Program Type: CTAF

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 […]

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 […]

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 […]

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 […]

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 […]

Interventions of Interest: The independent appraisal committee voted that currently available evidence is not adequate to demonstrate a net health benefit for lecanemab when compared to supportive care. Using best estimates from current data, ICER analyses suggest lecanemab would achieve common thresholds for cost-effectiveness if priced between $8,900 – $21,500 per year. Final Documents ICER’s […]

Interventions of Interest: Trilaciclib received FDA approval in 2021 to reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage small cell lung cancer. Plinabulin was under development to prevent chemotherapy-induced neutropenia when it received a complete response letter from the FDA in December 2021 requesting a second […]

Interventions of Interest: Concerns about long-term safety influenced majority of independent appraisal committee to vote that evidence is not currently adequate to demonstrate a net health benefit of mavacamten added to background therapy. Using estimates from modeling of short-term benefits, mavacamten does show an overall benefit but would have to be priced below $15,000 per […]