Real-world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe.

INTRODUCTION: The real-world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real-world evidence (RWE) on the performance of prophylactic use of rFVIIIFc. AIM: The aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe. METHODS: We searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality-of-life measures. RESULTS: 46 eligible publications (eight full-text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half-life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105 IU/kg/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors. CONCLUSION: rFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real-world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.

Semaglutide versus tirzepatide for people with type 2 diabetes: cost of glycemic control in Austria, the Netherlands, Lithuania, and the United Arab Emirates.

OBJECTIVES: The glucagon-like peptide-1 agonist semaglutide and the dual glucose-dependent insulinotropic polypeptide tirzepatide have proven to significantly reduce glucose levels in people with type 2 diabetes. However, the costs needed to achieve a sustained decrease in HbA1c level and disease control with semaglutide and tirzepatide, respectively, are unclear. Therefore, this study aimed to compare the cost of treatment with semaglutide with the cost of treatment with tirzepatide for type 2 diabetes in Austria, the Netherlands, Lithuania, and the United Arab Emirates in order to determine their respective value for money. METHODS: The primary outcome of this analysis was the cost in euros needed to achieve disease control in one person with type 2 diabetes based on the composite endpoint of HbA1c <7%, ≥5% weight loss, and no hypoglycemic events. In addition, analyses regarding the cost needed to reach relevant HbA1c endpoints were performed. Clinical data were obtained from the SURPASS 2 trial, registered at clinicaltrials.gov (NCT03987919), and drug cost was based on wholesale acquisition cost or pharmacy purchase prices from public domains obtained in Q1 of 2023. RESULTS: The cost needed to achieve disease control in one person with type 2 diabetes (HbA1c <7%, ≥5% weight loss, and no hypoglycemic events) was up to three times lower using semaglutide compared with all three doses of tirzepatide in most markets. In the HbA1c analyses, semaglutide was also found to be the least expensive treatment option. CONCLUSION: Semaglutide provides better value for money than tirzepatide for HbA1c-lowering endpoints.

Neurologists' views on patient reported outcomes in multiple sclerosis care.

Background: The value that patient reported outcomes (PROs) can bring to the clinical encounter is increasingly being recognized. Within the field of Multiple Sclerosis (MS), a number of activities have been initiated internationally with the aim of integrating PROs in MS care. Integration of PROs in MS care will, among other things, require MS neurologists' acceptance of PROs. This qualitative study aimed to explore MS specialized neurologists' view on the potentials and barriers for the use of PROs in the clinical setting. Methods: Eight neurologists specialized in MS participated in a series of individual in-depth semi-structured interviews. Interviews were audio-recorded and transcribed. A thematic analysis was conducted using a reflexive thematic approach to identify meaning units and themes emerging from the interviews. The analysis focused on barriers and potentials for PROs that relate to the specific characteristics of MS. Results: Three main themes emerged from the data. "The role of PROs in describing the patient's situation" describes how informants perceived MS as a complex disease, and in this context, PROs can bring forth new or otherwise hidden information, but they may also blur the picture. The theme "The validity of PROs reported by MS patients may be questionable" describes how impairments caused by MS, such as cognitive and physical disabilities, were identified as risk factors that could cause unreliable self-reported outcome measures within this patient group. Finally, the theme "Involving the patient" regards how the clinical conversation was viewed as the most important source of patient reported information, but at the same time PROs carry the potential to enhance shared decision making. Conclusion: This study indicates that, according to MS neurologists, integration of PROs in MS clinical practice, though possibly valuable, is not without challenges. Possible benefits of PROs include the ability to bring forth otherwise hidden information on the patient's health status and the enhancement of shared decision making. Barriers include difficulties in capturing the full situation of the patient via PROs due to the complexity of MS as well as the issue of various MS-related impairments compromising the validity of PROs reported by MS patients.

The substantial costs to society associated with obesity - a Danish register-based study based on 2002-2018 data.

INTRODUCTION: The Danish national health registers were used to investigate the economic burden of obesity, associated costs of comorbidities and a breakdown into direct and indirect costs. METHODS: The study population comprised all Danish adult citizens registered with a hospital diagnosis of obesity in the Danish National Patient Register between 2002 and 2018. Cases were matched with five controls via the Danish Civil Registration System. We estimated the difference in total healthcare costs and indirect costs between cases and controls and the difference in healthcare resource utilization. In a sub-analysis, we estimated total healthcare costs for persons who had been registered with one or more of 11 predefined comorbidities. RESULTS: People with obesity experienced a statistically significant twofold increase in average direct healthcare costs per year (EUR 5,934), compared with controls (EUR 2,788) and had statistically significantly higher indirect costs compared to controls. Total healthcare costs for people with obesity and one or more of the 11 comorbidities were 91.7%-342.8% higher than total healthcare costs of the population with obesity but none of the 11 comorbidities. CONCLUSION: Obesity was associated with an increase in both direct and indirect costs. The presence of comorbidities was associated with additional healthcare costs. KEY POINTS: Obesity is associated with an increase in direct and indirect costs in Denmark.Comorbidities are associated with additional healthcare costs.