Renal Failure Research
In terms of morbidity, mortality, and economic costs, End Stage Renal Disease (ESRD) is among the most serious chronic diseases in the United States. ESRD results from the permanent loss of kidney function and, without a regular course of dialysis or kidney transplantation, the disease is fatal. In 1991 Medicare insured approximately 93% of the 142,488 dialysis patients with kidney failure through the ESRD program established in 1973.
The following Table summarized ESRD research using enhanced medicare claims data:
Table 1. Types of Studies Contributing to the Science Base for Epoetin Therapy Among Dialysis Patients |
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Practice pattern studies - observational studies designed to report usage patterns of new and established technologies example: Cotter et.al. Kidney Int. 1998 Dec;54(6):2129-39. data source: enhanced Medicare claims |
findings: substantial increases in epoetin dose provided to anemic patients have resulted in only modest increases in hematocrit in the seven years since epoetin 's introduction. |
impact: These findings led to grant development to examine epoetin therapy and survival among dialysis patients.
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Randomized controlled trials (RCTs) - the gold-standard of tests can be very costly and might take years to complete, are limited by the type of patients included in those studies |
findings: Lower survival was found for patients assigned to complete (attainment of higher hematocrit levels) versus partial (attainment of lower hematocrit levels) correction of anemia. |
impact: These findings led to FDA issuance of a Black Box warning to curtail use of epoetin.
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Comparative effectiveness - RCTs or other studies designed to address differences in outcomes between two treats, but does not address questions of optimal use example: Thamer et.al JAMA. 2007 Apr 18;297(15):1667-74. data source: enhanced Medicare claims |
findings: Dialysis facility organiza-tional status and ownership are associated with variation in epoetin dosing in the United States; large for-profit chain facilities used larger dose adjustments and targeted higher hematocrit levels. |
impact: These findings resulted in a Congressional hearing and subsequent decision to remove perverse financial incentives related to overuse of epoetin.
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Observational studies - less costly than RCTs but can be highly confounded example: Cotter et.al.. J Clin Epidemiol. 2004 Oct;57(10):1086-95. data source: enhanced Medicare claims |
findings: Our results support earlier clinical trial and epidemiological data suggesting that hematocrit might not be a valid surrogate for survival among the epoetin-treated renal failure population. |
impact: These findings challenged assumption contained existing treatment guidelines for anemia management.
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Epidemiological studies - also less costly than RCTs, marginal structural model (MSM) observational studies, designed to mimic RCTs, are based on real-world data. The MSM technique adjusts for time dependent confounding that establishes the causal link between treatment and clinical outcomes example: #1 Cotter et. al. Kidney Int. 2008 Feb;73(3):347-53. and #2 Zhang et. al. Clin J Am Soc Nephrol. 2009 (accepted) data source: enhanced Medicare claims |
#1 findings: The dose–response curve found in our study suggests that a starting dose of 7500–15 000 units per week can maintain the hematocrit level in the desired target range of 33–36%. Given epoetin overuse concerns, the results from our analysis demonstrate the lowest requisite dose for patients. |
#1 impact: These findings have implications for establishing a fair an equitable Medicare payment for epoetin treatment.
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#2 findings: Our findings suggest that, on average, epoetin doses greater than 30,000 U/wk do not confer additional harm or benefit in elderly hemodialysis patients. |
#2 impact: These findings further challenge previous presumptions for a clinical benefit related to higher (aggressive) epoetin dose levels.
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Current work includes:
· Recombinant Human Erythropoietin
MTPPI recently announced an NIH grant to fund a study titled "Epoetin Therapy and Survival of Hemodialysis Patients". The abstract of that study is available here
Institute researchers will also be giving a talk at the 2005 National Kidney Foundation meeting in Washington, DC entitled "Making Sense of Retrospective Database Studies- How Much Weight Do We Give to These Studies?" For further information regarding the meeting, click on: http://www.kidney.org/meetings/clinical/pdf/CM05_Physicians.pdf
The Institute is also preparing comments on the rule proposed on 2/4/2005 by the Centers for Medicare and Medicaid Services regarding conditions for coverage: http://www.cms.hhs.gov/cop/3818.pdf. Our comments related to the proposed rule for monitoring claims for erythropoietin is also available here
Recent and upcoming posters:
· Factors affecting route of administration for Epoetin therapy among U.S. dialysis patients. ISPOR 10th Annual International Meeting, Marriott Wardman Park Hotel, Washington DC, May 15-18 2005
· Epoetin therapy and survival: Evidence-based reviews, clinical practice guidelines, and regulatory decisions. 41st DIA Annual Meeting, Washington, DC June 26-30, 2005
· Therapeutic risk management: A case study of recombinant human erythropoetin therapy among renal failure patients. 21st International Conference on Pharmacoepidemiology and Therapeutic Risk Management, Nashville, TN, August 21-24, 2005 (accepted for presentation).
· Translating research into practice: Case study of erythroietin therapy for renal failure patients. American College of Epidemiology (ACE) Conference, New Orleans, September 17 - 20, 2005 (accepted for presentation)
Past Activities The following studies were conducted by MTPPI or have been submitted for publication. They examine important health services research and economic issues related to renal failure:
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Do you have an idea for a research topic? MTPPI is always looking for research topics that can help the community and industry better utilize new medical technologies. Let us know. |
