The economic burden of pediatric growth hormone deficiency in Italy: a cost of illness study.

PURPOSE: Growth hormone deficiency (GHD) is a rare condition with a worldwide prevalence of 1 patient in 4000 to 10,000 live births, placing a significant economic burden on healthcare systems. The aim of this study is to generate evidence on the economic burden of children and adolescents with GHD treated with rhGH and their parents in Italy. METHODS: A cost of illness analysis, adopting the prevalence approach, has been developed, producing evidence on the total annual cost sustained by the Italian National Health System (NHS) and by the society. The study is based on original data collected from a survey conducted among Italian children and adolescents with GHD and their parents. RESULTS: 143 children/adolescents with GHD and their parents participated to the survey, conducted from May to October 2021. Patients had a mean age of 12.2 years (SD: 3.1) and were mostly males (68.5%). The average direct healthcare cost sustained by the NHS was € 8,497.2 per patient/year; adding the out-of-pocket expenses (co-payments and expenses for private healthcare service), the total expense was € 8,568.6. The indirect costs, assessed with the human capital approach, were € 847.9 per patient/year. The total of direct and indirect cost is € 9,345.1 from the NHS perspective, and € 9,416.5 from a social perspective. The total cost incurred by the Italian NHS for children with GHD (range: 5,708-8,354) was estimated in € 48.5-71.0 million, corresponding to 0.04-0.06% of the total Italian public health expense in the year 2020. CONCLUSIONS: The total annual cost for GHD children is close to € 10,000, and is mainly due to the cost of rhGH treatment. This cost is almost entirely sustained by the NHS, with negligible out-of-pocket expenses. The economic burden on the Italian NHS for the health care of established GHD children is fourfold higher than the prevalence of the disease in the overall Italian population.

Quality of life in children and adolescents with growth hormone deficiency and their caregivers: an Italian survey.

PURPOSE: The aim of this study was to produce evidence on quality of life (QoL) among Italian growth hormone deficiency (GHD) children and adolescents treated with growth hormone (GH) and their parents. METHODS: A survey was conducted among Italian children and adolescents aged 4-18 with a confirmed diagnosis of GHD and treated with GH therapy and their parents. The European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and the Quality of Life in Short Stature Youth (QoLISSY) questionnaires were administered between May and October 2021 through the Computer-Assisted Personal Interview (CAPI) method. Results were compared with national and international reference values. RESULTS: The survey included 142 GHD children/adolescents and their parents. The mean EQ-5D-3L score was 0.95 [standard deviation (SD) 0.09], while the mean visual analogue scale (VAS) score was 86.2 (SD 14.2); the scores are similar to those of a reference Italian population aged 18-24 of healthy subjects. As for the QoLISSY child-version, compared to the international reference values for GHD/ idiopathic short stature (ISS) patients, we found a significantly higher score for the physical domain, and lower scores for coping and treatment; compared to the specific reference values for GHD patients, our mean scores were significantly lower for all domains except the physical one. As for the parents, we found a significantly higher score for the physical domain, and a lower score for treatment; compared to reference values GHD-specific, we found lower score in the social, emotional, treatment, parental effects, and total score domains. CONCLUSIONS: Our results suggest that the generic health-related quality of life (HRQoL) in treated GHD patients is high, comparable to that of healthy people. The QoL elicited by a disease specific questionnaire is also good, and comparable with that of international reference values of GHD/ISS patients.

The role of procalcitonin outside of the Intensive Care Unit (ICU): a multidisciplinary approach.

OBJECTIVE: Biochemical markers are commonly used in medicine to guide diagnostic investigation or therapy duration and/or monitor treatment efficacy. Due to the emergence and spread of antimicrobial resistance, markers able to prompt a more rational use of antimicrobial therapy are regarded with the greatest attention. Procalcitonin (PCT) certainly stands out among others, yet its role must be better established especially outside of the critical care area. Data about PCT utilization in non-critical patients, optimal negativity cut-offs as well as a protocol for measurement timing are all lacking. MATERIALS AND METHODS: To address these issues, a focus group was set up to propose and endorse shared statements regarding the most beneficial use of PCT in real life as infection marker for non-critical patients, based on the authors' experience and a review of recent literature. RESULTS: A group of nine experts in the fields of Infectious Diseases, Internal Medicine, Microbiology, Clinical Chemistry, Surgery and Medical Economics participated in the discussion of nine pre-specified statements. CONCLUSIONS: The potential role for PCT in differentiating infectious and non-infectious clinical syndromes and guiding antimicrobial therapy discontinuation was acknowledged. Moreover, a shared measurement protocol and desirable cut-offs for the non-critical area were proposed. Finally, observations were made about a reasonable selection of the patient population to be tested.

A randomised controlled trial of the effectiveness of a program for early detection and treatment of depression in primary care.

OBJECTIVE: There is considerable uncertainty about whether depression screening programs in primary care may improve outcomes and what specific features of such programs may contribute to success. We tested the effectiveness of a program involving substantial commitment from local mental health services. METHODS: Prospective, randomised, patient- and evaluator-masked, parallel-group, controlled study. Participants were recruited in several urban primary care practices where they completed the PC-SAD screener and WHOQOL-Bref. Those who screened positive and did not report suicidal ideation (N=115) were randomised to an intervention group (communication of the result and offer of psychiatric evaluation and treatment free of charge; N=56) or a control group (no feedback on test result for 3 months; N=59). After 3 months, 100 patients agreed to a follow-up telephone interview including the administration of the PC-SAD5 and WHOQOL-Bref. RESULTS: Depression severity and quality of life improved significantly in both groups. Intent-to-treat analysis showed no effect of the intervention. As only 37% of patients randomised to the intervention group actually contacted the study outpatient clinic, we performed a per-protocol analysis to determine whether the intervention, if delivered as planned, had been effective. This analysis revealed a significant positive effect of the intervention on severity of depressive symptoms, and on response and remission rate. Complier average causal effect analysis yielded similar results. CONCLUSION: Due to the relatively small sample size, our findings should be regarded as preliminary and have limited generalizability. They suggest that there are considerable barriers on the part of many patients to the implementation of depression screening programs in primary care. While such programs can be effective, they should be designed based on the understanding of patients' perspectives.

The impact of biologic therapy in chronic plaque psoriasis from a societal perspective: an analysis based on Italian actual clinical practice.

OBJECTIVE: Psoriasis is one of the most common forms of chronic dermatitis, affecting 2-3% of the worldwide population. It has a serious effect on the way patients perceive themselves and others, thereby prejudicing their quality of life and giving rise to a significant deterioration in their psycho-physical well-being; it also poses greater difficulties for them in leading a normal social life, including their ability to conduct a normal working life. All the above-mentioned issues imply a cost for the society. This study proposes to evaluate the impact on societal costs for the treatment of chronic plaque psoriasis with biologics (etanercept, infliximab and adalimumab) in the Italian clinical practice. METHOD: A prospective observational study has been conducted in 12 specialized centres of the Psocare network, located throughout Italy. Direct and indirect costs (as well as the health-related quality of life of patients with plaque psoriasis undergoing biologic treatments) have been estimated, while the societal impact has been determined using a cost-utility approach. RESULTS: Non-medical and indirect costs account for as much as 44.97% of the total cost prior to treatment and to 6.59% after treatment, with an overall 71.38% decrease. Adopting a societal perspective in the actual clinical practice of the Italian participating centres, the ICER of biologic therapies for treating plaque psoriasis amounted to €18634.40 per QALY gained--a value far from the €28656.30 obtained by adopting a third-party payer perspective. CONCLUSION: Our study confirms that chronic psoriasis subjects patients to a considerable burden, together with their families and caregivers, stressing how important it is to take the societal perspective into consideration during the appraisal process. Besides, using data derived from Italian actual practice, treatment with biologics shows a noteworthy benefit in social terms.

The impact of real practice inappropriateness and devices' inefficiency to variability in growth hormone consumption.

INTRODUCTION: Growth hormone (GH) consumption is the object of a particular attention by regulatory bodies, due to its financial impact; nevertheless, GH treatment has been demonstrated to be cost-effective and is, therefore, usually reimbursed by public health service systems. In Italy, significant differences in GH consumption between regions have been recorded. Different appropriateness in real practice is a possible explanation, but the proportion of drug wasted due to different combinations of therapeutic regimes and types of devices used in drug administration is a complementary explanation. Aim of the study is, therefore, to determine how much of the variability in GH consumption is actually due to differences in clinical practice, and how much to waste. MATERIALS AND METHODS: A model was settled to estimate the population with indication for GH administration, separately for children, transition subjects and adults, based on both the scientific evidence available and directly collected clinical evaluations. A systematic literature search was conducted using Cochrane Library (HTA and NHSEE) databases, Medline via Ovid, Econlit via Ovid, Embase. CONCLUSION: The model applied to the Italian population showed that there was apparently unexplainable over-prescription and potential under-prescription in various regions, ranging from 20 to 40 % less than the estimated theoretical consumption to over 200 %. Wastage, at level of single device, could amount to as much as 15 % of the consumption, demonstrating that price per mg is not in general a good proxy of the cost per mg of therapy. Our estimates of the wastage shows a significant potential gap in the model assessment of the HTA bodies, as far as they do not explicitly take into account the issue of wastage and, consequently, the actual variability in local clinical practice.

Health-related quality of life in psoriasis: an analysis of Psocare project patients.

AIM: Psoriasis is a common, chronic, immune-mediated skin disorder that may be complicated by psoriatic arthritis in up to one-third of patients. Psoriasis treatments are increasingly effective, yet more expensive, thus requiring rational decision-making on interventional priorities. The ability to perform cost-utility analyses is hindered by the lack of algorithms that allow the inference of utility measures, like QALY, from specific dermatological health-related quality-of-life (HR-QoL) measures (e.g. Dermatology Life Quality Index [DLQI]). This study aimed to assess whether psoriasis-related HR-QoL data (DLQI) could be used to obtain utility measures for use in economic analyses. METHODS: Psoriasis patients attending 11 Italian Psocare project treatment centers over a 19-day period were enrolled and completed a questionnaire, including several HR-QoL scales and sociodemographic/clinical data, and underwent a clinical examination. Data were subjected to a Multiple Correspondence Analysis and multiple regression analysis to determine the contribution of single items to the HR-QoL. RESULTS: DLQI and Psychological General Well-Being Index (PGWBI) scores were most closely correlated with the EuroQol health status index. Age and gender were considered confounding factors, while pain and arthritis contributed significantly to HR-QoL deterioration. For disease severity, the need for hospitalization and the number of examinations, but not the Psoriasis Area Severity Index (PASI), contributed to HR-QoL deterioration. CONCLUSION: Recent historical clinical and HR-QoL data from psoriasis patients can reproducibly define a health status index, such as the EuroQol SD-5Q, that could be used reliably to estimate QALYs for use in cost-utility analyses to compare the cost-benefit profiles of competing therapies.

[Management control of cardiology: the experience of a departmental unit]. / Controllo di gestione della cardiologia: esperienza di una unità dipartimentale.

In most Italian hospitals, sanitary reform is being applied, while at the same time a new organization of the National Health System is being planned. The director of the medical hospital (head doctor) is becoming more and more involved in management and this aspect has modified his professional attributes. Cardiology is a branch of medicine that, through its scientific preparatory work consisting in debates, management courses, ethics, and production of managerial software, is closer to applying the reform without risking improper administrative aspects. This, obviously, comes about after thoroughly reviewing past work methods and the need to have an administrative organization, which allocates efficient use of manpower and materials, helping to eliminate any sources of inefficiency. The logical procedure foresees an actual analysis in terms of sanitary needs and availability of resources, and so attempting to better balance and harmonize both aspects of the problem. Certainly, the acquisition of theoretical norms and practices, which today are present because of the upsurge in training courses for doctors, is not enough to guarantee the achievement of optimal results. Furthermore, we find that theoretical models need to be validated and adapted to real work situations in the public hospital sector. This paper proposes, therefore, to explain the managerial experiences achieved in actual work situations at the Cardiology Department Unit of the San Giovanni Addolorata Hospital in Rome. In particular, it shows that in order to reach its clinical and economical objectives, it is essential to make available correct informative support for strategic and operational decisions. We can observe that there is a continuing lack of computer support systems being integrated into the present organization of most cardiology units. The use of software distributed to cardiology units from the Associazione Nazionale Medici Cardiologi Ospedalieri (ANMCO) has enabled us to partially remedy the above-mentioned inadequacy and, at the same time, has pointed out the limits of automated support with regards managerial needs which are rapidly changing and becoming increasingly more complex. The experience described shows the possibility of uniting an efficient organization, from a clinical point of view, with an adequate managerial automation without including any flaws from the previous organizational systems. Based on achieved results and the difficulties encountered, further development in computer technology support to hospital management is suggested. This paper, in particular, stresses the need to improve automated informative support available to doctors in managerial positions. This improvement would allow us to immediately evaluate "internal" administrative matters, have the data available for budget planning and analysis of budget variations, and be better able to integrate this with clinical data. The scientific community can give further assistance by promoting the genesis of data for a sensible bench-marking within medical organizations which is the only means sufficient to give reliable and clinically appropriate indications on the possibility of increasing its own structural efficiency.