Independent appraisal committee unanimously determined that evidence is adequate to demonstrate that beti-cel provides a net health benefit compared to standard clinical management.
Evidence judged to demonstrate that beti-cel provides net health benefit to patients with transfusion-dependent thalassemia.
Independent appraisal committee found the evidence is adequate to demonstrate that eculizumab and efgartigimod both provide a net health benefit when compared to conventional therapy alone.
Given the risk of side effects and the uncertainty of benefit, ICER assigns a draft rating of “insufficient” to the strength of evidence on whether aducanumab provides a net health benefit for patients. Results are preliminary and subject to change based on public comment, which ICER is accepting through June 2, 2021.
AbstractBackground: Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of treatment. Nusinersen is a disease-modifying therapy for patients with SMA.Objective: The aim of this study was to estimate the cost-effectiveness of nusinersen compared to […]
Interventions of Interest: remdesivir (VEKLURY®) ICER released the results of its initial analyses to inform public debate of pricing for remdesivir (Gilead Sciences) and other future treatments of COVID-19. Working with external academic partners, ICER has developed ICER-COVID models comprising two alternative pricing paradigms for COVID-19 treatments: 1) “cost recovery” for the manufacturer, representing an […]
— Based on an analysis of four studies evaluating remdesivir’s influence on mortality, ICER has concluded that the totality of evidence no longer supports an assumption of survival benefit from remdesivir; however, new data from US-based ACTT-1 trial does support savings on insurer payment for hospital services — — Given these new data and remdesivir’s expanded […]
— Independent appraisal committee votes that evidence is adequate to demonstrate clinical superiority of vedolizumab over adalimumab — — Due to competition introduced with biosimilars, the price for infliximab has dropped substantially in recent years; although prices are still marginally above traditional cost-effectiveness thresholds, the majority of independent appraisal committee members votes that infliximab and […]
ICER developed a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Luxturna®, Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be approved by the US Food and Drug Administration (FDA). The appraisal committee vote revealed differences […]
Value Assessment Framework The ICER value framework describes the conceptual framework and set of associated methods that guide the development of ICER evidence reports. The purpose of the value framework is to form the backbone of rigorous, transparent evidence reports that, as a basis for broader stakeholder and public engagement, will help the United States […]