Translating research into practice: Case study of erythropoietin therapy for renal failure patients

Purpose

Controversy continues to surround erythropoietin (epoetin) treatment of anemia among end-stage renal disease (ESRD) patients. Currently, 15 years after drug approval, CMS is undertaking an extraordinary review of their epoetin policy in an attempt to revise Medicare coverage and reimbursement based on evidence that enhances patient outcomes.

Methods

This paper reviews a decade of public and private sector assessments of epoetin and the ever-changing Medicare policies that have not stemmed the controversy surrounding this costly intervention.

Results

Survival studies cited in clinical practice guidelines for epoetin treatment of anemia among the ESRD population are fraught with methodologic shortcomings, but may have been viewed favorably in a continuing attempt to increase the target hematocrit level, thereby increasing the amount of epoetin used. As a result of changing reimbursement and coverage policies, epoetin doses have more than doubled in the last decade, access to epoetin is nearly universal and 40% of the ESRD population has achieved hematocrits above the recommended target level with no concomitant decline in the population mortality rate during this time. Many groups including the FDA, the research community, and the private sector are responsible for the current dilemma over appropriate epoetin dosing.

Conclusions

Translating research into practice, in the case of epoetin, has been elusive. As a result of political expediency and stakeholder pressure, tenets of evidence-based medicine were largely abandoned in addressing epoetin use. The issue is not purely academic, given the United States has the world's highest ESRD mortality rate. Hundreds of thousands of patients are currently being treated with epoetin to increase hematocrits with the firmly entrenched idea that higher hematocrits enhance survival. We contend that this belief is not founded in good science, and must be held in abeyance until sound evidence based on well designed studies is presented.

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