Research Type: Assessment

Paroxysmal Nocturnal Hemoglobinuria

Feb 2024 | Assessment

Interventions of Interest For questions please contact Becca Piltch, Program Manager, at bpiltch@icer.org. View the Key Stakeholder List.

Schizophrenia

Feb 2024 | Assessment

Interventions of Interest: Schizophrenia is a serious mental illness that affects how a person thinks, feels, and behaves. The symptoms are typically separated into three general categories: positive (delusions, hallucinations, disorganized speech, thought and behavior); negative (poor motivation, lack of pleasure and enjoyment, lack of speech, lack of social interaction), and cognitive (impaired executive function, […]

Pulmonary Arterial Hypertension

Dec 2023 | Assessment

Interventions of Interest: Pulmonary arterial hypertension (PAH) is a rare, progressive disease characterized by thickening of the walls of the pulmonary arteries that carry blood from the right side of the heart to the lungs, leading to increased pulmonary vascular resistance. The increased pressure in the pulmonary arteries makes it more difficult for the heart […]

Metachromatic Leukodystrophy

Sep 2023 | Assessment

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

Sickle Cell Disease

Jul 2023 | Assessment

Interventions of Interest: Despite uncertainty regarding longer-term outcomes, lovo-cel and exa-cel both estimated to produce substantial gains in length and quality of life; independent appraisal committee voted that currently available evidence is adequate to demonstrate a net health benefit over standard of care. Current evidence suggests that lovo-cel and exa-cel would achieve common thresholds for […]

Non-Alcoholic Steatohepatitis

Apr 2023 | Assessment

Interventions of Interest: An independent appraisal committee narrowly voted that currently available evidence for resmetirom is adequate to demonstrate a net health benefit over lifestyle management, whereas current evidence for obeticholic acid was deemed inadequate to demonstrate a net health benefit. ICER analyses suggest resmetirom would achieve common thresholds for cost-effectiveness if priced between $39,600 – $50,100 […]

Menopause: Vasomotor Symptoms

Dec 2022 | Assessment

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 […]

Hemophilia A and B

Nov 2022 | Assessment

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 […]

Multiple Sclerosis: CIS, RRMS, and SPMS

Jan 2023 | Assessment

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 […]

Obesity Management

Sep 2022 | Assessment

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 […]