Search

Objectives To measure and explain financial toxicity (FT) of cancer in Italy, where a public healthcare system exists and patients with cancer are not expected (or only marginally) to pay out-of-pocket for healthcare.Setting Ten clinical oncological centres, distributed across Italian macroregions (North, Centre, South and Islands), including hospitals, university hospitals and national research institutes.Participants From 8 October 2019 to 11 December 2019, 184 patients, aged 18 or more, who were receiving or had received within the previous 3 months active anticancer treatment were enrolled, 108 (59%) females and 76 (41%) males.Intervention A 30-item prefinal questionnaire, previously developed within the qualitative tasks of the project, was administered, either electronically (n=115) or by paper sheet (n=69).Primary and secondary outcome measures According to the protocol and the International Society for Pharmacoeconomics and Outcomes Research methodology, the final questionnaire was developed by mean of explanatory factor analysis and tested for reliability, internal consistency (Cronbach’s α test and item-total correlation) and stability of measurements over time (test–retest reliability by intraclass correlation coefficient and weighted Cohen’s kappa coefficient).Results After exploratory factor analysis, a score measuring FT (FT score) was identified, made by seven items dealing with outcomes of FT. The Cronbach’s alpha coefficient for the FT score was 0.87 and the item-total correlation coefficients ranged from 0.53 to 0.74. Further, nine single items representing possible determinants of FT were also retained in the final instrument. Test–retest analysis revealed a good internal validity of the FT score and of the 16 items retained in the final questionnaire.Conclusions The Patient-Reported Outcome for Fighting FInancial Toxicity (PROFFIT) instrument consists of 16 items and is the first reported instrument to assess FT of cancer developed in a country with a fully public healthcare system.Trial registration number NCT03473379.

Objective In 2017, the Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA) introduced a standardised process to appraise innovativeness of medicines. Innovative medicines are provided speeder market access and dedicated funds. Innovativeness criteria are: unmet therapeutic need, added therapeutic value and quality of the evidence (Grading of Recommendations Assessment, Development and Evaluation method). We investigated the role played by these three criteria on the final decision aimed to understand how the new Italian innovativeness appraisal framework was implemented.Design A desk research gathered AIFA’s appraisal reports on innovativeness and data analyses were conducted. No patients were directly involved in this study.Setting and participants We scrutinised all 77 appraisal reports available on AIFA’s website (2017–2020).Primary and secondary outcome measures The impact of the three domains on final decision was investigated through a series of univariate analyses.Results Among 77 appraisal reports on innovativeness available, 49 (64%) and 28 (36%) were for oncology and non-oncology medicines, respectively. The appraisals were equally distributed among ‘fully innovative’ (36%), ‘conditionally innovative’ (30%) and ‘not innovative’ (34%). Added therapeutic value was the most important driver on innovativeness decision, followed by quality of the evidence. Drugs for rare diseases and with paediatric/mixed indications were appraised ‘innovative’ by a larger proportion, but no statistical significance was found.Conclusions Despite some limitations, including the moderate number of appraisals, this paper provides an insight into the determinants of innovativeness appraisals for medicines in Italy and the accuracy of the appraisal process. This has important implications in terms of transparency and accountability in the prioritisation process applied to innovative medicines.

Introduction Financial toxicity (FT) is a well-recognised problem in oncology. US-based studies have shown that: (a) cancer patients have a 2.7 times risk of bankruptcy; (b) patients who declare bankruptcy have a 79% greater hazard of death; (c) financial burden significantly impairs quality of life (QoL) and (d) reduces compliance and adherence to treatment prescriptions. The aim of the project is to develop and validate a patient-reported-outcome (PRO) measure to assess FT of cancer patients in Italy, where, despite the universal health coverage provided by the National Health Service, FT is an emerging issue.Methods and analysis Our hypothesis is that a specific FT measure, which considers the relevant sociocultural context and healthcare system, would allow us to understand the main determinants of cancer-related FT in Italy, in order to address and reduce these factors. According to the International Society for Pharmaco-economics and Outcomes Research guidelines on PROs, the project will include the following steps: (1) concept elicitation (from focus groups with patients and caregivers; literature; oncologists; nurses) and analysis, creating a coding library; (2) item generation (using a format that includes a question and a response on a 4-point Likert scale) and analysis through patients’ cognitive interviews of item importance within different coding categories to produce the draft instrument; (3) factor analysis and internal validation (with Cronbach’s alpha and test–retest for reliability) to produce the final instrument; (4) external validation with QoL anchors and depression scales. The use of the FT measure in prospective trials is also planned.Ethics and dissemination The protocol is approved by the ethical committees of all the participating centres. The project will tentatively produce a validated tool by the spring 2021. The project might also represent a model and the basis for future cooperation with other European countries, with different healthcare systems and socioeconomic conditions.Trial registration number NCT03473379.