[Haemophilia B therapy: impact of the reimbursability of a long-acting recombinant factor on pharmaceutical expenditure in Italy]. / Terapia dell'emofilia B: impatto dell'ammissione alla rimborsabilità di un fattore ricombinante long-acting sulla spesa farmaceutica in Italia.

OBJECTIVES: to assess the variability in expenditure compared to 2022 assuming different rates of shifting of therapy days from current active ingredients used for the treatment of haemophilia B to nonacog beta pegolDesign: descriptive cross-sectional study. SETTING AND PARTICIPANTS: consumption in the year 2022 (data source: Medicines Utilisation Monitoring Centre, Italian Medicines Agency) of all medicinal products available in Italy containing coagulation factor IX. MAIN OUTCOMES MEASURES: for each active ingredient, the total number of therapy days and the variability in expenditure compared to 2022 were estimated on the basis of a switch of therapy days, between 5% and 20%, to nonacog beta pegol. RESULTS: on the basis of considered scenarios, the analysis shows that the total annual expenditure for clotting factors used in the treatment of haemophilia B could remain at most unchanged or reduced. Particularly, the extent of the reduction in spending could vary from 0.11% to 2.26%. This trend would be in contrast to the stable increase seen in recent years, particularly in 2022. CONCLUSIONS: this predictive spending assessment may be useful in evaluating the economic impact from new treatment options, such as etranacogene dezaparvovec gene therapy already approved by the European Medicines Agency and the Food and Drug Administration, and to improve pharmaceutical governance.

Postpartum hemorrhage: not only hypertensive disorders in oocyte donation pregnancies.

BACKGROUND: The aim of the study is to compare the obstetric outcome between single pregnancies obtained by medically-assisted procreation using oocyte donors (MAP-E) versus homologous gametes (MAP-O) and single spontaneous conception pregnancies (SC). METHODS: This is a retrospective case-control study on pregnancy outcome of consecutive singleton live birth pregnancies from MAP-E between January 2011 and August 2017 referred to Careggi University Hospital, Florence. The control group includes singleton pregnancies from MAP-O and pregnancies from spontaneous conceptions in the same period. The pregnancy outcomes considered were: postpartum hemorrhage (PPH), cesarean section (CS), gestational diabetes mellitus (GDM), hypertensive disorders including preeclampsia (HDP), preterm birth ≤34 weeks (PTB), and small-for-gestational-age (SGA) fetuses. RESULTS: The study group included 290 MAP-E pregnancies that were compared with 290 MAP-O and 870 singleton spontaneous conception pregnancies. The three groups did not show significant differences in maternal traits except for mean age (43.4±2.9 vs. 37.7±2.4 vs. 33.6±5.5, P<0.001), including a higher percentage of patients over 45 years (41.3% vs. 5% vs. 0.8%, P<0.001) and higher incidence of obesity (7.2% vs. 1.7%, P=0.02) in MAP-E than in MAP-O. The risk of HDP is increased in singleton pregnancies by oocyte donation with a significantly increased risk if compared to MAP-O (12% vs. 1%, P<0.001, OR=12.6). The risk of PPH in singleton pregnancies from oocyte donation is higher than in MAP-O (22% vs. 9% P<0.0001, OR=2.87). When we considered severe PPH (blood loss >1000 mL) the risk for MAP-E was higher if compared to MAP-O (OR=2.1, P=0.2) and mostly to SC (OR=14, P<0.005). Compared to SC, MAP-E pregnancies showed increased OR for all the outcomes: CS (78% vs. 30.8%, P<0.001, OR=7.91); GDM (26.1% vs. 10.8%, P<0.001, OR=2.92); HDP (12% vs. 2.2%, P<0.001, OR=5.99); PPH (22% vs. 8.5%, P<0.0001, OR=3.0); SGA (16% vs. 11%, P<0.05, OR=1.16); PTB ≤34 weeks (9.4% vs. 1%, P<0.001, OR=7.94). CONCLUSIONS: Most women who undergo MAP-E are in advanced age, representing a high-risk population for obstetric complications, like HPD and PPH, which stands as the main worldwide cause of maternal mortality.

[The economic value of vaccinations: a systematic review of Italian economic evaluations and HTA reports]. / Le implicazioni economiche delle vaccinazioni: una revisione sistematica delle valutazioni economiche e di HTA condotte in Italia.

INTRODUCTION: Decision-making in healthcare should rely on evidence-based approaches able to make possible a transparent and robust assessment of all the aspects related to health technologies. One of the assessment elements is represented by the efficiency that is the specific objective of economic evaluations and also of Health Technology Assessment (HTA). The collection and synthesis of evidence is the first indispensable step in order to foster a proper convey of scientific knowledge to the decision-makers. This work, carried out within a broader project on the transfer of evidence from the scientific to the decision making world, is aimed to release an overview of economic evaluations and HTA on vaccines conducted in Italy. The project was carried out within the activities of the ISPOR Italy-Rome Chapter. METHODS: A systematic review of Italian economic evaluations and HTA performed on vaccines and published up to May 2015 was carried out. PubMed, Scopus and the NIHR HTA databases were queried and a hand-search was performed on key journals in the field (Global & Regional Health Technology Assessment; PharmacoEconomics Italian Research Articles; Giornale italiano di HTA; Politiche Sanitarie; HTA Focus - Pills of Clinical Governance; Pillole di Farmacoeconomia; Giornale Italiano di Farmacoeconomia e Farmacoutilizzazione; IJPH; Quaderni dell'IJPH). Studies were considered eligible if showing the results of a full economic evaluations and if performed in Italy. RESULTS: The literature search yielded 10 HTA reports and 33 economic evaluations. Among the latter, 20 (60,6%) were cost-effectiveness analyses. Ten studies (23,3%) assessed the vaccination against S. pneumoniae figuring out that it is cost-effectiveness and even costsaving in cases of newborns and subjects at risk. Nine studies (20,9%) addressed influenza vaccination and demonstrated its dominance on non-vaccination in the elderly. Eight studies (18,6%) evaluated the HPV vaccines concluding that they are cost-effective. Five studies (11,6%) devoted to anti-rotavirus vaccination showing its dominance on non-vaccination, in particular from the society perspective. Vaccination against pertussis, hepatitis B, chicken pox, measles, rubella, mumps were eventually shown cost-saving. The vaccine against Neisseria meningitidis was considered potentially cost-effective. CONCLUSION: The Italian scientific evidence on efficiency of vaccination is broad and allows concluding that vaccinations are value for money interventions.