Research Type: Assessment

Interventions of Interest: For questions please contact Kelsey Gosselin, Program Manager, at kgosselin@icer.org. View the Key Stakeholder List.

Interventions of Interest: Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation of the HBB gene responsible for the β-globin component of hemoglobin; hemoglobin is the protein in red blood cells that carries oxygen. ICER will be examining the comparative clinical and value of two gene therapies: exagamglogene autotemcel (“exa-cel”, […]

Interventions of Interest: For questions, please contact Liis Shea, Program Director, at lshea@icer.org. View the Key Stakeholder List.

Interventions of Interest: The independent appraisal committee voted that evidence is not yet adequate to demonstrate a net health benefit for fezolinetant when compared to no pharmacological treatment. Using point estimates from short-term clinical trials, analyses suggest this drug would achieve common thresholds for cost-effectiveness if priced between $2,000 – $2,600 per year for women […]

Interventions of Interest: Fair pricing benchmarks suggest upper bounds for price of Roctavian at approximately $1.9 M and for Hemgenix at approximately $2.9 M. Payers should work with manufacturers to develop and implement outcomes-based agreements to address the uncertainty and the high cost of gene therapies for hemophilia. Finally, clinical experts affirm that it would […]

An independent appraisal committee voted that evidence is not adequate to demonstrate a superior net health benefit for ublituximab when compared to other monoclonal antibodies. Analyses suggest ublituximab and all currently available monoclonal antibodies labeled for MS would achieve common thresholds for cost-effectiveness if priced between $16,500- $34,900 per year, a range far lower than […]

Interventions of Interest: subcutaneous semaglutide (Wegovy, Novo Nordisk) liraglutide (Saxenda, Novo Nordisk) phentermine/topiramate (Qsymia, Vivus Pharmaceuticals) naltrexone/bupropion (Contrave, Currax Pharma) The independent appraisal committee voted that evidence is adequate to demonstrate that semaglutide provides a net health benefit compared to lifestyle modification alone; however, at current pricing, semaglutide represents “low” long-term value for money. Semaglutide […]

Interventions of Interest: AMX0035 (RELYVRIO™, Amylyx Pharmaceuticals, Inc.) oral edaravone (Mitsubishi Tanabe Pharma Development America, Inc.) The majority of independent appraisal committee voted that the evidence is adequate to demonstrate that both AMX0035 and oral edaravone (in population narrower than its label) provide a net health benefit compared to standard of care. They also determined […]

Key Updates: Interventions of Interest: For questions or to request a Spanish translated Report-at-a-Glance for this assessment, please contact Kelsey Gosselin, Program Manager, at kgosselin@icer.org. View the Key Stakeholder List.

Interventions of Interest: The independent appraisal committee unanimously determined that the evidence is adequate to demonstrate that beti-cel provides a net health benefit compared to standard clinical management. Given the high costs of standard care, cost-effectiveness modeling finds beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price […]