Paroxysmal Nocturnal Hemoglobinuria
Interventions of Interest For questions please contact Becca Piltch, Program Manager, at bpiltch@icer.org. View the Key Stakeholder List.
Interventions of Interest For questions please contact Becca Piltch, Program Manager, at bpiltch@icer.org. View the Key Stakeholder List.
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 […]
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 […]
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. […]
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