Research Type: Assessment

Post-Traumatic Stress Disorder

May 2024 | Assessment

Interventions of Interest Independent appraisal committee voted that current evidence is not adequate to demonstrate superior net health benefits for MDMA-AP compared to short-term trauma-focused psychotherapies. Click here for “Five Fast Facts on ICER and our PTSD Report.” For questions or to request a Spanish translated Report-at-a-Glance or ICER Snapshot for this assessment, please contact info@icer.org. […]

Paroxysmal Nocturnal Hemoglobinuria

Feb 2024 | Assessment

Interventions of Interest The independent appraisal committee voted that the current evidence is not adequate to demonstrate a net health benefit for iptacopan over a C5 inhibitor, although use in treatment-experienced patients with clinically significant extravascular hemolysis received more favorable votes. The committee also found that at current pricing, iptacopan represents “low” long-term value for […]

Schizophrenia

Feb 2024 | Assessment

Interventions of Interest: The independent appraisal committee voted that current evidence is not adequate to demonstrate superior net health benefits for KarXT compared to generically available aripiprazole. If long-term data confirm KarXT’s benefits and lack of weight gain, it would achieve common thresholds for cost-effectiveness if priced between $16,000 to $20,000 per year. Final Documents […]

Pulmonary Arterial Hypertension

Dec 2023 | Assessment

Interventions of Interest: Independent appraisal committee voted that sotatercept demonstrated a net health benefit when compared to background therapy; sotatercept would achieve common thresholds for cost-effectiveness if priced between $17,900 to $35,400 per year. Final Documents ICER’s Chief Medical Officer, David Rind, MD: “Pulmonary arterial hypertension is a progressive disease that can lead to debilitating […]

Metachromatic Leukodystrophy

Sep 2023 | Assessment

Interventions of Interest: Independent appraisal committee voted that across all patient subpopulations, arsa-cel demonstrated a net health benefit when compared to usual care. Arsa-cel would achieve common thresholds for cost-effectiveness if priced between $2.3M and $3.9M. Final Documents ICER’s Chief Medical Officer, David Rind, MD stated: “MLD is a devastating disease for children and their […]

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 […]