Interventions of Interest: plinabulin (BeyondSpring Pharmaceuticals) trilaciclib (Cosela™, G1 Therapeutics) Until recently, cytotoxic chemotherapy was the primary form of chemotherapy used to treat cancer, and it remains in widespread use today. Because it targets rapidly dividing cells, one of the common side effects of cytotoxic chemotherapy is low blood cell counts (myelosuppression), including low neutrophil […]
Interventions of Interest: Mavacamten (MyoKardia and Bristol-Myers Squibb) Hypertrophic cardiomyopathy (HCM) is a disorder of the heart due to dysfunction of the sarcomeres in cardiac muscle cells (myocytes). Sarcomeres are cellular structures that are critical in myocyte contraction. The sarcomere dysfunction in HCM can lead to hypertrophy (thickening) of the heart. HCM can be due […]
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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