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The present paper illustrates a reform proposal on early access for medicines. In this proposal early access stands for patient’s access, outside of clinical trials and covered by the Italian National Health Service (Servizio Sanitario Nazionale, SSN), to a new medicine or an indication of an already approved medicine before the approval o in between the approval and the decision on price and reimbursement status. The proposal emerged from a multi-stakeholder working group within the Sixth Edition of the “Seminari di Mogliano Veneto”. The reform proposal is aimed at converting the current early access programs (mainly, the programs introduced with Laws 648/96 and 326/03) into a single program inspired by the one introduced in France in 2021. The proposal provides indications on eligibility criteria for drugs and patients, application procedure, evaluation process, economic safeguard clauses, data collection, issues derived from the circumstance that the drug/indication included into the early access program is not reimbursed afterwards and the actions to implement this program.

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.

Equations for the best integrability exponent, for monotonic functions in one-dimensional Gehring and Muckenhoupt classes, are unified in more general Reverse Holder Inequality classes. Furthermore, the result is extended by removing the monotonicity assumptions.

Competing Interests: Conflict of interest: The authors declare no potential conflict of interest with respect to the research, authorship and/or publication of this article. The authors declare the following competing interests.

The debate around unmet clinical need (UCN) is still very much alive. How do we define UCN? How does it influence the definition of clinically relevant outcomes in a therapeutic area? Who defines UCN? What are the consequences of recognizing different grading of UCN? In this paper we will address these questions and finally formulate proposals for the Italian context. The paper is based on a discussion within a panel of experts. This topic is even more stimulating as this work takes place in a historical period which, on the one hand, sees the start of a new course of negotiation rules recently published by AIFA and, on the other hand, poses unprecedented challenges that emerged during the pandemic crisis. The working group formulated suggestions and proposals to further enhance the role of the UCN in decision-making processes, also in the light of the new negotiation procedure, and to help refine the tools for grading the UCN and the value of medicines in the interests of patients and society as a whole.

The transition from the traditional to the mutational model in oncology represents a crucial change, not only from the nosological point of view, since - unlike the traditional one - the new approach is no longer strictly linked to anatomical localization and histology, but also from the point of view of adaptation of the health and regulatory systems to the new paradigm. Agnostic oncology therefore represents a challenge for the evolution of the entire path of the drug, not only for its clinical development, but also concerning the diagnostic-therapeutic and regulatory approach to be followed for the authorization and evaluation of these drugs.