The determinants of market access for medicines: theoretical issues and empirical evidence

To frame the determinants of drug prices within a complex regulatory system and how these determinants influence the P&R decisions of medicine, an analysis of the Italian reference context as a whole was done, starting from the trends in the use and consumption of drugs, to the analysis of the regulatory process and market access for specific categories of drugs, orphan drugs for rare diseases. Some of the system's shortcomings are the result of limited quality control as a whole over the years, which has led to the adoption of compartmentalized pharmaceutical policies without a medium-long term strategic vision integrating clinical, pharmacoeconomic, and organizational aspects. By critically analyzing the regulatory and access system that regulates the price and reimbursement of drugs, it was possible to: (i) verify the difficult coexistence, in some cases, of European early access strategies with a rapid and complete assessment of HTA at the national level (which frustrate efforts to promote faster drug access pathways); (ii) highlight how the perception of a drug's innovativeness produces different policies in the main regulatory agencies; (iii) to underline the different view of the value of a drug between the company and the payer; (iv) the use of non-standardized P&R criteria for the definition of P&R by AIFA for different products (even for the same therapeutic area); (v) as some classes of drugs, such as orphan drugs, have had a significant increase in terms of products developed thanks to strategies to encourage investment in a previously unprofitable sector, but how this increase has also led to a significant increase in expenditure due to a P&R model not related to the low epidemiology of these diseases and/or the real impact in terms of clinical benefits of these products.