Research Type: Assessment

Transthyretin Amyloidosis Cardiomyopathy

Sep 2024 | Assessment

Interventions of Interest Transthyretin amyloid cardiomyopathy (ATTR-CM) is a type of heart muscle disease that occurs when amyloid fibrils – clumps of misfolded proteins – are deposited into heart tissue and cause it to stiffen. Eventually, the heart cannot fill properly, leading to shortness of breath, heart failure, and death. For questions please contact Liis […]

Myelodysplastic Syndrome

Jul 2024 | Assessment

Interventions of Interest Myelodysplastic syndromes (MDS) are an uncommon group of disorders characterized by abnormal blood-forming cells in the bone marrow, resulting in the reduction of peripheral blood cells, an elevated risk of acute myeloid leukemia (AML), and reduced survival.Anemia (low red blood cell counts), thrombocytopenia (low platelet counts), and leukopenia (low white blood cell […]

Chronic Obstructive Pulmonary Disease

Jun 2024 | Assessment

Interventions of Interest Chronic obstructive pulmonary disease (COPD) is a group of lung diseases characterized byprogressive and persistent airflow obstruction in the lungs. COPD affects approximately 15.7million people in the United States, with higher rates among non-Hispanic White individuals, Native American individuals, women, and adults older than 65. For questions please contact Liis Shea, Senior […]

Post-Traumatic Stress Disorder

May 2024 | Assessment

Interventions of Interest Post-traumatic stress disorder (PTSD) is a complex psychiatric disorder associated with substantial disability and poor quality of life that occurs in people who have experienced or witnessed one or more traumatic events. Traumatic events can include natural disasters, serious accidents, war and combat, rape and sexual assault, intimate partner violence and bullying. […]

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 […]