker recovery and significantly less pain during the hospitalization and after discharge. CONCLUSIONS: The present HTA while confirming higher total healthcare costs for RAS operations identified significant clinical benefits which may justify the increased expenditure incurred by this approach.

Assessment of the Economic Impact of Belimumab for the Treatment of Systemic Lupus Erythematosus in the Italian Setting: A Cost-Effectiveness Analysis.

OBJECTIVE: The purpose of this analysis is to evaluate the cost-effectiveness of belimumab, a new biological treatment specifically developed for the treatment of Systemic Lupus Erythematosus (SLE), in the Italian setting. SLE is a chronic non-organ specific autoimmune disease characterized by a disregulation of the immune system that involves many organs and systems. METHODS: A cost-effectiveness micro-simulation model with a lifetime horizon originally developed for the UK was adapted to the Italian setting. The analysis compared Standard of Care (SoC) alone vs belimumab plus SoC from a National Healthcare Service (NHS) and societal perspective. Health-economic consequences of treatments and organ damage progression were calculated. When available, Italian data were used, otherwise UK costs were converted using Purchasing Power Parities (PPPs). Utility values were based on the EQ-5D™ assessments in the belimumab clinical trials (BLISS 52 and 76). Results were discounted with 3% for costs and effects. A maximum belimumab treatment duration of 6 years was assumed and wastage costs were considered. RESULTS: Cost per life year gained (Incremental Cost-Effectiveness Ratio, ICER) and cost per Quality Adjusted Life Year (QALY) (Incremental Cost-Utility Ratio, ICUR) were €22,990 and €32,859, respectively. These values reduced to €20,119 and €28,754, respectively, when indirect costs were included. CONCLUSIONS: It may be concluded that in the Italian setting and according to the guidelines of the Italian Association of Health Economics (IAHE), belimumab was shown to be cost-effective, in terms of both ICER and ICUR, (€25-40,000/QALY).

Health technology assessment of belimumab: a new monoclonal antibody for the treatment of systemic lupus erythematosus.

OBJECTIVE: Systemic lupus erythematosus (SLE) is treated with anti-inflammatory and immunosuppressive drugs and off-label biologics. Belimumab is the first biologic approved after 50 years as an add-on therapy for active disease. This paper summarizes a health technology assessment performed in Italy. METHODS: SLE epidemiology and burden were assessed using the best published international and national evidences and efficacy and safety of belimumab were synthesized using clinical data. A cost-effectiveness analysis was performed by a lifetime microsimulation model comparing belimumab to standard of care (SoC). Organizational and ethical implications were discussed. RESULTS: Literature review showed that SLE affects 47 per 100,000 people for a total of 28,500 patients in Italy, 50% of whom are affected by active form of the disease despite SoC. These patients, if autoantibodies and anti-dsDNA positive with low complement, are eligible for belimumab. SLE determines work disability and a 2-5-fold increase in mortality. Belimumab with SoC may prevent 4,742 flares in three years being cost-effective with an incremental cost-effectiveness ratio of €32,859 per quality adjusted life year gained. From the organizational perspective, the development of clear and comprehensive clinical pathways is crucial. CONCLUSIONS: The assessment supports the use of belimumab into the SLE treatment paradigm in Italy.

Acceptability of robotic technology in neuro-rehabilitation: preliminary results on chronic stroke patients.

During the last decade, different robotic devices have been developed for motor rehabilitation of stroke survivors. These devices have been shown to improve motor impairment and contribute to the understanding of mechanisms underlying motor recovery after a stroke. The assessment of the robotic technology for rehabilitation assumes great importance. The aim of this study is to present preliminary results on the assessment of the acceptability of the robotic technology for rehabilitation on a group of thirty-four chronic stroke patients. The results from questionnaires on the patients' acceptability of two different robot-assisted rehabilitation scenarios show that the robotic approach was well accepted and tolerated by the patients.

Systemic lupus erythematosus and the economic perspective: a systematic literature review and points to consider.

Systemic lupus erythematosus (SLE) is a chronic, disabling, progressive disease, with many associated comorbidities, affecting patients during prime working years resulting in a high economic burden on society, producing high direct, indirect and intangible costs. In this article, our goals are two-fold. First, we review and discuss studies published in the period 2002-2012 concerning costs of SLE and point out gaps in the published literature. Second, we propose further research studies to advance our understanding of the economic perspective in SLE in the current area of new and emerging therapies. The literature evaluating disease costs in SLE remains limited and to date has only included a small number of countries. Despite these limitations, available studies indicate that SLE has significant socio-economic ramifications. Future studies are needed, especially to assess novel biologic therapies which have been made available or currently under investigation for SLE. An interesting approach in these new economic evaluations in SLE may be represented by the selection of the targets of the treatment to include in the cost-effectiveness and cost-utility analyses. Future treat-to-target strategies will likely include evaluation of their pharmacoeconomic implications.

A systematic literature review of the economic impact of ankylosing spondylitis.

This article reviews the last decade studies on the economic impact of ankylosing spondylitis (AS). Interestingly, a common observation is that in AS indirect costs are higher than the use of direct healthcare resources. Country, age, gender, and severity of the diseases impact on per patient annual costs AS related. Different payment and reimbursement regimes may impact on the amount and distribution of indirect costs. The differences observed among countries on absolute and relative (compared with direct costs) amounts of indirect costs can be explained with the capability of a country of actually measure productivity losses and indirect costs. Low indirect costs without other indicators should not be considered as a sign of efficiency in AS care, but may be due to an underestimation of AS-related costs; as a consequence, indirect costs may be a net loss for patients that nobody can repay. A private insurance reimbursement regime has the highest capability of inducing players to define, select and actually identify indirect costs better than in different reimbursement regimes. Therefore indirect costs may become very high in case of private insurance regimes because of their more detailed identification.

The economic impact of gout: a systematic literature review.

This article performs a systematic literature review of the last decade studies assessing the economic impact of gout. The literature review confirms the fact that gout reduces productivity and increases annual total healthcare costs, since care of gout absorbs relevant amounts of healthcare resources. One important aspect to be considered is represented by prevention and monitoring of the disease after the diagnosis, as gout is sometimes underestimated by patients and this leads to a reduced adherence to follow up and to treatment with consequences on the disease course and outcome. In fact, the lack of prevention and the scarce adherence to monitoring increase the number and costs of hospitalisation. Prevention, monitoring the level of sUA and using a urate-lowering therapy appear to have a central role for controlling gout and reducing hospitalisation, with positive advantages in terms of healthcare costs and healthcare utilisation. One limitation on the analysis of gout related costs, however, resides in the fact that the majority of the retrieved studies are retrospective and the definition of the economic impact of the disease is made difficult by differences in inclusion criteria, costs assessment, use of gout-related healthcare resources.

Systemic vasculitis: how little we know about their societal and economic burden.

This article attempts to perform an evaluation of the state of the art of the economic and societal burden of systemic vasculitis (VAs). Due to the rarity of these diseases and their variable clinical picture, few data are available in the literature on their health economic issues, and only some papers have been published that marginally examine the problem. Since VAs are severe conditions with a high medical and societal impact and determine high healthcare resource consumption, studies able to define societal, quality of life and economic burden of these pathologies are needed. Policy makers, private and public organisations involved in the care of VAs need data to programme future investment or make cost-effectiveness analysis for introducing new drugs or protocols.

Economic evaluation of da Vinci-assisted robotic surgery: a systematic review.

BACKGROUND: Health technology assessment (HTA) is frequently used when a new and expensive technology is being introduced into clinical practice. This certainly is the case with the da Vinci surgical robot, with costs ranging from $1 to $2.5 million for each unit. This systematic review documents major variability in the reported cost evaluation studies of da Vinci robot-assisted operations compared with those performed by the direct manual laparoscopic approach. METHODS: Published studies in the English language related to the period 2000-2010 were searched using economic and clinical electronic databases. RESULTS: All 11 reports included some form of cost analysis, which made it possible for the authors to extract information on certain specific economic outcomes: operating room time, hospital stay, and total costs. With the exception of two studies, the reported operating room time was higher with the robotic approach than with manual laparoscopic surgery, and the hospital stay was the same for the two techniques. Robotic surgery is significantly more expensive if the purchase and maintenance costs of the robot system are included in the total costs. Only 3 of the 11 publications included these costs. CONCLUSIONS: The disadvantage of robotic surgery is its higher costs related to purchase and maintenance of technology and its longer operating room time. However, emerging evidence shows that operating room time decreases with experience using the robot. From the HTA viewpoint, the result of this review is that the jury still is out on the HTA of da Vinci-assisted robotic surgery.