The Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries’ type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare

SIR-Spheres® Y-90 resin microspheres in chemotherapy refractory or intolerant patients with metastatic colorectal cancer:

Selective internal radiation therapy is a form of intra-arterial brachytherapy used to treat primary liver cancer and liver metastases. This article aims to provide an overview of the clinical, economic, organizational legal, social and ethical impact of selective internal radiation therapy using SIR-Spheres Y-90 resin microspheres in the treatment of patients with unresectable, liver-dominant metastatic colorectal cancer who are refractory to or intolerant of chemotherapy. A systematic literature review was performed by querying PubMed, Scopus, EBSCO, CRD and GIN. Two reviewers blindly screened the records retrieved against predefined inclusion/exclusion criteria. The selected studies where summarized following a simplified version of the EuNetHTA Core Model® 2.1. The studies included evaluated selective internal radiation therapy in first-line or further-line treatment and showed a good safety and tolerability profile and significant improvement in efficacy expressed as time to liver progression, progression-free survival and overall survival. Selective internal radiation therapy should be provided in specialized centres and administered by a multidisciplinary team. A hub-and-spoke network could be a viable option to guarantee access to this technology across jurisdictions. The lack of a specific diagnosis-related group tariff accounting for the cost of the device could be seen as the major obstacle to a fair diffusion of this technology. The economic evaluations currently available show the cost-effectiveness of this technology in the population under study. Selective internal radiation therapy using SIR-Spheres Y-90 resin microspheres appears to be a clinically effective and cost-effective option in the treatment of metastatic colorectal cancer patients who are chemotherapy refractory or chemotherapy intolerant

VP164 Applying health technology assessment to pharmacy: the Italian-Medicine-Use-Review-Health Technology Assessment

There is a lack of Health-Technology-Assessment (HTA) tools in pharmacy practice and the collection of real-world-evidence (RWE) in community pharmacy to populate longer-term-disease-progression-modelling (1). This project is looking at the development and application of a novel Patient-Reported-Outcome- Measure (PROM) in community pharmacy that can enable: the evaluation of the quality of care delivered from the patient perspective in terms of economic impact, patient health outcomes and ‘utilities’; the collection of RWE and evaluate long-term effect of care; to provide different stakeholders with unique evidence-based information that help formulate health policies in community pharmacy that are safe, effective, patient-focused and cost-effective, balancing access to innovation and cost containment. Evidence from the Italian-Medicine-Use-Review (I-MUR) trial (2) showed that the I-MUR intervention provided by community pharmacists to asthma patients is effective, cost-saving and cost-effective (3). The trial allowed to model a framework (I-MUR-HTA) that would enable to routinely deliver the intervention, but also collect and analyse PROM data on its clinical-effectiveness, quality-of-life and cost-effectiveness. I-MUR-HTA was discussed within three expert-panel discussions including policy-makers, commissioners, academics, healthcare-professionals and patient-representatives in Italy, United Kingdom and Europe. Current plan include testing the use of the tool in the real world environment. Evidence collected from the panel discussions confirmed that I-MUR-HTA evidence-based information is relevant to meet current National-Health-Care-System plans and this is what is needed to support the evaluation of innovative effective and cost-effective health policies and promote their implementation across nations. Current Italian law on pharmacy services provides the appropriate institutional framework to regulate the introduction of I-MUR-HTA across the territory. Its implementation is underway and a real-world pilot is planned to take place in Italy. I-MUR-HTA appears to be an innovative tool to promote active patient involvement into policy-decision-making and pharmacy-service

TURVALLISESTI MAAILMAAN Yhteispäivystyksen hoitohenkilökunnan osaamisen kehittäminen matkasynnytysvalmiuteen

Tämä opinnäytetyö on suunnattu Pohjois-Karjalan sairaanhoito ja sosiaalipalvelujen kuntayhtymän (PKSSK) yhteispäivystyksen hoitohenkilökunnalle. Opinnäytetyön tarkoituksena on järjestää koulutusta yhteispäivystyksen hoitohenkilökunnalle. Koulutuksessa käydään läpi synnytyksen eri vaiheet ja avustaminen matkasynnytyksessä. Opinnäytetyön tavoitteena on, että jokaisella koulutukseen osallistuvalla olisi perusvalmiudet avustaessaan mahdollisessa matkasynnytyksessä. Opinnäytetyö sai alkunsa opinnäytetyön tekijän omasta kokemuksesta ja muun yhteispäivystyksen hoitohenkilökunnan kanssa käydyistä keskusteluista. Keskusteluissa tuli esille, että tiedot ja taidot ovat puutteelliset, jos he joutuisivat avustamaan synnytyksessä. Opinnäytetyö on toteutettu toimintatutkimuksena, ja opinnäytetyön tuotos on valokuvien kanssa tehty PowerPoint-esitys synnytyksen vaiheista. Opinnäytetyötä varten on kerätty anonyymisti tietoa koulutustarpeesta ja siitä, millaista koulutusta haluttiin järjestettäväksi. Kyselyyn vastasi kolmannes (n=32) yhteispäivystyksen hoitajista. Opinnäytetyö on ajankohtainen ja hyödyllinen, koska esimerkiksi synnytyssairaaloiden lakkauttamisen takia välimatkat synnytyssairaaloihin ovat pidentyneet ja matkasynnytykset lisääntyneet. Yhteispäivystyksessä täytyy olla valmius erilaisten yllättävien tilanteiden varalle ja yksi tällainen tilanne on matkasynnytyksessä avustaminen. Opinnäytetyön tuotos on vapaasti käytettävissä koko PKSSK:n alueella, koska se on lisätty PKSSK:n oppimateriaaleihin. Vastaavia opinnäytetöitä on tehty esimerkiksi ambulanssi henkilökunnalle, mutta niissä koulutusmateriaali on suunniteltu ambulanssissa tai kotona tapahtuvaa synnytystä varten.The thesis targets nursing staff of the emergency department at North Karelia Medical care and Social Services (PKSSK). The objective of the thesis is to help provide in out-of-hospital delivery to the staff. The training covers the different phases of the child-birth and assisting in an out-of-hospital delivery.The purpose of the thesis is to ensure that all participants will have the basic skills in assisting in an out-of-hospital delivery. The idea of this thesis rose from the personal experiences of the thesis author and from the discussions with other nursing staff members at the emergency department. It became evident that both skills and knowledge were considered as inadequate to assist in an ouf-ofhospital delivery. The thesis has been carried out as an action research, and the educational material will be performed as a PowerPoint presentation with photographs of the different steps of child birth. The information about what kind of education is needed at the emergency department was collected anonymously. The third (=32) of the nursing staff answered to the inquiry. The thesis is useful and current, because closing several maternity hospitals has increased the distances between hospitals and therefore also the out-of-hospital deliveries have increased. Emergency departments have to be prepared for different kinds of unexpected situations, out-of-hospital deliveries being one of them. The output of this bachelor’s thesis is freely available for use in every department in PKSSK and can be found in educational materials in PKSSK intranet. Similar theses have been made, for example, for paramedics, the difference being in focusing on child births in an ambulance or in one’s home

Health Care Financing Reforms in Italy: Projects of Jefferson\u27s Center for Research in Medical Education and Health Care

No abstract available

Assessing social and economic impact of subcutaneous mAbs in oncology

Background: Rituximab and trastuzumab were the first monoclonal antibodies (mAbs) approved for the treatment of cancer patients. Both antibodies are administered intravenously (EV), but subcutaneous (SC) formulations have recently been developed. SC formulations proved to be as safe and effective as EV and to offer substantial benefits to the patient. Objective: The aim of this study was to provide a multidimensional assessment of the impact of rituximab and trastuzumab SC compared to the EV formulation, providing a particular focus on expected social and economic benefits for the patient. Methods: The best established HTA methods were applied to gather and organize evidence concerning the clinical, economic, organizational, social and ethical impact of SC formulations of rituximab and trastuzumab. Social aspects were investigated applying regression methods to data collected with a previous research, while the potential savings associated with the use of SC formulations were estimated by a simple economic model applying the societal perspective. Results Patients undergoing subcutaneous formulation are significantly more satisfied with their treatment experience than those treated with intravenous formulation. Subcutaneous formulation reduces patient dosing times, with a positive effect on the care provider's autonomy and productivity. Potential savings associated with the use of rituximab SC were estimated in € 4,050 per patient per year on average. For trastuzumab SC the estimated potential savings amounted to € 3,400 per patient per year, on average. Conclusion Rituximab and trastuzumab are promising treatment options significantly improving patients qol and reducing the treatment burden in terms of societal costs

The economic and fiscal impact of incremental use of cell-based quadrivalent influenza vaccine for the prevention of seasonal influenza among healthcare workers in Italy

Background. Seasonal influenza has a significant impact on public health, generating substantial direct healthcare costs, production losses and fiscal effects. Understanding these consequences is crucial to effective decision-making and the development of preventive strategies. This study aimed to evaluate the economic and the fiscal impact of implementing an incremental strategy for seasonal influenza prevention using the cell-based quadrivalent influenza vaccine (QIVc) among healthcare workers (HCWs) in Italy.Methods: To estimate the economic impact of implementing this strategy, we performed a cost analysis that considered direct healthcare costs, productivity losses and fiscal impact. The analysis considered a 3-year time horizon. A deterministic sensitivity analysis was also conducted.Results: Assuming a vaccination coverage rate of 30% among HCWs, the analysis considered a total of 203 018 vaccinated subjects. On analysing the overall differential impact (including direct costs, indirect costs and fiscal impact), implementing QIVc vaccination as a preventative measure against influenza among HCWs in Italy would yield societal resource savings of euro23 638.78 in the first year, euro47 277.56 in the second year, and euro70 916.35 in the third year, resulting in total resource savings of euro141 832.69.Conclusions:The study demonstrated that implementing the incremental use of QIVc as part of a preventive strategy for seasonal influenza among HCWs in Italy could yield positive economic outcomes, especially in terms of indirect costs and fiscal impact. The resources saved could be utilized to fund further public health interventions. Policy-makers should consider these findings when making decisions regarding influenza prevention strategies targeting HCWs

Strategies to achieve HPV-related disease control in Italy: results from an integrative approach

Background: achieving Human Papilloma Virus (HPV) - related diseases control is an important challenge in public health. In Italy HPV vaccination uptake does not rise a sufficient level. The aim of this project is to identify strategies to promote HPV vaccination in Italy.  Methods: an integrated approach consisting of a systematic review and a two-step panel consultation was used to identify strategies to increase vaccination uptake among adolescents, population target of the national vaccination program, and to promote vaccination in additional targets. Overall, ten experts in the fields of Gynecology, Public Health, General Practice and Pediatrics were involved along with Patients representatives. Recommendations were elaborated according to a set of criteria drawn from the Evidence to Decision (EtD) framework.  Results: the systematic review led to the identification of three categories of strategies: reminds, education and multicomponent approaches respectively. A strong recommendation was formulated to use reminds tailored to vaccine recipients or their parents, and a moderate recommendation to use reminds directed to health professionals. A moderate recommendation was developed on the implementation of multicomponent interventions. A strong recommendation was yielded with respect to the promotion of HPV vaccination among women already treated for HPV-related diseases, fertile women not previously vaccinated and 25 year-old women undergoing cervical cancer screening. Lastly, a strong recommendation was formulated for catch-up initiatives targeted to women and men turning 18 years of age.  Conclusion: this project led to the identification of several valuable strategies to improve HPV vaccination and strengthen HPV-related diseases control at national level.&nbsp

A new decision model for economic evaluation of novel therapies for HCV

In 2014, the European Medicines Agency (EMA) has given the license to two new direct-acting antiviral: sofosbuvir and simeprevir. The evidence provided by the studies, reported a high rate of SVR even in patients with decompensated cirrhosis. This and other innovative elements are potentially adept at changing the entire natural course of HCV. However, the dramatic prevalence rates of HCV observed in Italy, and the high prices that are expected to be required by the pharmaceutical industry, raises some critical issues about how to regulate access to such drugs. The objective of this article is to present a new decision model for the evaluation of novel therapies for HCV. This model is intended to provide a tool for the decision-maker that seeks to address the main issues related to the introduction of HCV new treatments. The model that we have structured follows the classic Bayesian approach, using data from reference literature for staging the action of treatments depending on the level of fibrosis (F0, F1, F2, F3, F4). The model is designed to consider patients with all genotypes and allows to make comparisons between innovative and traditional therapies (dual, triple, IFN free, PI combinations, etc.), for both experienced and naïve patients. In addition, the model is used to simulate mixed cohorts of patients, representing a population with HCV with different levels of fibrosis and different genotypes. To show the potential of the model, we created some simple scenarios assuming different levels of SVR and pricing. The results of our model show that, even assuming an SVR rate of 100%, the administration of new treatments for HCV subjects F1 / 2 has an incremental cost-effectiveness ratio not sustainable. In contrast, for the subjects F3 and F4, low incremental SVR rates and an incremental cost of the innovative therapy of € 40,000 would be cost effective. The added value of this model is its versatility and applicability to diverse assessment needs. In addition, the model offers an opportunity for reflection even to the industry, which in the years to come will have to develop strategies for entering the market and offer sustainable prices for decision-makers and at the same time remunerate the investment in research and development consistently with the expectations of the shareholders

A cost-minimization analysis of a preventive testing strategy for relatives of patients with BRCA mutated ovarian cancer

Purpose: This study aims to estimate the cost-minimization strategy of a preventive testing strategy destined to relatives of patients with BRCA mutated cancer versus a no test strategy in Italia. Methods: A BRCA testing pathway was designed by a panel of experts based on the MSTM Excel (2010) tool; the analysis was carried out considering the perspective of the Italian National Health Service. Two alternatives were considered: 1) preventive BRCA testing for relatives of patients affected by ovarian cancer carrying a BRCA1/BRCA2 mutation; 2) no test. Cost and effectiveness data, derived from literature and published sources validated by a Board of experts, were discounted using a discount factor equal to 3%. Probabilistic sensitivity analysis was performed. Results: Considering an average cost of therapy for breast and ovarian cancer major of €90,000.00 per case, the economic impact related to the preventive testing strategy are equal to –€17,814,767.25. The sensitivity analysis confirms these results in the totality of the simulations performed. Conclusions: Preventive genetic testing in relatives of patients affected by ovarian cancer is cost-effective and represents a sustainable cost for the National Healthcare System in Italia, also in the light of its reference values