The Importance of the Pharmacoeconomic Analyses in Drug Negotiation. A Farewell Editorial

Decision analysis is considered an essential tool that every responsible decision-maker should use to make rational, informed choices that are ideally optimal according to predefined and possibly shared criteria. In the management of public healthcare, decision analysis tends to propose more cost-effective choices for the benefit of the community, operating in a specific context of limited healthcare resources that do not allow satisfying all patient needs. The use of decision analysis in the public healthcare context inevitably refers to the bioethical value of health equity: the most cost-effective choice aims to satisfy the maximum number of patients with the scarce available resources, adopting a utilitarian interpretation of the equity criterion. Unfortunately, within the management of the public healthcare system, the use of decision analysis has faced challenges due to cultural, social, and organizational reasons. In Italy, the management of the National Healthcare System (SSN) and regions is structured into separate compartments (silos): pharmaceutical spending is managed separately from hospital and community care. Even today, managers of the pharmaceutical service must pursue the primary objective of keeping pharmaceutical spending within a predetermined maximum limit. During the early years of AIFA, this criterion hindered or at least discouraged the use of traditional pharmacoeconomic analyses in preparing Price and Reimbursement (P&R) dossiers for new drugs to be negotiated. The fundamental economic analysis required by AIFA was a simulated estimation of the budget impact of pharmaceutical spending in the first three years after the introduction of a new drug to the market. However, pharmacoeconomists from pharmaceutical companies progressively started submitting dossiers with BIA prepared according to international standards, which require evaluating the overall impact of the new drug not only on pharmaceutical spending but also on all types of healthcare costs (e.g., hospital costs, home care, etc.). Only in recent years, AIFA has explicitly valued cost-effectiveness analyses as a tool to support its decisions on drug prices and reimbursement. The recent AIFA Monitoring Report 2022, prepared by the Economic Evaluations Office (Table I), highlights that in recent years, there has been a progressive increase in the percentage of P&R dossiers including pharmacoeconomic analyses. In 2022, one hundred six dossiers, equivalent to 62% of the dossiers submitted to AIFA, were accompanied by a pharmacoeconomic study: 105/106 (99%) included Budget Impact Analysis (BIA), 73/106 (69%) also featured a Cost-Effectiveness (CEA) or Cost-Utility (CUA) analysis, and only 1/106 (1%) exclusively included a CEA. In summary, AIFA still considers BIA fundamental for negotiating the P&R of a drug intended for the Italian market. However, the percentage of dossiers including CEA studies is now significant and growing, particularly important for the negotiation of new drugs, especially orphan drugs. It should be emphasized that in the last two years, all dossiers for orphan drugs included a BIA and 86% also included a CEA. A similar trend, with slightly lower percentages, was observed for P&R dossiers of new chemical entities (Table 1). Analyzing the BIA and CEA studies presented to support the negotiation of recent innovative drugs, especially some orphan drugs, reveals that these analyses can justify very high reimbursement prices that these drugs have obtained. For instance, the purchase cost of a CAR-T therapy has reached several hundred thousand Euros in Italy. When successful, a single-dose CAR-T treatment for a young patient allows for a normal life expectancy and accumulates healthcare savings for each avoided year of illness. The importance of the pharmacoeconomic analyses in drug negotiation. A Farewell Editorial