Controversy continues to surround recombinant human erythropoetin (epoetin or EPO) for treatment of anemia among end-stage renal disease (ESRD) patients, despite 15 years for experience with the drug. Approximately 190 ESRD patients die each day – a mortality rate that has remained essentially unchanged since 1994 – regardless of improvements in the ‘adequacy of dialysis, vascular access, and anemia management’. Use of epoetin has evolved from a low dose treatment to correct severe anemia among patients with chronic kidney failure in the early 1990s to, more recently, the treatment of mild and moderate anemia using very high doses of epoetin. Understanding the relationship between treatment with epoetin and patient survival and quality of life is a necessary first step to determining what policies will achieve optimal patient benefit.
At MTPPI, we have studied epoetin therapy extensively since its approval by the FDA in 1989. Specifically, MTPPI is examining the following issues related to epoetin use:
- Extent to which the science base and policy decisions have supported the evolution in practice patterns;
- Validity of the scientific data suggesting that the mortality rate for those in the higher hematocrit range is less than for patients in the lower ranges;
- Causal effect of epoetin treatment that does not confuse the relationship between treatment response and outcomes; and
- A better understanding of the epoetin/survival relationship, well-grounded in science, to provide a basis for health care insurers to improve their current epoetin policies and to decrease patient mortality.
Current Activities
The following studies are currently being conducted by MTPPI that examine important health services research and policy implications related to epoetin:
- Use of a Time Based Analysis to Examine Causal Effects of Epoetin on Patient Survival and Outcomes
- Clinical Implications of Patient Responsiveness to Epoetin Therapy
- Use of Surrogate Outcomes to Measure Survival Among Epoetin-treated Dialysis Patients
- Monitoring Medicare’s New Prescription Drug Benefit: Case Study of Epoetin Therapy
- Analysis of Efficacy/Effectiveness of Epoetin Treatment
- Epoetin Practice Patterns (e.g., Route of Administration, Dose, Frequency of Administration, Dose Variability, Etc.) and Provider Characteristics
- Route of Administration for Epoetin Therapy: Are There Differences in Cardiovascular-Related Outcomes and Mortality
- Relationship Between Antihypertensive Therapy and Epoetin Resistance
- Role of Serum Phosphorus in Patient Outcomes Among the ESRD Population
Publications
Some recent publications prepared by the Institute in this field are:Formulation of a Medicare Epoetin Coverage and Reimbursement Policy: Need for Valid Patient Survival Information
Use of clinical guidelines for treatment of anemia among hemodialysis patients.
Secular Trends in Recombinant Erythropoietin Therapy Among the U.S. Hemodialysis Population: 1990-1996
A Cross-National Comparison of Orphan Drug Policies: Implications for the U.S. Orphan Drug Act
The Effect of Insurance Status on Use of Recombinant Erythropoietin Therapy Among End-Stage Renal Disease Patients in Three States
Epoetin Requirements Predict Mortality in Hemodialysis Patients.
Hematocrit Was Not Validated as a Surrogate End Point for Survival Among Epoetin-Treated Hemodialysis Patients