In 2021, the national expenditure for blood coagulation factors was 541.4 million, growing steadily over the past decade. Hemophilia A is the congenital hemorrhagic disease with the highest drug consumption and expenditure. It has also the highest annual increase. Data from the OsMed report showed an increased use of long-acting recombinant factors and a concomitant reduction in consumption of short-acting ones and also an increasing trend of emicizumab. Based on these findings, two possible expenditure scenarios were described: 1) assuming a 25% of reduction in consumption of short-acting recombinant factors with proportional redistribution to the consumption observed in 2022 for long-acting recombinant factors; 2) assuming that all new patients with a moderate and severe form of the disease will start prophylaxis with emicizumab and also calculating different switch percentages (20%, 30%, 50% or 70%). The first hypothesis showed a potential increase in expenditure of 3.3% (approximately 10 million euros) switching from short- to long-acting factors. In the second one, based on estimated number of patients with Hemophilia A on treatment, a total expenditure of about 457.6 million euros was estimated. Based on these findings, different expenditure outlooks were hypothesized and that there should be a switch from the recombinant factors to emicizumab. Specifically, an expenditure increase of 8% in the case of 20% switch and 28.1% in the case of 70% switch were estimated.

Download full-text PDF

Source
http://dx.doi.org/10.1701/4032.40075DOI Listing

Publication Analysis

Top Keywords

recombinant factors
16
expenditure
9
long-acting recombinant
8
reduction consumption
8
consumption short-acting
8
based findings
8
findings expenditure
8
factors
6
consumption
5
[pharmacotherapy hemophilia
4

Similar Publications

Hotspots of genetic change in Yersinia pestis.

Nat Commun

January 2025

State Key Laboratory of Pathogen and Biosecurity, Academy of Military Medical Sciences, Beijing, China.

The relative contributions of mutation rate variation, selection, and recombination in shaping genomic variation in bacterial populations remain poorly understood. Here we analyze 3318 Yersinia pestis genomes, spanning nearly a century and including 2336 newly sequenced strains, to shed light on the patterns of genetic diversity and variation distribution at the population level. We identify 45 genomic regions ("hot regions", HRs) that, although comprising a minor fraction of the genome, are hotbeds of genetic variation.

View Article and Find Full Text PDF

Objective: To investigate the effectiveness of initial hemostatic resuscitation(IHR) on the treatment of bleeding with recombinant human coagulation factor VIIa after cardiac surgery.

Methods: The clinical data of patients who received rFVIIa hemostatic treatment after cardiac surgery at Beijing Anzhen Hospital, Capital Medical University, from January 1, 2021, to December 31, 2021 were retrospectively collected. A total of 152 cases were included in the study.

View Article and Find Full Text PDF

Proteomic analysis of the nonstructural protein 2-host protein interactome reveals a novel regulatory role of SH3 domain-containing kinase-binding protein 1 in porcine reproductive and respiratory syndrome virus replication and apoptosis.

Int J Biol Macromol

January 2025

College of Veterinary Medicine, South China Agricultural University, Guangzhou 510642, China; Zhaoqing Branch Centre of Guangdong Laboratory for Lingnan Modern Agricultural Science and Technology, Zhaoqing 526238, China; Zhaoqing Institute of Biotechnology Co., Ltd., Zhaoqing 526238, China; Guangdong Wens Dahuanong Bio-Pharmaceutical Co., Ltd., Xinxing 527400, China. Electronic address:

Virus-host protein interaction is critical for successful completion of viral replication cycles. As the largest nonstructural protein (NSP) of porcine reproductive and respiratory syndrome virus (PRRSV), NSP2 plays multiple and critical roles in viral replication, antiviral immunity, cellular tropism and virulence. An interactome of this protein with host proteins would be instrumental in full understanding of these multifunctional roles.

View Article and Find Full Text PDF

Unraveling the complexity of HRD assessment in ovarian cancer by combining genomic and functional approaches: translational analyses of MITO16-MaNGO-OV-2 trial.

ESMO Open

January 2025

Uro-Gynecologic Oncology Unit, Istituto Nazionale Tumori IRCCS Fondazione G. Pascale, Naples, Italy. Electronic address:

Background: Ovarian cancer (OvC) constitutes significant management challenges primarily due to its late-stage diagnosis and the development of resistance to chemotherapy. The standard treatment regimen typically includes carboplatin and paclitaxel, with the addition of poly (ADP-ribose) polymerase inhibitors for patients with high-grade serous ovarian cancer (HGSOC) harboring BRCA1/2 mutations. However, the variability in treatment responses suggests the need to investigate factors beyond BRCA1/2 mutations, such as DNA repair mechanisms and epigenetic alterations.

View Article and Find Full Text PDF

Sargramostim, a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) inhalation therapy, was recently approved for pharmaceutical use in Japan and shows promise as a treatment for autoimmune pulmonary alveolar proteinosis (APAP). For APAP patients with severe respiratory failure due to advanced lung fibrosis, lung transplantation is also a treatment option; however, APAP may recur after the procedure. Here, we report a case of successful sargramostim inhalation therapy for post-transplant APAP relapse in a patient who underwent living lung transplantation owing to severe fibrosis.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!