Intervontions of Interest: etranacogene dezaparvovec (CSL Behring) valoctocogene roxaparvovec (Roctavian ™; BioMarin) ICER reviewed valoctocogene roxaparvovec for hemophilia A in 2020 (Valoctocogene Roxaparvovec and Emicizumab for Hemophilia A without Inhibitors: Effectiveness and Value). In this review, the two interventions will be considered separately as if we were performing two independent reviews in two different populations. […]
Key Updates: As a result of additional information recently becoming available, ICER has decided to extend the timeline for its upcoming report on Alzheimer’s disease, “Beta-Amyloid Antibodies for Early Alzheimer’s Disease” and add lecanemab back into the report. For more information, click here. ICER decided to extend its timeline for assessing beta-amyloid antibodies for early […]
Interventions of Interest: plinabulin (BeyondSpring Pharmaceuticals) trilaciclib (Cosela™, G1 Therapeutics) Trilaciclib received FDA approval in 2021 to reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage small cell lung cancer. Plinabulin was under development to prevent chemotherapy-induced neutropenia when it received a complete response letter from the […]
Interventions of Interest: mavacamten (Camzyos™, MyoKardia and Bristol-Myers Squibb) Concerns about long-term safety influenced majority of independent appraisal committee to vote that evidence is not currently adequate to demonstrate a net health benefit of mavacamten added to background therapy. Using estimates from modeling of short-term benefits, mavacamten does show an overall benefit but would have […]
Interventions of Interest: aducanumab (Aduhelm™, Biogen) During the public meeting, an independent appraisal committee voted unanimously (15-0) that the evidence was not adequate to demonstrate that aducanumab plus supportive care provides a net health benefit when compared to supportive care alone. In light of substantial uncertainty of patient benefit, policy recommendations call for: Reducing the price to avoid […]
Interventions of Interest: lanadelumab (Takhzyro™,Shire) C1 esterase inhibitors (Haegarda®, CSL Behring; Cinryze®, Shire) In 2018, ICER assessed the comparative clinical effectiveness and value of therapies for hereditary angioedema, a rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. The cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks. […]
Interventions of Interest: Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals) Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals) Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) Elexacaftor/tezacaftor/ivacaftor (Trikafta®, Vertex Pharmaceuticals) Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in […]
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