Regional Formularies in Italy: current state and future perspectives

Regional Formularies in Italy: current state and future perspectives Regional Formularies (RF) are considered part of pharmaceutical policies implemented by regions to govern access of medicines to regional market. However, they have been actually challenged, because of their presumed impact on differences of patient's access across the regions. The paper aimed at investigating the current status of RF and Regional Therapeutic Committees (CTR) and at suggesting/recommending possible reforms. The current status was investigated through a questionnaire administered to the regional pharmaceutical departments. Recommendations were retrieved from a multi -stakeholder work group carried out on 30-31 March 2023, embedded into a Forum focused on the regional pharmaceutical policies. Nineteen out of twenty-one regions responded to the survey: 12 use RF, mainly managed by the CTR; the RF frequency of update and the time needed for drugs listing greatly vary across regions; pharmacists, specialists and general practitioners are always represented in CTR, whereas other healthcare professionals and experts are more rarely involved; in 3 regions the CTR does not publish any RF update; the CTR mainly rely, to take decisions, on the dimension of the target population, the cost of therapy compared to alternative treatments and the impact on pharmaceutical expenditure. The working group recommended to overcome the RFs, if they are merely considered a list of available drugs at regional level, focusing CTR activities to ensure market access and to govern the prescribing behaviour, and strengthening/anticipating the flow of information from the Italian Medicines Agency (AIFA) to the regions, to enable a more efficient approach to local access to drugs

Early Access in Oncology: Why Is It Needed?

Timely access to cancer therapies with significant added value is an important expectation for patients and a primary responsibility for every public health service. Over time, collaboration between the pharmaceutical industry and regulatory agencies has made it possible to agree to implement tools in order to accelerate the development and approval of potentially innovative drugs. In Italy, too, several early access tools have been introduced. In June 2018 a panel of experts agreed on the need to simplify and streamline early access assessment criteria and processes. The panel developed a proposal to categorize cancer drugs eligible for early access. In the curative setting, the evaluation of the medical need should take into account both the relapse rate, attributed on the basis of the disease free survival (DFS), and the strength of the recommendations of the Italian Association of Medical Oncology (AIOM) for any therapeutic alternatives already available. The panel then found it appropriate to use the European Society for Medical Oncology (ESMO) criteria for the evaluation of the clinical benefit. The sum of the scores assigned to the three parameters should allow the clinical value of the drug to be defined and, consequently, the priorities for early access to be established. This multiparameter approach can also be adapted to the non-curative setting. The early access process should be reserved for first-in-class drugs and should provide for the recognition of a conditional reimbursement within 60 days, financed by a special fund. The proposal developed by the panel has the objective of starting a proactive discussion with the Italian health authority

Therapeutic Value and Innovation of a Drug and Criteria for Evaluation: A Multidisciplinary Experience in the Veneto Region

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Good practices for the development of budget impact models at regional level

Introduction: The present work aims to discuss the current scenario of procedures and regulations regarding budget impact analysis/models (BIA/BIM) at regional level in Italy and to provide a standardized approach and detailed recommendations for developing these analyses. Method: A systematic review of the literature was conducted in order to collect existing guidelines or specific regional procedures for budget impact analysis in Italy. All the records were analysed in qualitative terms according to a pre-specified analytical framework, based on the ISPOR BIA guidelines. At the end of the analysis, a consensus questionnaire was developed to establish agreed approaches and to provide possible solutions to any critical issues. A list of 39 statements was developed. The survey was distributed to 69 experts who rated their level of agreement with each statement on a 5-point Likert scale. Consensus was predefined as more than 66% of the panel agreeing/disagreeing with any given statement. Results: Sisty-nine experts answered the questionnaire; a total of 30/39 statements achieved consensus. There was agreement on most of the statements. Time horizon to consider and costs were the issues on which no agreement was found. The results allowed the working group to define a list of good practices. Conclusion: While the structure and development of BIM are now well-known and well-applied at national level, there remains a great diversity of management of BIM tools at regional level. Consensus was reached among participating experts, as to the main characteristics, determinants and features of regional BIA/BIM in the perspective of the Italian payer

Clinical efficacy of different monoclonal antibody regimens among non-hospitalised patients with mild to moderate COVID-19 at high risk for disease progression: a prospective cohort study

This study aimed to compare the clinical progression of COVID-19 in high-risk outpatients treated with the monoclonal antibodies (mAb) bamlanivimab, bamlanivimab-etesevimab and casirivimab-imdevimab. This is an observational, multi-centre, prospective study conducted from 18 March to 15 July 2021 in eight Italian tertiary-care hospitals including mild-to-moderate COVID-19 outpatients receiving bamlanivimab (700 mg), bamlanivimab-etesevimab (700-1400 mg) or casirivimab-imdevimab (1200-1200 mg). All patients were at high risk of COVID-19 progression according to Italian Medicines Agency definitions. In a patient subgroup, SARS-CoV-2 variant and anti-SARS-CoV-2 serology were analysed at baseline. Factors associated with 28-day all-cause hospitalisation were identified using multivariable multilevel logistic regression (MMLR) and summarised with adjusted odds ratio (aOR) and 95% confidence interval (CI). A total of 635 outpatients received mAb: 161 (25.4%) bamlanivimab, 396 (62.4%) bamlanivimab-etesevimab and 78 (12.2%) casirivimab-imdevimab. Ninety-five (15%) patients received full or partial SARS-CoV-2 vaccination. The B.1.1.7 (Alpha) variant was detected in 99% of patients. Baseline serology showed no significant differences among the three mAb regimen groups. Twenty-eight-day all-cause hospitalisation was 11.3%, with a significantly higher proportion (p 0.001) in the bamlanivimab group (18.6%), compared to the bamlanivimab-etesevimab (10.1%) and casirivimab-imdevimab (2.6%) groups. On MMLR, aORs for 28-day all-cause hospitalisation were significantly lower in patients receiving bamlanivimab-etesevimab (aOR 0.51, 95% CI 0.30-0.88 p 0.015) and casirivimab-imdevimab (aOR 0.14, 95% CI 0.03-0.61, p 0.009) compared to those receiving bamlanivimab. No patients with a history of vaccination were hospitalised. The study suggests differences in clinical outcomes among the first available mAb regimens for treating high-risk COVID-19 outpatients. Randomised trials are needed to compare efficacy of mAb combination regimens in high-risk populations and according to circulating variants

SARS-CoV-2 and COVID-19 in diabetes mellitus. Population-based study on ascertained infections, hospital admissions and mortality in an Italian region with 3c5 million inhabitants and 3c250,000 diabetic people

Background and aims: Diabetes conveys an increased risk of infectious diseases and related mortality. We investigated risk of ascertained SARS-CoV-2 infection in diabetes subjects from the Veneto Region, Northeastern Italy, as well as the risk of being admitted to hospital or intensive care unit (ICU), or mortality for COVID-19. Methods and results: Diabetic subjects were identified by linkage of multiple health archives. The rest of the population served as reference. Information on ascertained infection by SARS-CoV-2, admission to hospital, admission to ICU and mortality in the period from February 21 to July 31, 2020 were retrieved from the regional registry of COVID-19. Subjects with ascertained diabetes were 269,830 (55.2% men; median age 72 years). Reference subjects were 4,681,239 (men 48.6%, median age 46 years). Ratios of age- and gender-standardized rates (RR) [95% CI] for ascertained infection, admission to hospital, admission to ICU and disease-related death in diabetic subjects were 1.31 [1.19-1.45], 2.11 [1.83-2.44], 2.45 [1.96-3.07], 1.87 [1.68-2.09], all p < 0.001. The highest RR of ascertained infection was observed in diabetic men aged 20-39 years: 1.90 [1.04-3.21]. The highest RR of ICU admission and death were observed in diabetic men aged 40-59 years: 3.47 [2.00-5.70] and 5.54 [2.23-12.1], respectively. Conclusions: These data, observed in a large population of 3c5 million people of whom 3c250,000 with diabetes, show that diabetes not only conveys a poorer outcome in COVID-19 but also confers an increased risk of ascertained infection from SARS-CoV-2. Men of young or mature age have the highest relative risks