Objectives This study aimed to provide normative data obtained in response to the EQ-5D-5L questionnaire in Italy and compare this with data from other countries. Methods A sample of the Italian adult population (aged ≥ 18 years) was recruited and interviewed online using videoconferencing software (Zoom) between November 2020 and February 2021. The distribution of answers was estimated as per the descriptive system of the EQ-5D-5L, and descriptive statistics were calculated for the EQ VAS score and EQ-5D-5L index value in the whole sample and relevant subgroups. An ordinary least square (OLS) regression was performed to evaluate the impact of sociodemographic variables on EQ-5D-5L results. Lastly, a comparison was made with EQ-5D-5L population norms of other countries. Data analysis was performed using Microsoft Excel and Stata 13. Results Overall, 1182 people representative of the Italian population (2020) in terms of sex and geographical area responded to the survey. Of the 3125 potential EQ-5D-5L health states, only 106 (3.4%) were selected, and the ‘11111’ and ‘11112’ states were chosen by half of the participants. In terms of EQ-5D-5L dimensions, the frequency of any problems (from slight to extreme) associated with anxiety and depression was high among the very young (18–24 years, 56.0%) and in women of all ages (49.7%). The mean index value (± standard deviation [SD]) was 0.93 (± 0.11) for the entire sample and gradually decreased with age, moving from 0.95 (± 0.06) in the youngest group (18–24 years) to 0.91 (± 0.13) in the oldest age group (≥ 75 years). Similarly, the mean EQ VAS score (± SD) was 81.8 (± 13.5), and decreased from 87.0 (± 8.9) in the 18–24 years age group to 75.1 (± 16.4) among participants > 75 years of age. The existence of self-reported chronic conditions (e.g., cardiovascular disease), female sex, and social assistance recipiency were negatively associated with the EQ-5D index value, while the EQ VAS score was significantly lower in people with chronic conditions and aged > 55 years. Conversely, higher income levels had a positive impact on both the EQ-5D index value and the EQ VAS score. Lastly, both the EQ-5D index value and EQ VAS score in Italy were, on average, higher than in most European countries. Conclusions EQ-5D-5L population norms provide useful insights into the health status of the Italian population and can be used as a reference for other surveys using the same instrument. Supplementary Information The online version contains supplementary material available at 10.1007/s40258-022-00772-7.

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Early access programs (EAPs) generally refer to patient access to medicines/indications before marketing authorization, possibly extended to price and reimbursement approval. These programs include compassionate use, which is usually covered by pharmaceutical companies, and EAPs reimbursed by third-party payers. This paper aims at comparing EAPs in four European countries (France, Italy, Spain, UK) and providing empirical evidence on EAPs in Italy. The comparative analysis was conducted through a literature review (including scientific and grey literature), complemented by 30-min semi-structured interviews with local experts. The Italian empirical analysis employed data available on the National Medicines Agency website. Although EAPs are very different across countries, they exhibit some common features: (i) eligibility criteria refer to the absence of valid therapeutic alternatives and a presumed favourable risk–benefit profile; (ii) payers do not allocate a pre-determined budget to these programs; (iii) total spending on EAPs is unknown. The French EAPs seem to be the most structured, financed through social insurance, covering pre-marketing, post-marketing and pre-reimbursement phases and providing for data collection. Italy’s approach to EAPs has been varied, with several programs covered by different payers, including the cohort-based 648 List (for both early access and off-label use), the nominal-based 5% Fund, and Compassionate Use. Most applications to EAPs are from the Antineoplastic and immunomodulating drug class (ATC L). Some 62% of indications in the 648 List are either not under clinical development or have never been approved (pure off-label use). For those subsequently approved, most approved indications coincide with those covered through EAPs. Only the 5% Fund provides data on economic impact (€ 81.2 million in 2021; average cost per patient € 61.5K). Diverse EAPs are a possible source of inequalities in access to medicines across Europe. A harmonization of these programs, though difficult to achieve, could be modelled on the French EAPs and provide key advantages, not least of which a common effort to collect real-world data in parallel with clinical trials and clear separation between EAPs and off-label use programs.

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.

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