Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatment for Anemia in Myelodysplastic Syndrome

— Independent appraisal committee voted that current evidence is not adequate to demonstrate net health benefits for imetelstat added to best supportive care when compared to best supportive care alone; treatment would meet common thresholds of cost effectiveness if priced between $94,800 to $113,000 per year, therapy currently priced at approximately $365,000 per year —

BOSTON, August 22, 2024 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of imetelstat (Rytelo, Geron Corporation) for the treatment of anemia in patients with low-to-intermediate risk myelodysplastic syndrome (MDS). 

ICER’s report on this treatment was the subject of the July 2024 public meeting of the of the CTAF, one of ICER’s three independent evidence appraisal committees. 

Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations 

“MDS-associated anemia requires frequent blood transfusions, which pose a significant burden for patients and their caregivers,” said ICER’s Chief Scientific Officer and Director of Health Technology Assessment Methods and Engagement, Dan Ollendorf, PhD, MPH. “While current data suggest that imetelstat may reduce or eliminate the need for transfusion in some patients, there is no evidence of its effect on the trajectory of MDS itself, there are key side effects of concern, and perhaps most importantly, the price set by the company is far out of line with the modest short term benefit suggested by the clinical trials, especially given the uncertainty regarding those results. Such excessive pricing may in fact limit patient access to this new treatment option.”

ICER’s Public Meeting: Voting Results on Clinical Effectiveness and Benefits Beyond Health 

ICER assessed, and the independent appraisal committee voted on, the evidence of imetelstat for patients with lower risk myelodysplastic syndrome without the del(5q) mutation who are transfusion dependent and ineligible for, or refractory to erythropoiesis-stimulating agents (ESAs): 

A majority of panelists (11-4) found that current evidence is not adequate to demonstrate a net health benefit for imetelstat plus best supportive care when compared to best supportive care alone. 

For patients with lower risk myelodysplastic syndrome without the del(5q) mutation who are transfusion dependent and ineligible for, or refractory to ESAs, and are ring sideroblast positive:

All panelists (15-0) found that current evidence is not adequate to demonstrate a net health benefit for imetelstat plus best supportive care when compared to luspatercept plus best supportive care. 

Panel members also weighed potential benefits and disadvantages beyond the direct health effects and special ethical priorities. Voting highlighted the following as particularly important for payers and other policymakers to note: 

There is substantial unmet need despite currently available treatments.

ICER’s Public Meeting: Voting Results on Long-Term Value for Money 

For patients with lower risk myelodysplastic syndrome without the del(5q) mutation who are transfusion dependent and ineligible for, or refractory to ESAs: 

A majority of panelists (14-1) found at the current pricing, imetelstat plus best supportive care (compared to best supportive care alone) represents a “low” long-term value for money.

For patients with lower risk myelodysplastic syndrome without the del(5q) mutation who are transfusion dependent and ineligible for, or refractory to ESAs, and are ring sideroblast positive:

A majority of panelists (13-2) found at the current pricing, imetelstat plus best supportive care (compared to luspatercept plus best supportive care) represents a “low” long-term value for money.

On June 6, 2024, imetelstat was approved by the FDA. The manufacturer announced a US 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.

Key Policy Recommendations: 

ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including clinical experts, patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include: 

Payers should use the inclusion and exclusion criteria from the Phase 3 trial of imetelstat as the guide to coverage policy, and engage clinical experts and diverse patient representatives in considering how to address coverage issues for which there is limited or no evidence at the current time.  

Specialty societies should ensure adequate training of community hematologists about new therapies like imetelstat.

Manufacturers should set prices that will foster affordability and good access for all patients by aligning prices with the patient-centered therapeutic value of their treatments. In the setting of these new interventions for MDS, while there is considerable hope associated with the promise of the therapies, there also remains substantial uncertainty regarding their longer-term safety and effectiveness. Manufacturer pricing should also reflect these considerations in moderating launch pricing.

Patient organizations have a vital role to play to promote objective descriptions of the risks and benefits of new therapies in order to support shared decision-making for every patient. In addition, patient groups have a powerful voice and should apply it to create significant pressure for fair pricing and appropriate insurance coverage across all sectors of the health system.

ICER’s detailed set of policy recommendations is available in the Final Evidence Report and in the standalone  Policy Recommendations document. 

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 www.icer.org.