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MEDCAC Meeting 3/24/2010 - Erythopoiesis Stimulating Agents (ESA) in Anemia Related to Kidney Disease

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CMS has called this meeting of the panel to review the available evidence on the use of erythropoiesis stimulating agents (ESAs) to manage anemia in patients who have chronic kidney disease (CKD). Medicare currently does not have a national coverage determination for the use of ESAs for anemia in patients who have CKD. ESAs are synthetic analogs of the hormone erythropoietin.

Anemia is prevalent in patients with kidney disease and is more common as CKD progressively worsens. Recombinant genetic technologies are used to produce the ESAs that are commonly used to treat anemia in CKD patients. ESAs raise hemoglobin and hematocrit levels in anemic patients who have CKD, including both those on dialysis and those who do not require dialysis.

ESAs were introduced in 1989 and initially administered usually only to patients at risk for blood transfusions. During the subsequent 20 years, ESA use increased, resulting in the common practice of administering ESAs to CKD patients who have anemia, but not necessarily only to those who are at risk for transfusion. Currently, over 95% of end stage renal disease (ESRD) patients receive ESAs. It is unclear how many pre-ESRD CKD patients receive ESAs for anemia.

In the fall of 2006 through winter 2007, a succession of published reports highlighted the dangers, including increased mortality, associated with the use of ESAs. These were in addition to other danger signals seen since the introductions of ESAs in 1989. These findings, based on the results of clinical trials, were followed by a series of changes including boxed warnings in the FDA approved labeling for ESAs and a “Dear Doctor Letter” regarding concerns about the safety of ESAs.

February 19, 2010

Posted questions to panel.

March 18, 2010

Posted agenda, roster and speaker list for meeting.

March 25, 2010

Posted scoresheet [PDF, 94KB] from meeting.

April 13, 2010

Posted presentations [ZIP, 2MB] from meeting.

August 3, 2010

Poated minutes [PDF, 139KB] and transcript [PDF, 430KB] from meeting.
Federal Register Notice
Agenda
Medicare Evidence Development & Coverage Advisory Committee
March 24, 2010
7:30 AM – 4:30 PM
CMS Auditorium

Clifford Goodman, PhD, Chair
Saty Satya-Murti, MD, Vice Chair
Louis Jacques, MD, Director, Coverage and Analysis Group
Maria Ellis, Executive Secretary


7:30 – 8:00 AM  

Registration

8:00 – 8:15 AM

Opening Remarks—Maria Ellis/ Louis Jacques, MD/ Clifford Goodman, PhD

8:15 - 8:40 AM  

CMS Presentation & Voting Questions – Maria Ciccanti/Elizabeth Koller, MD

8:40 – 9:05 AM  

Thomas MaCurdy, PhD, Director, Acumen, LLC DEcIDE

9:05 – 9:15 AM  

Jerry A. Holmberg, PhD, Senior Advisor for Blood Safety, Executive Secretary of the Advisory Committee on Blood Safety and Availability, HHS

9:15 – 10:00 AM

Ajay Singh, MBBS, FRCP, MBA, Physician, Renal Chief, Brigham and Women’s Health, Associate Professor of Medicine, Harvard Medical School

10:00 – 10:15 AM  

BREAK

10:15 – 11:25 AM

Scheduled Public Comments
(Refer to Speaker List)


Public attendees, who have contacted the executive secretary prior to the meeting, will address the panel and present information relevant to the agenda. Speakers are asked to state whether or not they have any financial involvement with manufacturers of any products being discussed or with their competitors and who funded their travel to this meeting.


11:25 – 11:35 AM

Open Public Comments

Public Attendees who wish to address the panel will be given that opportunity

11:35 – 12:00 PM

Questions to Presenters

12:00 – 1:00 PM  

LUNCH (on your own)

1:00 – 2:00 PM    

Initial Open Panel Discussion: Dr. Goodman

2:00 – 3:00 PM

Formal Remarks and Voting Questions

The Chairperson will ask each panel member to state his or her position on the voting questions

3:00 – 4:00 PM   

Final Open Panel Discussion: Dr. Goodman

4:00 – 4:30 PM     

Closing Remarks/Adjournment: Dr. Jacques & Dr. Goodman

Download Meeting Minutes [PDF, 139KB].

CKD/ESA MEDCAC
March 24, 2010
Questions
  1. How confident are you that there is sufficient evidence to determine whether using a medical intervention (e.g., blood transfusion, iron therapy, or ESAs) to maintain or raise the hemoglobin or hematocrit levels of anemic CKD patients affects each of the health outcomes below?

    1. Exercise (activity) tolerance
    2. Vascular events (stroke, myocardial infarction, congestive heart failure)
    3. Patient perceived quality of life
    4. Survival
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. For any health outcome listed in Question 1 for which the panel indicates at least intermediate confidence (mean score ≥ 2.5) in the sufficiency of evidence, how confident are you that maintaining or raising hemoglobin or hematocrit levels of anemic CKD patients improves each such health outcome?
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. a. For any health outcome addressed in Question 2 for which the panel indicates at least intermediate confidence (mean score ≥ 2.5), how confident are you that there is sufficient evidence to determine whether the use of ESAs to maintain or raise hemoglobin or hematocrit levels of CKD patients improves each such health outcome?
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. For any health outcome addressed in Question 3.a. for which the panel indicates at least intermediate confidence (mean score ≥ 2.5), how confident are you that the use of ESAs to maintain or raise hemoglobin or hematocrit levels of CKD patients improves each such health outcome?
 
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. a. How confident are you that there is sufficient evidence to determine whether the use of ESAs to maintain or raise the hemoglobin or hematocrit levels of anemic CKD patients worsens any health outcome listed in Question 1?
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. For any health outcome addressed in Question 4.a. for which the panel indicates at least intermediate confidence (mean score ≥ 2.5), how confident are you that the use of ESAs to maintain or raise hemoglobin or hematocrit levels of CKD patients worsens each such health outcome?
1      —      2      —      3      —      4     —      5
Low                   Intermediate                      High
Confidence                   Confidence                       Confidence
  1. Please discuss any impact of the following factors on the conclusions reached above.
    1. Whether the CKD patient is undergoing chronic dialysis or is in pre-dialysis status.

    2. Whether the CKD patient has pretreatment baseline hemoglobin levels:
      1. < 7 g/dL
      2. ≥7g/dL to < 9 g/dL
      3. ≥9 g/dL to <12g/dL
      4. ≥ 12 g/dL

    3. Whether an appropriate target hemoglobin or hematocrit has been set for the CKD patient

    4. Whether the ESA dosing strategy has been implemented to minimize the rapidity of hemoglobin or hematocrit rise and/or oscillations in their levels

    5. Whether the CKD patient has demonstrated a blunted or “non-response” to interventions to raise hemoglobin or hematocrit

    6. Whether the CKD patient has been evaluated to determine the etiology (cause) of the anemia

    7. Whether the CKD patient demonstrates cardiac, cerebral or other vascular comorbidities

    8. Other
  1. What clinical trials designs would be most desirable to fill in any identified evidence gaps?

Download scoresheet [PDF, 94KB]

Clifford Goodman, PhD Chair
Senior Vice President
The Lewin Group

Saty Satya-Murti, MD, FAAN Vice Chair
Health Policy Consultant

Phyllis Atkinson, RN, MS, GNP-BC
Gertonological Nurse Practitioner
Co-Owner, Advanced Geriatric Education & Consulting, LLC

Virginia C. Calega, MD, MBA
Vice President
Medical Management and Policy
Highmark, Inc.

Marion Danis, MD
Chief, Bioethics Consultation Service
Head, Section on Ethics & Health Policy
NIH Clinical Center

Susan A. Levine, DVM, MS, PhD
Vice President
Technology Assessment/Editor-in Chief
Hayes, Inc.

Stephen Pauker, MD, MACP, FACC
Professor of Medicine
Division of Clinical Decision Making
Tufts University School of Medicine

Leonard M. Pogach, MD, MBA, FACP
Director, Center for Healthcare Knowledge Management for Chronic Complex Illness
VA New Jersey Healthcare System

James E. Puklin, MD
Professor of Ophthalmology
Department of Ophthalmology
Kresge Eye Institute
Chair, Human Investigation Committee
Wayne State University School
Robert L. Steinbrook, MD
Adjunct Associate Professor
Medicine and Community and Family Medicine
Dartmouth Medical School

Industry Representative
Eleanor M. Perfetto, PhD, MS
Senior Director
Evidence Based Strategies
Pfizer, Inc.

Guest Panel Members
Rajiv Agarwal, MD, FAHA, FASN
Professor of Medicine
Indiana University School of Medicine
VA Medical Center

Daniel W. Coyne, MD
Professor of Medicine
Washington University School of Medicine
Chromalloy American Kidney Center

Joseph M. Messana, MD
Associate Professor of Internal Medicine
Nephrology Division
University of Michigan Health System

Guest Speakers
Ajay Singh, MBBS, FRCP, MBA
Physician
Renal Division
Brigham and Women’s Hospital
Associate Professor of Medicine
Harvard Medical

CMS Liaison
Louis Jacques, MD
Director
Coverage and Analysis Group

Executive Secretary
Maria A. Ellis
SPEAKER LIST
*5 MINUTES PER SPEAKER*


  • Marc A. Pfeffer, MD, PhD, Dean Professor of Medicine, Harvard Medical School
  • Celeste C. Lee, MD, Chief of Staff, Office of the President & CEO, Chancellor for Health Affairs, Duke University Medical Center
  • Wolfgang Winkelmayer, MD, ScD, MPH, Associate Professor of Medicine (Acting), Stanford University School of Medicine, Representing: American Society of Nephrology
  • Kerry Willis, PhD, Senior Vice President, Scientific Activities, National Kidney Foundation
  • Scott McKenzie, MD, Senior Director, Health Economics and Outcomes Research, North America Pharmaceuticals, Centocor Ortho Biotech Products, LP (a Johnson & Johnson Company)
  • Alan S. Kliger, MD, Clinical Professor of Medicine, Yale University School of Medicine and Chairman, Department of Medicine Hospital of St. Raphael, New Haven Connecticut
  • Reshma Kewalramani, MD, Nephrology Therapeutic Area Head, Executive Director, Global Development, Amgen, Inc.
  • Kathe LeBeau, weKAN Program Manager, Renal Support Network
  • Douglas M. Silverstein, MD, Representing: American Society of Pediatric Nephrology
  • Denise Eilers, BSN, RN, Davenport, IA
  • J. Michael Lazarus, MD, Senior Executive Vice President, Fresenius Medical Care NA
  • Shad Ireland, Executive Director, Shad Ireland Foundation
  • Robert S. Rosenson, MD, FACP, FACC, FAHA, Chief of Endocrinology, Diabetes and Metabolism, Coordinator, Preventive Services, Department of Medicine and Senior Cardiologist, SUNY Downstate Medical Center and Daniel J. Cho, Vice President, ProMetrics Inc.
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