[Unmet clinical need and new therapeutic options.] / Unmet clinical need e nuovi farmaci.

The debate around unmet clinical need (UCN) is still very much alive. How do we define UCN? How does it influence the definition of clinically relevant outcomes in a therapeutic area? Who defines UCN? What are the consequences of recognizing different grading of UCN? In this paper we will address these questions and finally formulate proposals for the Italian context. The paper is based on a discussion within a panel of experts. This topic is even more stimulating as this work takes place in a historical period which, on the one hand, sees the start of a new course of negotiation rules recently published by AIFA and, on the other hand, poses unprecedented challenges that emerged during the pandemic crisis. The working group formulated suggestions and proposals to further enhance the role of the UCN in decision-making processes, also in the light of the new negotiation procedure, and to help refine the tools for grading the UCN and the value of medicines in the interests of patients and society as a whole.

Out of hospital sudden cardiac death in Italy: a population-based case-control study.

BACKGROUND: Sudden cardiac death (SCD) is a major cause of death in western countries, with coronary heart disease (CHD) being the basis of over 70% of SCD. Incidence in high-CHD risk countries has already been studied, but this information is not available for Mediterranean low-CHD risk countries. Incidence is of paramount importance when cost-effectiveness rate of actions against SCD must be estimated. METHODS: We estimated the incidence of SCD and its potential risk associated with clinical variables, by a means of a case-control study in a general practice setting. The enrolled general practitioners (GPs) provided data about the total number of their patients, and identified all their patients who suffered an out-of-hospital SCD during the previous 365 days. Two age-matched and gender-matched controls visiting GPs office after the SCD selection were also selected. We used a structured questionnaire to obtain information about potential risk factors for SCD. Covariates that were univariately associated with SCD were included in the multivariate regression analyses. RESULTS: In a population of 297 340 (age greater than 14 years), a total of 230 cases were identified (0.77 per 1000 individuals), mostly occurring at home and among persons with known high cardiovascular risk. In the multivariate analysis only CHD (OR: 1.67; 95% CI: 1.09-2.58), arrhythmia (OR: 2.2; 95% CI: 1.3-3.9), obesity (OR: 2.3; 95% CI: 1.5-3.6), alcohol abuse (OR: 1.8; 95% CI: 1.2-2.7), and family history of CHD (OR 3.1; 95% CI: 1.8-5.3) resulted in a significant association with SCD. CONCLUSIONS: The incidence of SCD in Italy is lower than that reported in high-CHD risk population, most of the cases occurring at home and among persons with known high cardiovascular risk. Implementing recommendations for these patients seems to be the most effective strategy to reduce the incidence of SCD.

Diagnostic pathway of syncope and analysis of the impact of guidelines in a district general hospital. The ECSIT study (epidemiology and costs of syncope in Trento).

BACKGROUND: The ECSIT study was aimed at evaluating the hospital management of syncope patients, at comparing the appropriateness and costs of the hospital diagnostic pathway before (phase 1) and after (phase 2) the introduction of new guidelines and at analyzing the physicians' compliance to the guidelines. METHODS: All syncope patients admitted to the emergency room between August 1 and October 31, 1999 (phase 1) and between March 1 and May 31, 2000 (phase 2) were enrolled and their clinical records were analyzed in a blind fashion. RESULTS: During the study 538 consecutive patients came to the emergency room for syncope with a hospitalization rate of 53% in phase 1 (n = 151) and of 42% in phase 2 (n = 107). The in-hospital stay increased from 9 days in phase 1 to 11.3 days in phase 2 and diagnostic tests from 2.6 per patient (phase 1) to 2.9 per patient (phase 2) with total costs that rose from [symbol: see text] 3,474 to [symbol: see text] 3,647. Patients with no diagnosis decreased from 51 to 45.8% and the principal causes were identified as vascular brain disease (36.1 vs 33.7%) and neurally-mediated mechanisms (35.3 vs 42.2%). CONCLUSIONS: Despite the high costs of syncope management, the appropriateness and efficacy of the hospital diagnostic pathway remains far from ideal and simply introducing new guidelines seems unable to modify clinical practice.