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Within the European evolutionary framework concerning citizens’ health, the focus shifts to the new HTA regulation, set to alter processes and influence decision-making at the individual country level regarding reimbursement and pricing. The ambitious goal of achieving faster and more uniform access will significantly reshape the evaluative model stabilized over the years, characterized by the creation of different rules and processes among member states and diverse output timelines across the countries. The imminent adoption of a more collaborative process necessitates member countries and companies to address several steps to ensure a smooth transition without penalizing the unique aspects of individual countries or impeding real access. Italy, actively participating in European preparatory activities with AIFA, faces challenges in adapting its formal process due to AIFA’s ongoing reform since November 2022, which is not yet materialized. National-level companies heavily depend on leadership and involvement rules from their HTA agencies, with the risk that agencies with more established engagement models with external stakeholders may present themselves on January 12, 2025, the start date of the new process for oncological and ATMP drugs, better prepared and may excel in designing scoping meetings and in formulating PICO. Therefore, the activation of a country culture of HTA and a robust and extended “readiness” phase involving all stakeholders are desirable, along with the establishment of collaboration networks with universities and scientific societies to make valuable knowledge and qualified resources more readily available, crucial element for the delicate transition from the old to the new evaluative system.

This paper illustrates the recommendations of a Working Group (WG) on the assessment of drugs innovativeness and the negotiation of price and reimbursement. The WG included researchers, institutions, clinicians, patient representatives and pharmaceutical companies. The first part of the contribution summarizes the literature on drug pricing models, which was considered in the WG, and, in particular, the pricing criteria, the evaluation and negotiation processes, the management of the uncertainty of the evidence, the use of cross-reference pricing and price negotiation for new indications of existing drugs. The second part illustrates the results of the WG with a focus on innovativeness assessment, value framework and price negotiation. The main recommendations of the WG are: to define more specific criteria for the identification of comparators and endpoints for macro therapeutic areas/settings; to produce guidelines on the use of indirect comparisons and studies supporting this evidence; to consider the drug value as the main driver of price and reimbursement negotiation; to maintain flexibility in the negotiation process, but, at the same time, to give greater structure and predictability in the assessment of value for money, with a more qualified role of cost-effectiveness and a range of threshold values for the incremental cost-effectiveness ratio; to selectively reintroduce Managed Entry Agreements and the Indication-based pricing model; to implement an early dialogue between the Italian Medicine Agency and the pharmaceutical companies in order to optimize the negotiation process, and a structured involvement of scientific societies and patient representatives.

The definition of criteria and processes for the submission of price and reimbursement requests (P&R) of a drug in Italy cannot be separated from the definition of an overall “Pharmaceutical Policy” that includes, in an organic vision: (i) the governance related to the research, marketing and monitoring of drugs in the Italian market; (ii) the availability of drugs on the national territory as an element included in the essential levels of care (LEA) and the related conditions in terms of timing and equity of access between different regions, as well as towards other European countries; (iii) the assessment criteria used. The decree published in the Italian Official Journal in the summer of 2020, which defines the new criteria for the regulation of P&R of medicines in Italy, focuses on the final part of the process, i.e. the price negotiation. It would be necessary to frame this last step within a broader and more organic structure of drug policies aimed at: 1. optimising healthcare funding by encouraging competition between healthcare technologies; 2. reducing assessment time by simplifying processes; 3. improving early access to drugs for unmet need; 4. increasing the quality of P&R dossiers by improving interaction with the companies; 5. encouraging innovative agreements and complementary elements to the price; 6. encouraging the reproducibility of assessment methodologies in a value-based pricing system; 7. considering cost-benefit analyses as tools for the definition of price and conditions of reimbursability; 8. creating a place for discussion on drug policies.

The XXII National Conference on Pharmaceuticals, held in Catania from 29 February to 1 March 2024, involved the participation of representatives from more than forty pharmaceutical industries, national authorities, academia, clinicians and clinical pharmacists. The 22nd edition represented a key forum for the analysis of value assessment of medicines, focusing on the impact of new European and National regulations, including the Italian Medicines Agency (AIFA) reform and the HTA regulation. This point of view summarizes insights from focus groups discussions that took place during the Conference, highlighting the pillar role of the new HTA regulation for homogenizing the evaluation across Europe, and the importance to strengthen the collaboration among the parties involved from an early phase, and implementing adaptive and flexible assessment, especially for orphan medicines. The re-evaluation of the innovativeness criteria and framework of the Pricing and Reimbursement (P&R) dossier are points raised among the different focus groups, especially for aligning these tools to the upcoming HTA regulation. The proposals emerged could be useful for AIFA, which is facing a reorganization aimed to optimizing the reimbursement process in Italy.

Pharmaceutical industry investments in Italy must be supported with a reform process that promotes simplifications to generate an attractive ecosystem that can enhance innovation. Key actions include facilitating the start-up of clinical trials, promoting public-private partnerships to support technology transfer, integrating data infrastructures to overcome the logic of silos, expanding programs for prevention and early identification of diseases, simultaneous reimbursement for therapy and diagnostic testing, and launching a structural program for early access to therapies. The total contribution of the sector in the last 10 years to the Italian economy was € 315 bn. Looking at employment (67 thousand people employed in the sector in 2020), it is possible to estimate about 280 thousand jobs activated in Italy just in 2020. An Altems study quantified a leverage effect of 2.77 in terms of benefits to the Italian NHS from direct investment in clinical research, which means that for every € 1 invested by companies in clinical trials, € 1.77 of additional savings were generated for the NHS. Applying the multiplier on R&D investments of the entire pharmaceutical sector in 2020 (€ 1.6 bn), we could estimate approximately € 4.4 bn in benefits for the NHS. In addition to these benefits there are the noneconomic benefits of clinical research, including early access for patients to innovative therapies, resulting in improved clinical outcomes and quality of life for patients and caregivers in general.

The XXIII National Conference on Pharmaceuticals, held in Catania on February 27-28, 2025, represented a key event for multidisciplinary and cross-sectoral dialogue on the pharmaceutical governance in Italy, with a particular focus on stakeholders’ cooperation. Structured around a plenary session and four thematic focus groups, the event aimed to critically and proactively analyse the main challenges related to the assessment and access processes in Italy. The focus groups discussions addressed four priority areas: equitable and timely access to medicines; transparency and collaborative governance; assessment of drug value; the implementation of the European Health Technology Assessment (HTA) Regulation.Each focus group followed a structured methodology based on the analysis of the current landscape, identification of systemic gaps, and formulation of concrete, actionable proposals. The Conference gathered over 70 experts from 30 pharmaceutical companies along with academic institutions, highlighting the growing commitment to redefining pharmaceutical policy in a more participatory, integrated, and sustainable way. The outcomes revealed a strong thematic and methodological convergence across the groups, emphasizing the need to overcome fragmented and sectoral approaches aimed to a cohesive, transparent and equitable governance model. The proposals that emerged outline a strategic roadmap for modernizing the Italian regulatory system, by promoting faster and fairer access to medicines, strengthening transparency and stakeholder engagement, updating value assessment frameworks, and effectively integrating the new European HTA Regulation.

Within the European evolutionary framework concerning citizens’ health, the focus shifts to the new HTA regulation, set to alter processes and influence decision-making at the individual country level regarding reimbursement and pricing. The ambitious goal of achieving faster and more uniform access will significantly reshape the evaluative model stabilized over the years, characterized by the creation of different rules and processes among member states and diverse output timelines across the countries. The imminent adoption of a more collaborative process necessitates member countries and companies to address several steps to ensure a smooth transition without penalizing the unique aspects of individual countries or impeding real access. Italy, actively participating in European preparatory activities with AIFA, faces challenges in adapting its formal process due to AIFA’s ongoing reform since November 2022, which is not yet materialized. National-level companies heavily depend on leadership and involvement rules from their HTA agencies, with the risk that agencies with more established engagement models with external stakeholders may present themselves on January 12, 2025, the start date of the new process for oncological and ATMP drugs, better prepared and may excel in designing scoping meetings and in formulating PICO. Therefore, the activation of a country culture of HTA and a robust and extended “readiness” phase involving all stakeholders are desirable, along with the establishment of collaboration networks with universities and scientific societies to make valuable knowledge and qualified resources more readily available, crucial element for the delicate transition from the old to the new evaluative system.

This document illustrates the results of the work of two interdisciplinary and multistakeholder panels (researchers, public institutions, and industry representatives) on drug territorialization and digitalization, organized as part of a residential seminar held on 30 September and 1st October 2021. Arising from some considerations about the demand for health and the provisions of the National Recovery and Resilience Plan (PNRR), the discussion touched various aspects of managing the transition from current to future management models. The importance of identifying criteria for prioritizing interventions in the area emerged: different methods of drug delivery, scientific information and measurement, re-evaluation of pathologies that can be managed in this area. Finally, the role of digitization within this change was explored. The opinions provided by the experts move towards making the most of the opportunities arising from PNRR, in terms of investments in healthcare and data application, with a view to improve health system efficiency, patient care and related outcomes.

The definition of criteria and processes for the submission of price and reimbursement requests (P&R) of a drug in Italy cannot be separated from the definition of an overall “Pharmaceutical Policy” that includes, in an organic vision: (i) the governance related to the research, marketing and monitoring of drugs in the Italian market; (ii) the availability of drugs on the national territory as an element included in the essential levels of care (LEA) and the related conditions in terms of timing and equity of access between different regions, as well as towards other European countries; (iii) the assessment criteria used. The decree published in the Italian Official Journal in the summer of 2020, which defines the new criteria for the regulation of P&R of medicines in Italy, focuses on the final part of the process, i.e. the price negotiation. It would be necessary to frame this last step within a broader and more organic structure of drug policies aimed at: 1. optimising healthcare funding by encouraging competition between healthcare technologies; 2. reducing assessment time by simplifying processes; 3. improving early access to drugs for unmet need; 4. increasing the quality of P&R dossiers by improving interaction with the companies; 5. encouraging innovative agreements and complementary elements to the price; 6. encouraging the reproducibility of assessment methodologies in a value-based pricing system; 7. considering cost-benefit analyses as tools for the definition of price and conditions of reimbursability; 8. creating a place for discussion on drug policies.