Monitoring and assessing diagnostic-therapeutic paths with healthcare utilization databases: experiences, concerns and challenges

The aim of this thesis is to provide the methodology used to develop and validate population-based prognostic scores, and to assess the effectiveness and cost-effectiveness of the diagnostic-therapeutic path of diabetes, using the healthcare utilization databases (or administrative databases) of Italian regions. Thus, the thesis is structured into the following three main parts. First, the reasons to justify the need of real-world studies in addition to evidence from randomized controlled trials, the definitions of real-world data and real-world evidence, and an overview of the Italian healthcare utilization databases are given. Second, because patients should be monitored according to their risk to experience adverse outcomes (e.g., all-cause mortality, hospital admissions), prognostic scores could be used. However, the main limitation in the use of pre-existing score is that they are usually developed in countries different from Italy and from hospital-based or pharmacy-based surveys, so hindering their applicability to all beneficiaries of the National Health Service. Therefore, two population-based prognostic scores were developed and validated using data from some Italian regions. The usefulness of one of these scores (i.e., the so-called Multisource Comorbidity Score) in the risk adjustments and as a tool for health policy planning is also shown. Third, tracing the work carried out from the “Monitoring and assessing care pathways” working group of the Italian Ministry of Health, a description of the following activities is provided: I. the development of process indicators to monitor and assess the quality of care of patients suffering from some chronic disease; II. the comparison of care quality between regions; III. the validation of the diabetes care indicators with respect to selected outcomes (i.e., the assessment of their effectiveness); IV. the assessment of the costs from the National Health Service perspective (calculated by the amount that the Regional Health Authority reimbursed to health providers) according to different levels of adherence to the diagnostic-therapeutic path of diabetes. Finally, the Beaver® regional research platform, able to compute the set of process and outcome indicators defined by the Health Ministry and to generate evidence on effectiveness and cost-effectiveness profile, is described.