— Other than a proven ability to reduce the need for IV iron supplements, the evidence is insufficient to determine if roxadustat is safer or more effective than ESAs; possibility exists for greater mortality than ESAs —
— At February 11 virtual meeting, the California Technology Assessment Forum will review the evidence, hear further testimony from stakeholders, and deliberate over treatment’s comparative clinical effectiveness, other potential benefits, and long-term value for money —
BOSTON, January 28, 2021 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of roxadustat (FibroGen) for treatment of anemia in chronic kidney disease (CKD).
“Roxadustat represents an oral alternative for patients living with anemia from CKD, and it may be particularly valuable for those not receiving hemodialysis” said David Rind, MD, MSc, ICER’s Chief Medical Officer. “However, pill burden also remains a problem for many of these patients, and other than avoiding the need for IV iron, there is significant uncertainty about whether roxadustat is safer or more effective than erythropoiesis-stimulating agents (ESAs). Given prior concerns around cardiovascular risks when ESAs are used to correct anemia, as well as the high baseline risk of such events in people with CKD, this is an important issue to fully evaluate in larger clinical trials.”
This Evidence Report will be reviewed at a virtual public meeting of the California Technology Assessment Forum (CTAF) on February 11, 2021. 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
The evidence suggests that, when compared to the ESA darbepoetin alfa, roxadustat does not significantly increase hemoglobin levels, reduce the risk of cardiovascular (CV) events, or lead to clinically meaningful differences in health-related quality of life (HRQoL); however, use of roxadustat did reduce the need for IV iron supplementation. ICER determined that the evidence is insufficient (“I”) to know whether roxadustat provides a net health benefit over an ESA.
For the population of patients who are dialysis independent (DI-CKD) and for whom ESAs were not available (i.e., where those patients would otherwise receive blood transfusions), ICER determined that roxadustat would likely provide a net clinical benefit over placebo, despite the potential for harms. In this scenario, we have rated the evidence promising but inconclusive (“P/I”).
Key Cost-Effectiveness Findings
ICER’s economic modeling shows that, at a placeholder price of $6,500 per year, roxadustat may be cost saving in the DI-CKD population with similar or improved benefit, but this includes a high degree of uncertainty. In the dialysis dependent population (DD-CKD), roxadustat may increase mortality compared with ESAs; in the absence of evidence that it is superior to ESAs, value-based pricing would suggest it cost no more than ESAs.
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.