Il ruolo degli esiti riferiti dal paziente nelle decisioni di rimborso e innovativita dei farmaci in Italia

The role of patient-reported outcomes in reimbursement decisions and drug innovation in Italy The attention paid by regulatory authorities to patient-reported outcomes (PROs) and their related measures (PROMs) has progressively increased to allow for a more complete description of the patient's health status. This work aimed at investigating the use of PROs/PROMs in relation to reimbursement agreement and innovativeness The list of European Public Assessment Reports (EPARs), downloaded from the European Medicine Agency (EMA) website, was analyzed to identify drugs authorized between 2017 and 2021. The associations between the use of PROs/PROMs in the EPARs and the reimbursement and innovativeness status in Italy were explored through Chi2 test and logistic regression. In addition, each innovativeness report was analyzed to identify any further evidence about the consideration of PROs/PROM in the decision-making process. Of the 403 EPARs analyzed, 197 (48.9%) reported the use of PROs/PROMs. A subsample of 229 drugs with a reimbursement class (A, H, or C) officially assigned by AIFA was further investigated. Of them, 49.3% reported the use of PROs/PROMs. The Chi2 test revealed a statistically significant association between the use of PROs/PROMs and innovativeness (p = 0.001) and orphan status (p = 0.012). In the logistic regression, orphan status and availability of PROs/PROMs are significant predictors of the innovativeness status. However, of the 46 innovative drugs, only 9 (19.6%) explicitly reported a consideration of a PRO/PROM in their innovativeness reports. Robust instrument's validation, reliable data collection and growing potential's awareness are keys to increase the consideration of PROs at every step of the drug development process.

Automated summary

The role of patient-reported outcomes (PROs) and their related measures (PROMs) in reimbursement decisions and drug innovation in Italy is explored. The use of PROs/PROMs in European Medicine Agency (EMA) authorized drugs is analyzed, with 48.9% of drugs reporting their use. There is a significant association between the use of PROs/PROMs and innovativeness, although only 19.6% of innovative drugs explicitly consider PROs/PROMs in their reports.