RESUMEN
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.
Asunto(s)
Costo de Enfermedad , Costos de la Atención en Salud , Hormona de Crecimiento Humana , Humanos , Masculino , Italia/epidemiología , Niño , Femenino , Adolescente , Hormona de Crecimiento Humana/deficiencia , Hormona de Crecimiento Humana/economía , Hormona de Crecimiento Humana/uso terapéutico , Costos de la Atención en Salud/estadística & datos numéricos , Gastos en Salud/estadística & datos numéricos , Enanismo Hipofisario/economía , Enanismo Hipofisario/epidemiología , Enanismo Hipofisario/tratamiento farmacológico , Trastornos del Crecimiento/economía , Trastornos del Crecimiento/epidemiología , Prevalencia , PadresRESUMEN
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.
Asunto(s)
Enanismo Hipofisario , Hormona de Crecimiento Humana , Humanos , Adolescente , Calidad de Vida/psicología , Cuidadores , Enanismo Hipofisario/tratamiento farmacológico , Enanismo Hipofisario/epidemiología , Enanismo Hipofisario/psicología , Italia/epidemiología , Hormona de Crecimiento Humana/uso terapéutico , Hormona del Crecimiento , Encuestas y CuestionariosRESUMEN
BACKGROUND: Cushing's syndrome (CS) is a rare clinical condition caused by excessive cortisol secretion from adrenal glands. CS is associated with increased mortality and morbidity; therefore, a prompt diagnosis and an effective therapeutic approach are strongly necessary to improve the patient's clinical management. The first-line treatment for CS is surgery, while medical treatment has historically played a minor role. However, thanks to the availability of novel compounds, the possibility of improving hypercortisolism control using different drug combinations emerged. PURPOSE: No absolute recommendations are available to guide the therapeutic choice for patients with CS and, consequently, the awareness of unmet needs in CS management is growing. Although new data from clinical trials are needed to better define the most appropriate management of CS, an expert consensus approach can help define unmet needs and optimize the current CS management and treatment. METHODS: Twenty-seven endocrinologists from 12 Italian regions, working among the main Italian referral centers for hospital endocrinology where they take care of CS patients, were involved in a consensus process and used the Delphi method to reach an agreement on 24 statements about managing CS patients. RESULTS: In total, 18 statements reached a consensus. Some relevant unmet needs in the management of CS were reported, mainly related to the lack of a pharmacological treatment successful for the majority of patients. CONCLUSION: While acknowledging the difficulty in achieving complete disease control, a significant change in CS management requires the availability of medical treatment with improved efficacy and safety over available therapeutic options at the time of the current study.
Asunto(s)
Síndrome de Cushing , Humanos , Síndrome de Cushing/tratamiento farmacológico , Síndrome de Cushing/diagnóstico , Endocrinólogos , Glándulas Suprarrenales , ItaliaRESUMEN
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.
Asunto(s)
Productos Biológicos/economía , Productos Biológicos/uso terapéutico , Costos Directos de Servicios/estadística & datos numéricos , Costos de los Medicamentos/estadística & datos numéricos , Psoriasis/tratamiento farmacológico , Psoriasis/economía , Adalimumab/economía , Adalimumab/uso terapéutico , Adolescente , Adulto , Anciano , Antiinflamatorios no Esteroideos/economía , Antiinflamatorios no Esteroideos/uso terapéutico , Enfermedad Crónica , Costo de Enfermedad , Análisis Costo-Beneficio , Etanercept/economía , Etanercept/uso terapéutico , Femenino , Humanos , Infliximab/economía , Infliximab/uso terapéutico , Italia , Masculino , Persona de Mediana Edad , Estudios Prospectivos , Años de Vida Ajustados por Calidad de Vida , Adulto JovenRESUMEN
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.