相关临床试验
5
3 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
已完成
1
20.0%
尚未招募
3
60.0%
招募中
1
20.0%
暂无批准数据
- A comprehensive systematic review of 17 pharmacoeconomic studies found that emicizumab prophylaxis is more cost-effective than bypassing agents in hemophilia A patients with inhibitors across multiple countries. - The cost-effectiveness of emicizumab compared to recombinant factor VIII varies significantly across different healthcare systems and economic settings, with mixed results depending on local pricing and GDP levels. - Emicizumab demonstrated lower cost-effectiveness compared to newer treatments including recombinant factor VIII Fc fusion protein and gene therapy valoctocogene roxaparvovec in patients without inhibitors. - The review identified significant quality gaps in current pharmacoeconomic studies, with 76% lacking adequate stakeholder engagement and 58% missing subgroup analysis methods.
• Maintaining high Factor VIII (FVIII) levels in people with hemophilia A provides significant clinical benefits, including reduced bleeding risk and better joint health preservation. • Despite advances in prophylactic treatment, standard-of-care prophylaxis does not completely prevent joint damage or chronic pain, with approximately 47% of hemophilia patients experiencing ongoing pain. • Recent innovations in hemophilia treatment, including extended half-life therapies, non-factor replacement options, and gene therapy, offer new approaches to sustaining higher FVIII levels and improving patient outcomes.
• Sanofi has voluntarily suspended its phase 3 clinical trials of fitusiran, an RNAi therapeutic for hemophilia A and B, following reports of non-fatal thrombotic events in study participants. • The safety pause, announced by major hemophilia patient organizations, marks the second significant safety setback for fitusiran, following a previous trial halt in 2017 due to a patient death. • The development impacts Sanofi's strategic pipeline, as fitusiran was positioned to compete with Roche's Hemlibra in the hemophilia treatment market.
