Global & Regional Health Technology Assessment最新文献

Haemophilia B (HB) is a rare disease which may lead to chronic disabling arthropathy, resulting in a significant clinical, social and economic impact. In recent years, new extended half-life (EHL) factor IX concentrates produced by recombinant technology (rFIX) have been developed. They have shown significantly prolonged half-life as compared to other rFIX products and improved bleeding control when used as prophylaxis. To date, EHL rFIX products reimbursed in Italy are a recombinant coagulation factor IX produced with Fc technology (rFIXFc) and a recombinant fusion protein containing rFIX fused with recombinant albumin (rIX-FP). The results of extension studies with injection intervals with a median of almost every 14 days for the complete individualized interval prophylaxis (IP) group on rFIXFc and 21 days for a selected subgroup of patients on rIX-FP have recently been published. The aim of this analysis was to estimate the cost of prophylactic treatment with rFIXFc and rIX-FP in adult patients, in the light of new clinical evidence and current average prices in Italy. The cost of therapy was estimated on the basis of the results of extension studies, the average prices reported in regional drug tenders and assuming an average patient weight of 70 kg. The analysis estimated a cost per patient/year between €224,407 and €230,355 for rFIXFc and between €242,259 and €368,587 for rIX-FP. The sensitivity analysis confirmed the robustness of the results. The use of rFIXFc over rIX-FP proves to be the least expensive choice for the treatment of HB in Italy.

Introduction: Health state valuation and diagnostic-therapeutic pathways at the junction between non-metastatic and metastatic castration-resistant prostate cancer (CRPC) are not well documented. We aimed at: (i) estimating the disability weights (DWs) for health states across a continuum of disease from asymptomatic non-metastatic (nmCRPC) to symptomatic metastatic state (mCRPC); (ii) mapping the diagnostic-therapeutic pathway of nmCRPC in Italy.

Methods: Structured qualitative interviews were performed with clinical experts to gather information on nmCRPC clinical pathway. An online survey was administered to clinical experts to estimate DWs for four CRPC health states defined from interviews and literature review (i.e., nmCRPC, asymptomatic mCRPC, symptomatic mCRPC, mCRPC in progression during or after chemotherapy). Clinicians' preferences for health states were elicited using the Person-Trade-Off (PTO) and Visual Analogue Scale (VAS) methods. DWs associated with each health state, from 0 (best imaginable health state) and 1 (worst imaginable health state), were estimated.

Results: We found that the management of nmCRPC is heterogeneous across Italian regions and hospitals, especially with respect to diagnostic imaging techniques. DWs for PTO ranged from 0.415 (95% confidence interval [CI] 0.208-0.623) in nmCRPC to 0.740 (95% CI 0.560-0.920) in mCRPC, in progression during or after chemotherapy. DWs for VAS ranged between 0.246 (95% CI 0.131-0.361) in nmCRPC to 0.689 (95% CI 0.583-0.795) in mCRPC, in progression during or after chemotherapy.

Conclusions: Estimated DWs suggest that delaying transition to a metastatic state might ease the disease burden at both patient and societal levels.

Epilepsy is a diffuse chronic neurological disease affecting around 50 million people worldwide. The diagnostic criteria by the International League against Epilepsy must be fulfilled to diagnose the disease, which is characterized by brief and transient episodes of abnormal neuronal activity involving one or both hemispheres, depending on the epilepsy type. The diagnosis of epilepsy should be properly and timely made because patients suffering from the disease are affected not only by seizure recurrence but also by epilepsy-related psychiatric and/or cognitive comorbidities that may have a huge impact with severe professional and social implications. It is of vital importance to define a specific governance model that has to be virtuously applied into the different phases of the clinical pathway of the patients with epilepsy in order to guarantee them the best model of care possible.

Pharmacoeconomic data are widely used along drug life cycle for supporting decision-making processes on research and development, pricing and reimbursement, and market access. In this context, the incremental cost-effectiveness ratio (ICER) is the gold standard of either cost-effectiveness analyses (CEAs) or cost-utility analyses (CUAs) of pharmaceuticals and health technologies. However, the widespread use of confidentiality clauses in the agreements between pharmaceutical companies and the payers may affect the reliability of ICER value based on transparent price. The aim of this article is to evaluate a case study and simulate the impact of price confidentiality and other managed-entry agreement conditions on the ICER value. The case study was conducted selecting a CEA submitted to the Health Economic Evaluations Office of the Italian Medicines Agency by the pharmaceutical company, which specifically compared two alternative options reimbursed by the Italian NHS using confidential managed-entry agreements. So, a real model was used to collect the output of ICERs generated by the simulation model, considering price inputs of alternative options ranging from the transparent prices to the confidential net price. The simulation showed that price confidentiality may affect the estimated value of the ICER of a new medicine and, consequently, its interpretation. From a different point of view, the published ICER values may also give a completely false economic evidence if non-disclosure agreements are not taken into account. A proposal for editors of pharmacoeconomic journals to improve reliability of CEA is also discussed.

The World Health Assembly recognizes the growing economic and societal burden of neurological disorders, a leading cause of disability and the second cause of mortality in the world. In this context we analysed the socio-economic impact of epilepsy in Italy with a specific focus on hospitalizations and costs related to disability pensions (DPs) and ordinary disability allowances. In the case of epilepsy, between 2009 and 2015 we observed an alarming increasing trend for DPs (+26%), indicating that substantial expenses must be supported throughout the patients' lifetimes by both the social security system and the National Health Service (NHS) on top of the impact on caregivers. We also analysed the hospital expenditure on epilepsy through the information available in the Hospital Discharge Cards between 2015 and 2018. Almost all admissions (76% ordinary hospitalizations, 24% day hospitals) were acute (95%), followed by rehabilitation (4%) and long-term care (1%). The cost of acute and ordinary hospitalizations was by far the highest in 2018, the last year of analysis. This large expense due to hospitalizations could be reduced through the implementation of different organizational and management approaches. Our recommendation is that the policy maker should consider the best approach to ensure an early diagnosis for patients and provide early access to drugs and/or surgery. Finally, the adoption of new innovative treatments should improve effectiveness and, at the same time, reduce the expense of the NHS, of the social system as a whole, with a tangible improvement in patients' quality of life.