— Independent appraisal committee unanimously determined that evidence is adequate to demonstrate that beti-cel provides a net health benefit compared to standard clinical management —

— Given the high costs of standard care, cost-effectiveness modeling finds beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price is subject to an 80% payback for treatment failure —

— Consideration should be given to assigning a lower share of cost-offsets to determinations of fair pricing, but if pricing and payment agreements match those assumed in this report, payers should use the FDA label as the guide to coverage policy without seeking to narrow coverage using clinical trial eligibility criteria —

BOSTON, July 19, 2022 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of betibeglogene autotemcel (beti-cel, bluebird bio) for the treatment of beta thalassemia.

ICER’s report on these therapies was reviewed at the June 2022 public meeting of the New England CEPAC (New England CEPAC), one of ICER’s three independent evidence appraisal committees.

“Beta-thalassemia is a serious blood disorder, and while treatment has improved, patients with transfusion-dependent-thalassemia (TDT) still have decreased life expectancy and burdensome care that impacts all aspects of their lives,” said David Rind, MD ICER’s Chief Medical Officer. “Previously, a minority of patients had access to curative allogeneic bone marrow transplant; beti-cel provides an additional potentially curative option for many patients with TDT. New potentially curative therapies for beta-thalassemia bring the promise of considerable lifetime benefit, but there also remains substantial uncertainty regarding longer-term safety and the durability of benefits. Beti-cel is cost-effective at a high price in part because it offsets current very high costs of care; a somewhat lower price would be needed if half of those offsets were returned to the medical system.”

Voting on Clinical Effectiveness and Contextual Considerations

All panelists (12-0) found the evidence is adequate to demonstrate a net health benefit when beti-cel is compared to standard clinical management.

During their deliberations, panel members also weighed the therapy’s other potential benefits, disadvantages, and contextual considerations. Voting highlighted the following as particularly important for payers and other policymakers to note:

For any patient with TDT:

  • Magnitude of the lifetime impact of TDT on individuals.

The implications of treatment with beti-cel on:

  • Patients’ ability to achieve major life goals related to education, work, or family life;
  • Caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life;
  • Patients’ ability to manage and sustain treatment given the complexity of regimen;
  • A potential advantage for therapies that offer a new treatment choice with a different balance or timing of risks and benefits that may be valued by patients with different risk preferences.​

Voting on Long-Term Value for Money

Beti-cel is not yet approved by the FDA. Its manufacturers have suggested the treatment’s US price would be based on its clinical value to patients only, rather than its ability to offset costs of current therapy if approved. Traditional cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at a cumulative price of $2.1 million with an 80% payback option for patients who do not achieve and maintain transfusion independence over a five-year period.

After reviewing the clinical evidence and considering the treatments’ other potential benefits, disadvantages, and contextual considerations noted above, the New England CEPAC evaluated the long-term value for beti-cel.  A majority (9-3) of panelists found that beti-cel represents “high” long-term value for money.   

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, representatives from US payers, and the pharmaceutical manufacturer — convened to discuss the pricing implications and recommendations to ensure fair access.  Key recommendations stemming from the roundtable discussion include:

  • All stakeholders have a responsibility to facilitate meaningful patient access to curative therapies for beta thalassemia in ways that do not exacerbate disparities.
  • Should the pricing and payment structure for beti-cel match the assumptions in this report, the treatment will meet traditional cost-effectiveness standards, and payers should therefore use the FDA label as the guide to coverage policy without seeking to unduly narrow coverage using clinical trial eligibility criteria.  However, policy roundtable experts felt that it would not be unreasonable for payers to seek confirmation that patients do not have accessibility to a sibling-matched HSCT as first-line therapy. 
  • Manufacturers should align prices with independent estimates of the patient-centered therapeutic value of their treatments.  In the context of high-impact single or short-term therapies, transparent consideration should be given to a pricing scenario that “shares” any substantial cost-offset of treatment so that potentially large cost-offsets are not used to justify exceedingly high one-time prices.

ICER’s detailed set of policy recommendations, including comprehensive considerations for establishing evidence-based prior authorization criteria, 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 produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.