Introduction: Despite the growing attention on Patient-Reported Outcome (PRO) and Patient-Reported Experience (PRE), their use for drugs approval and, especially, price and reimbursement is still limited. The present paper illustrates the discussion and recommendations of three interdisciplinary and multi-stakeholder Working Groups (WGs) on this topic.Method: The discussion, oriented by a report from the coordinators, focused on the role of PRO in the comparative evaluation of efficacy and risk, of PRE as an expression of preference for the drugs value components and of PRO and PRE in the regulatory process and national and regional access.Results: The results are reported as reflections and recommendations on the three topics.Conclusions: There is widespread consensus on the need to enhance the use of PRO and PRE, provided that (i) methodological and operational issues are addressed, (ii) PRO and PRE role in the regulatory and access process is clarified, (iii) representatives of patients and institutions are involved in a more structured way (iv) the actual role played in decision-making is clarified.
Introduction: Despite the growing attention on Patient-Reported Outcome (PRO) and Patient-Reported Experience (PRE), their use for drugs approval and, especially, price and reimbursement is still limited. The present paper illustrates the discussion and recommendations of three interdisciplinary and multi-stakeholder Working Groups (WGs) on this topic.Method: The discussion, oriented by a report from the coordinators, focused on the role of PRO in the comparative evaluation of efficacy and risk, of PRE as an expression of preference for the drugs value components and of PRO and PRE in the regulatory process and national and regional access.Results: The results are reported as reflections and recommendations on the three topics.Conclusions: There is widespread consensus on the need to enhance the use of PRO and PRE, provided that (i) methodological and operational issues are addressed, (ii) PRO and PRE role in the regulatory and access process is clarified, (iii) representatives of patients and institutions are involved in a more structured way (iv) the actual role played in decision-making is clarified.
Introduction: Dry eye disease (DED) is a multifactorial, immune-mediated inflammatory condition affecting the ocular surface and lacrimal glands. Multiple ciclosporin-based therapies with different formulations are currently available in Europe, although clear guidance on their optimal use remains limited. The aim of this study was to determine the main unmet needs and the current management landscape of ciclosporin-based therapies, from the perspective of clinical experts using multi-criteria decision analysis (MCDA). Methods: A systematic literature review was conducted to gather evidence for each of the MCDA framework criteria. An international expert panel of seven clinical specialists in DED from four European countries independently weighted the criteria and scored the non-comparative criteria in the evidence matrix. Results were presented and discussed in a group online session. Results: DED is a highly prevalent, chronic inflammatory disease associated with significant visual and functional burden. Its management is not uniform and often involves a stepwise approach. Recent ciclosporin-based treatment alternatives address some unmet needs; however, others—such as the need for greater standardization of diagnostic tools and improved objective markers of inflammation—remain to be addressed. Highest importance was assigned to Efficacy/effectiveness (4.7 ± 0.5), Quality of evidence (4.7 ± 0.5), and Safety/tolerability (4.6 ± 0.5) criteria, highlighting the relevance of treatment-related outcomes in clinical decision-making. Conclusions: The expert opinion, based on MCDA, enabled the identification of key value drivers and unmet needs in the current management of DED based on ciclosporin-based therapies. These early insights may contribute to promoting future work to support informed, evidence-based decision-making.
Introduction: Severe asthma imposes a substantial clinical, social, and economic burden, particularly within healthcare systems facing increasing pressure to ensure access to innovations such as biologics. Traditional economic evaluations, focused on single strategies, are often insufficient to capture the broader impact of integrated policy interventions. This study assesses the clinical, economic, social, and fiscal impact of three policy strategies for severe asthma management in Italy: (i) improved treatment adherence, (ii) expanded access to biologics, and (iii) earlier disease identification through structured healthcare approaches, using a multidimensional predictive modeling framework.Methods: A combined methodological approach was adopted, integrating a Budget Impact Analysis, a cost-of-illness model, and a Markov model over a five-year horizon. The model was developed from the perspectives of the National Health Service, society, and the fiscal system. Clinical and economic inputs were derived from national tariffs, AIFA transparency lists, and the scientific literature.Results: The baseline scenario shows increasing costs over five years, mainly driven by exacerbation management and productivity losses. Policy 1 and Policy 3 generate cost reductions compared with the baseline scenario, whereas Policy 2 increases expenditures due to higher biologic uptake. The integrated scenario yields an overall benefit, with a cumulative reduction of € 3.5 billion and improvements across all perspectives.Conclusions: Integrated care pathways combining early diagnosis, improved adherence, multidisciplinary management, and appropriate biologic use represent the most effective strategy to improve outcomes while maintaining sustainability. These findings highlight the need for systemic policies supporting equitable access to innovation.
Introduction: Intermittent catheterization is the standard of care for neurogenic bladder dysfunction; however,urinary tract infections (UTIs) remain a major clinical and economic complication in patients with multiple sclerosis(MS) or spinal cord injury (SCI). Micro-hole Zone Technology (MhZT), implemented in the Luja™ catheter, isdesigned to support bladder emptying.Methods: A static 5-year budget impact model was developed from the perspective of the Italian National HealthService, assuming a stable eligible population and comparing a current scenario without Luja™ with progressiveadoption from 5% to 25%. Direct medical costs of catheters and UTI management were included; no discountingwas applied. Deterministic scenario analyses explored uncertainty in clinical effect, UTI costs and uptake.Results: The eligible population included 64,600 users with SCI and 40,600 with MS. In patients with SCI, Luja™adoption reduced cumulative 5-year expenditure from € 1,652.9 million to € 1,643.3 million, preventing 28,861 UTIs.In patients with MS, expenditure decreased from € 1,039.2 million to € 1,033.1 million, preventing 18,139 UTIs. Overall, the model estimated approximately 47,000 avoided UTIs and € 15.8 million in related savings.Conclusions: Under the model assumptions, gradual adoption of MhZT may be economically sustainable andpotentially cost-saving for the Italian National Health Service. Results are mainly driven by an indirectly estimatedreduction in UTIs and should be confirmed using comparative real-world evidence.
