Nuovi farmaci per le patologie croniche. Individuazione delle popolazioni target con i real world data

The target populations for new drugs, in particular when these are labelled for the treatment of chronic diseases, are a central point of health planning as regulatory agencies make their decisions on pharmacoeconomic analyses performed on real-world data, health institutions have the need to plan care pathways starting from the identification of subjects requiring a specific care process and research has recognized real world evidence (RWE) studies as a complementary approach to clinical trials. On the topic of target populations for new drugs for chronic diseases, the first working group of the MaCroScopio project (observatory on chronic diseases) was launched, in which various stakeholders participated, whose reflections have been collected in this consensus document. Starting from the entry of RWE into national and international regulatory procedures, as a strategy for identifying target populations, the document describes the advantages and limitations of administrative health databases as a data source to achieve this goal. In the document is highlighted the need to adapt the identifying algorithms according to the aim to be achieved: exploratory aim, economic/organizational scope, planning/evaluation purpose in order to set up the health policy in relation to the positioning of a new drug in a given care path. Finally, the article points out the main methodological challenges that all those interested in this topic are called to face: the complementarity between RWE and randomized controlled clinical trials, the need to measure and evaluate the complexity and variability of clinical reality, as well as the opportunity to carry out validation studies of the identification algorithms of the target populations. The resulting consensus document, therefore, intends to lay the foundations for the application of the epidemiological methodology and the use of its results in the area of planning/evaluation of care interventions, in order to identify their effectiveness, quality and sustainability.