ICER suggests $119K price for Geron's imetelstat ahead of FDA decision

Despite anticipation and a favorable FDA panel vote for Geron’s imetelstat, a treatment for certain patients with myelodysplastic syndromes (MDS), the telomerase inhibitor did not sufficiently impress a U.S. drug pricing watchdog. Leading up to imetelstat’s regulatory decision on June 16, the Institute for Clinical and Economic Review (ICER) released a draft report suggesting that the expected annual cost of the anemia treatment would need to be halved to be cost-effective.

ICER's concerns primarily centered around the incremental improvements in patient symptoms and the treatment's side effects. In the Phase III IMerge trial, imetelstat led to a 50% improvement in fatigue, the most troubling symptom for MDS patients, compared to 40% for the placebo. However, patients on imetelstat experienced a higher incidence of severe cytopenias, with grades 3 and 4 being particularly prevalent.

The safety issues were debated during a March FDA advisory committee meeting, though the majority of participants agreed that the improvement in transfusion independence, achieved by 40% of patients in the IMerge study, outweighed the risks associated with cytopenias. The panel voted 12-2 in favor of imetelstat. Amer Zeidan, an associate professor of medicine at Yale University and chief of the Division of Hematologic Malignancies, acknowledged the cytopenia concerns but deemed the side effect manageable. Zeidan emphasized the high rate of patients who achieved and maintained transfusion independence for about a year, calling the results "clinically meaningful" for heavily transfusion-dependent patients.

According to ICER’s lifetime time horizon model, the use of imetelstat alongside best supportive care may only offer modest gains in quality-adjusted life years for MDS patients. With an estimated annual price of $250,000, imetelstat would surpass commonly used price thresholds, rendering it not cost-effective. At this price, each additional life year gained by a patient would cost approximately $708,000. To meet ICER’s $200,000 per quality-adjusted life year threshold, the price of imetelstat would need to be reduced to about $119,000 annually.

Despite these financial concerns, ICER also noted that a $250,000 annual price tag for imetelstat would not exceed its budget impact threshold of $735 million per year, assuming all eligible patients were treated. This suggests that while the treatment is expensive, it may still be manageable within the broader healthcare budget.

In summary, while imetelstat shows promise in improving transfusion independence and managing fatigue in MDS patients, its high cost and significant side effects present challenges. The drug's anticipated pricing raises concerns about its cost-effectiveness, suggesting that a significant price reduction would be necessary to justify its use from an economic standpoint. The FDA’s final decision and potential pricing adjustments will ultimately determine imetelstat's place in treating MDS.