Confirmatory validation analysis of the PROFFIT questionnaire to assess financial toxicity in cancer patients.

BACKGROUND: The Patient Reported Outcome for Fighting FInancial Toxicity (PROFFIT) questionnaire was developed to measure financial toxicity (FT) and identify its determinants. The aim of the present study was to confirm its validity in a prospective cohort of patients receiving anticancer treatment. PATIENTS AND METHODS: From March 2021 to July 2022, 221 patients were enrolled at 10 Italian centres. Selected items of the EORTC-QLQ-C30 questionnaire represented the anchors, specifically, question 28 (Q-28) on financial difficulties, and questions 29-30 measuring global health status/quality of life (HR-QOL). The study had 80% power to detect a 0.20 correlation coefficient (r) between anchors and PROFFIT-score (items 1-7, range 0-100, 100 indicating maximum FT) with bilateral alpha 0.05 and 80% power. Confirmatory factor analysis was conducted. FT determinants (items 8-16) were described. RESULTS: Median age of patients was 65 years, 116 (52.5%) were females, 96 (43.4%) had low education level. Confirmatory factor analysis confirmed goodness of fit of the PROFFIT-score. Significant partial correlation of PROFFIT-score was found with Q-28 (r = 0.51) and HR-QOL (r = -0.23). Mean (SD) PROFFIT-score at baseline was 36.5 (24.9); it was statistically significantly higher for patients living in South Italy, those with lower education level, those who were freelancer/unemployed at diagnosis and those who reported significant economic impact from the COVID-19 pandemic. Mean (SD) scores of determinants ranged from 17.6 (27.1) for item 14 (support from medical staff) to 49.0 (36.3) for item 10 (expenses for medicines or supplements). PROFFIT-score significantly increased with worsening response to determinants. CONCLUSIONS: External validation of PROFFIT-score in an independent sample of patients was successful. The instrument is now being used in clinical studies.

The association of financial difficulties with clinical outcomes in cancer patients: secondary analysis of 16 academic prospective clinical trials conducted in Italy.

BACKGROUND: Cancer may cause financial difficulties, but its impact in countries with public health systems is unknown. We evaluated the association of financial difficulties with clinical outcomes of cancer patients enrolled in academic clinical trials performed within the Italian public health system. PATIENTS AND METHODS: Data were pooled from 16 prospective multicentre trials in lung, breast or ovarian cancer, using the EORTC quality of life (QOL) C30 questionnaire. Question 28 scores financial difficulties related to disease or treatment in four categories from 'not at all' to 'very much'. We defined financial burden (FB) as any financial difficulty reported at baseline questionnaire, and financial toxicity (FT) as score worsening in a subsequent questionnaire. We investigated (i) the association of FB with clinical outcomes (survival, global QOL response [questions 29/30] and severe toxicity), and (ii) the association of FT with survival. Multivariable analyses were performed using logistic regression models or the Cox model adjusting for trial, gender, age, region and period of enrolment, baseline global QOL and, where appropriate, FB and global QOL response. Results are reported as odds ratio (OR) or hazard ratio (HR) with 95% confidence intervals (CI). RESULTS: At baseline 26% of the 3670 study patients reported FB, significantly correlated with worse baseline global QOL. FB was not associated with risks of death (HR 0.94, 95% CI 0.85-1.04, P = 0.23) and severe toxicity (OR 0.90, 95% CI 0.76-1.06, P = 0.19) but was predictive of a higher chance of worse global QOL response (OR 1.35, 95% CI 1.08-1.70, P = 0.009). During treatment, 2735 (74.5%) patients filled in subsequent questionnaires and 616 (22.5%) developed FT that was significantly associated with an increased risk of death (HR 1.20, 95% CI 1.05-1.37, P = 0.007). Several sensitivity analyses confirmed these findings. CONCLUSION: Even in a public health system, financial difficulties are associated with relevant cancer patients outcomes like QOL and survival. CLINICAL TRIALS NUMBER: Any registered clinical trial number should be indicated after the abstract.

[Innovative medicines and market access agreements]. / Médicaments innovants et contrats d'accès au marché

Market Access Agreements (MAA) for drugs have emerged in response to the need to control health expenditures, as well as to the uncertainty about the true benefit of a drug. It is possible to group MAA in two types of agreements: financial agreements and outcome-based agreements. MAA is a growing trend and is shifting towards conditional access. However, the willingness to use these contracts and their implementation differ across countries, and some are still resistant to put them in place. The MAA challenges to overcome encompass the complexity of the schemes, the administrative burden and the difficulty of evaluating MAA. It is likely that these agreements might experience further evolution in the future to become a faster pathway for therapeutic innovations, at a fair price.

New funding arrangements in the Italian National Health Service.

In the 1990s, the Italian National Health Service (INHS) experienced a major reform introducing regionalization, quasi-markets and managerialism. The combination of quasi-markets and regionalization has produced an interesting scenario: 21 Regional Governments designing their own organizational and funding models to achieve the desired combination of equity, efficiency, freedom of choice and cost containment. This paper reports the results of a research project carried out in 1998-99 to identify such models, verify their actual states of implementation and analyse the resulting incentives for individual health-care organizations. Overall, most Regions have designed their models according to the 'LHU-centred' template, under which most public hospitals remain under Local Health Unit (LHU) control, LHUs are funded by their Regions on a capitation basis and each LHU is expected to reimburse other LHUs, Independent INHS Hospitals (IHs) and accredited private providers for services supplied to its residents. Reimbursements are activity-based according to Regional fee schedules. The major exception is Lombardy, Italy's largest and wealthiest Region, which has formally opted for the 'purchaser-provider split' template, with LHUs acting mostly as purchasers while IHs and accredited private professionals and organizations act as providers. In practice, however, many Regions still show significant features of the traditional cost-reimbursement system.

The new pharmaceutical policy in Italy.

Pressed by an impressive series of corruption scandals and by a change of attitude towards cost-containment, the Italian pharmaceutical sector's regulatory environment was radically changed in 1994. Regulatory power was concentrated on a national technical body (CUF) and a new set of measures was taken, including a nationwide drug expenditure budget, a redefinition of both the positive list and the cost-sharing rules, and new price-setting models. As a result, in the period 1993-1996, nominal expenditures decreased by about L 1600 billion (ECU 83.6 billion at 1997 exchange rate), that is from 13.3% to 11.0% of current National Health Service (NHS) expenditure. While in the 1980s Italy was one of the most generous countries in funding pharmaceuticals, it is now one of the most parsimonious. Although the overall pharmaceutical market shrank in 1994 and 1995, a substantial part of NHS drug-bill savings resulted from cost-shifting from the public sector to patients, mainly because physicians have not aligned their prescribing behaviour to the new positive list. The new Italian approach to containing pharmaceutical costs has been certainly effective, at least in the short run. However, new relevant issues are emerging regarding the fall of NHS pharmaceutical coverage, the centralised nature of the Italian pharmaceutical policy and the gap between scientific based policies and actual prescribing behaviours.