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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: AMM, FC, GCo, AG, ML, MMed , MMel, SP, MP, FP, MZ declare no conflict of interest. WM reports holding a fiduciary role in FIMMG, EPCCS and Consulta. EA is an employee of Amgen and reports stock ownership in the company. MB is an employee of Sanofi. GB is an employee of Dephaforum. RC reported receiving consulting fees from MSD Italia, Eli Lilly Italy, Menarini Diagnostics, Roche Diabetes Care Italy, honoraria from Novo Nordisk, Sanofi, Abbott, Mundipharma Farmaceuticals, participation to an advisory boards supported by Boehringer Ingelheim. GCa reported receiving consulting fees from Novartis, Alexion, AstraZeneca, Abbvie, Astellas, Roche, Takeda, Jannsen and Biomarin. ACa reported receiving consulting fees from MSD Italia, Eli Lilly Italy, Menarini Diagnostics, Roche Diabetes Care Italy, honoraria from Novo Nordisk, Sanofi, Abbott, Mundipharma Farmaceuticals, participation to advisory boards supported by Boehringer Ingelheim. ACo reported receiving research grants by AstraZeneca, honoraria from Novo Nordisk, Eli Lilly, Merck Sharp & Dhome, participation on advisory boards supported by Novo Nordisk and Eli Lilly. GD reports stock ownership in Bristol Meyers Squibb. MDG is an employee of Sanofi and reports stock ownership in the company. EP is an employee or Gilead Sciences and reports stock ownership in the company. DS is an employee of Abbvie. VT reported receiving consulting fees from Biogen Italia and grants from FEEM Fondazione Eni Enrico Mattei. EC is an employee of Chiesi Italia.

Competing Interests: Conflict of interest: FP and FV are employees of AstraZeneca Spa. EB is employee of The European House – Ambrosetti. After the completion of this work FV is no longer affiliated with AstraZeneca. FV is now Associate Director Public Affairs at Incyte (Nasdaq: INCY).

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.

Background: Early access of medicines occurs with an uncertainty in the evidence even higher than the one experienced when price and reimbursement status is negotiated. Our aim is discussing the role of managed entry agreements (MEA) within early access programs (EAP) in Italy., Methods: The discussion relied on a Focus Group, participated by twelve experts, including clinicians and representatives of regulatory authorities, regional and local pharmaceutical departments, pharmaceutical companies, and an association advocating for active citizenship., Results: The Focus Group emphasised that the topic under discussion should be embedded into a more general reform of EAP in Italy. The 648 List mostly includes mature products and indications that are rarely launched into the market afterwards. The 5% Fund is affected by an important administrative burden uncertainty of the timing of reimbursement., Conclusions: Starting from the discussion on MEA and EAP, the Focus Group recommended a new legislation better regulating EAP, that early access concerns specific classes of medicines selected on the grounds of the need to guarantee a rapid access and to collect real world data, that early access can be accompanied by outcome-based and population-based MEA, and that MEA are embedded into the subsequent price and reimbursement negotiation.

Competing Interests: Conflict of interest: FP and FV are employees of AstraZeneca Spa. The views expressed in this article are the authors’ own and do not necessarily reflect the company’s.

In the last decade regulators, payers and health care providers tried to react to three major problems in drug development and drug use in clinical practice: the pharmaceutical R&D productivity crisis, the immaturity of benefit-risk profile for several newly approved drugs and the overall impact on economic sustainability of reimbursing new high cost drugs in their systems. The potentiality of create a continuum between the evidence requirements relevant for registration, for reimbursement and for post authorization research is clear. All different parties involved, like regulators, HTA agencies, scientific communities and manufacturers, are working to improve the knowledge profile of new drugs in order to anticipate the patient access to innovation, limiting or preventing the clinical and economical risks deriving from an incomplete safety and effectiveness profile. The Italian example of "New Drugs AIFA Registries", with or without the application of risk sharing schemes (cost sharing, pay for performance, etc.), introduced a new process and increased the sensitivity on this topic. However this might probably represents only a partial answer to the problem of how to set up the governance of coverage with evidence, drug utilization monitoring, comparative effectiveness research, outcome research programs and may be how to link them to access, pricing and reimbursement. The step change in post authorization research could be to "integrate" different sources and stakeholders in a wider and continuous approach, in a well designed and inclusive "second generation" HTA approach, where all resources (competencies, data, funding) will concur to increase the evidence profile and reduce the risks, and where any "evidence generation approach" is really compliant with the standard and rules of best research practices.

The present study aims at assessing the role of epidemiology on health decision-making processes in the public administrations of the Veneto region, north-eastern Italy. In 2003 a questionnaire was mailed to chief-executives of local public health organizations (21 Local Health Units, 2 Public Hospitals), and 18 responded. All public health organizations were practicing Epidemiology, but only 4 had dedicated services. Sixty-two people were working as epidemiologists in public health, but only half played the role for most of their Full Time Employment. Personal educational qualifications were mainly degree in Medicine (62%) and Statistical Sciences (23%). Among physicians, 78% was specialized in Hygiene and Preventive Medicine. The head office of most public health organizations (15 out of 18) appraised epidemiology as fundamental with regard to the health decision-making process, however two thirds of them reported that the epidemiological informative needs were only little or not at all satisfied. To improve the situation, most Public Health Organizations intended to perform educational activities and to use external advisors. In conclusion, Public Health Organizations of the Veneto region appreciated the importance of epidemiology in the health decision-making process. However, few resources were devoted to this field so that informative needs were only partly satisfied.