Program Type: CTAF

Metachromatic Leukodystrophy

Sep 2023 | Assessment

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

Sickle Cell Disease

Jul 2023 | Assessment

Interventions of Interest: Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation of the HBB gene responsible for the β-globin component of hemoglobin; hemoglobin is the protein in red blood cells that carries oxygen. ICER will be examining the comparative clinical and value of two gene therapies: exagamglogene autotemcel (“exa-cel”, […]

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 […]

Alzheimer’s Disease

Mar 2023 | Assessment

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 […]

Chemotherapy-Induced Neutropenia

Mar 2022 | Assessment

Interventions of Interest: plinabulin (BeyondSpring Pharmaceuticals) trilaciclib (Cosela™, G1 Therapeutics) 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 […]

Hypertrophic Cardiomyopathy

Oct 2021 | Assessment

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 […]

Alzheimer’s Disease

Jul 2021 | Assessment

Interventions of Interest: During the public meeting, an independent appraisal committee voted unanimously (15-0) that the evidence was not adequate to demonstrate that aducanumab plus supportive care provides a net health benefit when compared to supportive care alone.  In light of substantial uncertainty of patient benefit, policy recommendations call for: Date of Review: July 2021 For questions, please […]

Hereditary Angioedema

Aug 2021 | Assessment

Interventions of Interest: lanadelumab (Takhzyro™,Shire) C1 esterase inhibitors (Haegarda®, CSL Behring; Cinryze®, Shire) In 2018, ICER assessed the comparative clinical effectiveness and value of therapies for hereditary angioedema, a rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. The cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks. […]

Cystic Fibrosis

Aug 2020 | Assessment

Interventions of Interest: Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals) Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals) Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) Elexacaftor/tezacaftor/ivacaftor (Trikafta®, Vertex Pharmaceuticals) Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in […]