The role of patient-reported outcomes in reimbursement decisions and drug innovation in Italy
Open Access
- 9 February 2023
- journal article
- research article
- Published by Aboutscience Srl in Global and Regional Health Technology Assessment
- Vol. 10, 12-17
- https://doi.org/10.33393/grhta.2023.2514
Abstract
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 recognition by the Italian Medicines Agency (AIFA). 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.Keywords
This publication has 5 references indexed in Scilit:
- A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016)Value in Health, 2019
- Payer Perspectives on Patient-Reported Outcomes in Health Care Decision Making: Oncology ExamplesJournal of Managed Care & Specialty Pharmacy, 2017
- Article Commentary: Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs)Health Services Insights, 2013
- Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidanceHealth and Quality of Life Outcomes, 2006
- Published by Georg Thieme Verlag KG