There are significant uncertainties regarding long term efficacy and safety of imetelstat; treatment would achieve common thresholds for cost-effectiveness if priced between $94,800 to $113,000 per year —

BOSTON, July 2, 2024 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of imetelstat (Rytelo, Geron Corporation) for the treatment of anemia in myelodysplastic syndrome (MDS).  

“Patients that have anemia related to MDS may have to plan around frequent blood transfusions, which can significantly affect their daily activities,” said ICER’s Chief Scientific Officer and Director of Health Technology Assessment Methods and Engagement, Dan Ollendorf, PhD, MPH. “Imetelstat is a new treatment option for adults with low-to-intermediate MDS and transfusion-dependent anemia. While available clinical evidence suggests that imetelstat may reduce or eliminate the need for transfusions, its impact on the severe fatigue that often accompanies MDS anemia is less clear. There is no evidence to suggest that imetelstat reduces the progression or trajectory of MDS itself, and there are some key side effects of concern. As a result, we view the evidence as promising but inconclusive, and the current list price is not at all aligned with the modest benefit we do see.”

This Evidence Report will be reviewed at a virtual public meeting of the CTAF on July 19, 2024. The CTAF is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

Compared with best supportive care, the net benefit of imetelstat is promising but inconclusive (“P/I”). There are clear benefits in the reduction of transfusions, but the sustained improvement in fatigue is modest.

Compared with luspatercept, ICER rated the evidence for imetelstat as insufficient (“I”). There is no evidence suggesting greater reductions in transfusions or improvements in quality of life for imetelstat.

Key Cost-Effectiveness Findings

Imetelstat has been approved by the FDA and has a list price of approximately $365,000 per year. ICER has calculated a health-benefit price benchmark (HBPB) for imetelstat to be between $94,800 to $113,000 per year.

ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent, non-profit research institute that conducts evidence-based reviews of health care interventions, including prescription drugs, other treatments, and diagnostic tests. In collaboration with patients, clinical experts, and other key stakeholders, ICER analyzes the available evidence on the benefits and risks of these interventions to measure their value and suggest fair prices. ICER also regularly reports on the barriers to care for patients and recommends solutions to ensure fair access to prescription drugs. For more information about ICER, please visit