— Evidence judged to demonstrate that beti-cel provides net health benefit to patients with transfusion-dependent thalassemia —
— 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 with an outcomes-based agreement —
— However, if the cost savings from successful treatment are shared equally with society, discounts between 15% and 38% off the anticipated price are required to meet commonly accepted thresholds —
— At the June 17 virtual public meeting, ICER’s independent appraisal committee will review the evidence, hear further testimony from stakeholders, and deliberate over beti-cel’s comparative clinical effectiveness, other potential benefits, and long-term value for money —
BOSTON, June 2, 2022 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of betibeglogene autotemcel or “beti-cel” (Zynteglo/LentiGlobin, bluebird bio) for the treatment of beta thalassemia.
“Beta-thalassemia is a serious blood disorder, and while treatment has improved, patients with transfusion-dependent-thalassemia (TDT) still have decreased life expectancy and large burdens of care that impact 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. The manufacturer publicly suggested pricing based on the value of beti-cel to patients rather than based on offsetting other costs, and so we are highlighting two different pricing analyses, one in which half the cost-offsets provided by elimination of the need for expensive ongoing therapy are ’shared‘ with society and not included in calculating a fair price.”
This Evidence Report will be reviewed at a virtual public meeting of the New England CEPAC (New England CEPAC) on June 17, 2022. The New England CEPAC 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
Because of the questions about the durability of clinical benefit, as well as potential risks from gene therapy, we judge that the evidence demonstrates that beti-cel is superior overall to the current standard of care, but the magnitude of that overall net health benefit is less certain, ranging from incremental to substantial (B+).
Key Cost-Effectiveness Findings
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.
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.