Program Type: CTAF

Multiple Sclerosis: SPMS

Jun 2025 | Assessment

Interventions of Interest For questions please contact Becca Piltch, Program Manager, bpiltch@icer.org. View the Key Stakeholder List.

Anemia in Myelodysplastic Syndrome

Jul 2024 | Assessment

Interventions of Interest An independent appraisal committee voted that the current evidence is not adequate to demonstrate net health benefits for imetelstat added to best supportive care when compared to best supportive care alone. Imetelstat would meet common thresholds of cost effectiveness if priced between $94,800 to $113,000 per year. The therapy currently priced at […]

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 […]

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 […]

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: 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 […]

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 […]