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The distribution of drugs reimbursed by the NHS in Italy can be summarized in three forms: direct (DD), on behalf of (DPC) and affiliated. The following document presents the results of the discussion of a multidisciplinary experts’ panel, from different professional realities in the healthcare system, on alternative methods of drugs’ distribution. It was highlighted how regional autonomy has led to extremely innovative experiences but also to a lack of homogeneity regarding the access to pharmaceutical assistance across Italy. The main recommendations developed by the experts can be summarized as follows: To ensure decisions regarding prescription and classification, with respect to the healthcare delivery settings, with the consequent distribution model and purchasing processes, is AIFA’s responsibility. To evaluate the reclassification of drugs from class H to class A-PHT for drugs intended to be taken at patient’s home, in cases where close monitoring by specialistic structures is not necessary. To limit the inclusion of a drug in PHT to conditions of differential diagnostic complexity, need for recurrent patient’s referral to the healthcare facility, presence of AIFA monitoring register, settings of drugs’ administration (home hospitalization and home care). In the field of PHT drugs, AIFA should, with the support of a multidisciplinary technical table: Update the list periodically. Identify the drugs on the list for which DD is preferential, leaving the others in DPC. Evaluate the conditions to transfer some drugs to distribution under an agreement.

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.

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.

This document illustrates the results of a discussion of two multi-disciplinary expert panels on pricing and reimbursement of medicines. Experts work in different organizations. The discussion focused on comparator(s), endpoint(s), negotiation of prices of new medicines and/or indications to include in the List 648, as well as the role of cost-effectiveness in the price and reimbursement negotiation. The debate took place during the fourth edition of the Seminari di Mogliano, organized on the 30th of September/1st of October, 2021. The two panels agreed on a general need to enhance interaction among the different stakeholders, in the early assessment and negotiation phases, and to increase the transparency/reproducibility of the decisions taken. The experts have also emphasized the need (i) to improve clarity in the evaluation of additional therapeutic value and the place in therapy with respect to comparators and how comparators are identified; (ii) to create work groups to identify the most appropriate endpoint(s), for each therapeutic area and level of unmet needs; (iii) to provide for a systematic use of cost-effectiveness when an added therapeutic value is delivered by a new medicine. With regard to the 648 List, the experts advocated for an overall reorganization of the current rules governing the special uses of drugs.

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.

Use of real world data is spreading at increasing pace in response to the growing need of ensuring effectiveness and appropriateness in the delivery of health and medical care. Data from clinical trials are essential to define efficacy and safety of medical therapies in experimental conditions, while real world data assume greater relevance when one considers the differences between study populations of clinical trials and people who take the same drug in real life conditions, and the changes that happen over time with the acquisition of new evidence on drugs and treatments. Rheumatoid arthritis is a chronic disease with significant social (direct and indirect) costs that today can be medically managed ensuring both effectiveness and appropriateness. Real world data can contribute significantly to the efficient management of this disease as demonstrated by outstanding examples from Italian registries and databases.

Defining therapeutic innovation is a key point to determine the price of newly marketed drugs; however, a pre-requisite for an appropriate management of innovation is that some general rules have been identified to recognize the therapeutic value of any pharmacological agent. These issues are a matter of debate for Regulatory Agencies and for the scientific community as well. Cost-effectiveness principles still represent the main approach to quantify the economic value of a drug based on its clinical effectiveness, and QALYs still represent the main tool to compare outcomes across different therapeutic areas. The main problem in this area is represented by the difficulty in translating general principles into practical decisions. Factors that still hamper the application of the value-based approach include organizational constraints (e.g. drug fixed budgets), the lack of familiarity with cost-effectiveness by most decision-makers, and the history of local decisions.