Anti-VEGF Treatment of Diabetic Macular Edema

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=60 | meeting_date=2012-03-21

The Centers for Medicare and Medicaid Services (CMS) has convened this meeting for the panel to review the available evidence on anti-vascular endothelial growth factor (anti-VEGF) treatment of diabetic macular edema (DME). The standard of care for DME has been focal and grid laser, and we are interested in evaluating results of studies using anti-VEGF agents to treat this condition. CMS does not have a national coverage determination for the use of anti-VEGF treatment in DME. In diabetic retinopathy there may be damage to or formation of small blood vessels in the eye's light-sensitive retinal tissue. If these damaged blood vessels begin to leak near the center of the retina, known as the macula, the surrounding area swells and causes a condition called macular edema. DME can lead to vision loss if left untreated. Repeated eye injections of anti-VEGF medications may help to manage these conditions, preventing visual loss and promoting recovery.

Actions Taken: December 29, 2011 Updated Panel Voting Questions to align with ongoing Technology Assessments March 2, 2012 Posted Technology Assessment for meeting. March 8, 2012 Posted additional Technology Assessment for meeting. March 16, 2012 Posted presentations [PDF, 11KB] for meeting. March 16, 2012 Posted agenda , roster and speakers list March 23, 2012 Posted scoresheet [PDF, 38KB] from meeting. May 10, 2012 Posted minutes [PDF, 130KB] and transcript [PDF, 318KB] from meeting. Registration Information about visiting CMS Webcast of meeting Agenda: Federal Register Notice Agenda Medicare Evidence Development & Coverage Advisory Committee March 21, 2012 7:30 AM – 4:30 PM CMS Auditorium Clifford Goodman, PhD, Chair Steve Phurrough, MD, Vice Chair James Rollins, MD, Division Director, Division of Items and Devices, Coverage and Analysis Group Maria Ellis , Executive Secretary 7:30 – 8:00 AM Registration 8:00 – 8:20 AM Opening Remarks— Maria Ellis/James Rollins, MD/Clifford Goodman, PhD 8:20 - 8:30 AM CMS Presentation & Voting Questions – Kimberly Long 8:30 – 9:00 AM Robert Frank, MD , The Robert S. Jampel, MD, PhD, Professor of Ophthalmology and Professor of Anatomy and Cell Biology, Wayne State University School of Medicine 9:00 – 9:30 AM TA Presentation: Donna M. Dryden, PhD, Associate Director, University of Alberta, Evidence-based Practice Center 9:30 – 10:00 AM TA Presentation: Daniel Ollendorf, MPH, ARM, Chief Review Officer, Institute for Clinical and Economic Review 10:00 – 10:15 AM BREAK 10:15 – 11:00 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. ****************************************************************************** 11:00 – 11:15 AM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity 11:15 – 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. Rollins & Dr. Goodman Issue: The Centers for Medicare and Medicaid Services (CMS) has convened this meeting for the panel to review the available evidence on anti-vascular endothelial growth factor (anti-VEGF) treatment of diabetic macular edema (DME). The standard of care for DME has been focal and grid laser, and we are interested in evaluating results of studies using anti-VEGF agents to treat this condition. CMS does not have a national coverage determination for the use of anti-VEGF treatment in DME. In diabetic retinopathy there may be damage to or formation of small blood vessels in the eye's light-sensitive retinal tissue. If these damaged blood vessels begin to leak near the center of the retina, known as the macula, the surrounding area swells and causes a condition called macular edema. DME can lead to vision loss if left untreated. Repeated eye injections of anti-VEGF medications may help to manage these conditions, preventing visual loss and promoting recovery. Minutes: Download meeting minutes [PDF, 130KB] Other Material: Transcript 1 Panel Voting Questions: Intravitreal Targeted Treatment of Diabetic Retinal Disease: Diabetic Macular Edema (DME) MEDCAC QUESTIONS Outcomes of Interest: CMS is most interested in meaningful changes to beneficiaries’ visual function that enable their independent accomplishment of routine daily activities. We also seek the panel’s input on the preferred measures for determining progression in clinical trials of DME treatment. DISCUSSION QUESTION In a 2005 MEDCAC on wet age-related macular degeneration (WAMD), the following commonly used outcomes or intermediate endpoint measures were discussed: Visual acuity VFQ 25 Dilated eye exam (to assess retinal damage) Grade of diabetic retinopathy (DR) Amsler grid Extent/progression as measured by retinal photography Fluorescein angiography (to assess blood flow/leakage in retina and choroid) Visual fields Ocular coherence tomography (OCT) (to assess retinal thickening, other damage) Please discuss the suitability of these measures for assessing DME treatment-related health outcomes, i.e., benefits and harms. VOTING QUESTIONS For the voting questions, use the following scale identifying level of confidence - with 1 being the lowest or no confidence and 5 representing a high level of confidence. 1 2 3 4 5 Low Intermediate High How confident are you that there is adequate evidence to determine whether or not DME management using intravitreal targeted anti-VEGF treatment improves patient health outcomes compared to DME management without intravitreal targeted anti-VEGF treatment? 1 2 3 4 5 Low Intermediate High If the result of Question 2 is at least intermediate (mean vote ≥ 2.5), how confident are you that there is adequate evidence to conclude that DME management using intravitreal targeted anti-VEGF treatment improves patient health outcomes compared to DME management without intravitreal targeted anti-VEGF treatment? 1 2 3 4 5 Low Intermediate High Please discuss any patient characteristics, treatment regimens, or other factors that may have important impacts on the degree of patient benefit or harm from these treatments. If the result of Question 3 is at least intermediate (mean vote ≥ 2.5), how confident are you that there is also adequate evidence to determine whether or not there are clinically meaningful differences in health outcomes among the available intravitreal targeted anti-VEGF treatments for the management of DME? 1 2 3 4 5 Low Intermediate High If the result of Question 4 is at least intermediate (mean vote ≥ 2.5), how confident are you that there is adequate evidence to conclude that there are clinically meaningful differences in the health outcomes when comparing the following available intravitreal targeted anti-VEGF treatments? 1 2 3 4 5 Low Intermediate High Ranibizumab vs Pegaptanib Bevacizumab vs Pegaptanib Ranibizumab vs Bevacizumab Please discuss whether your conclusions are based on evidence of: Different benefits with similar harms Similar benefits with different harms Different benefits and different harms How confident are you that the conclusions above are generalizable to: Medicare beneficiaries? Community-based settings? 1 2 3 4 5 Low Intermediate High DISCUSSION QUESTIONS To what extent are the conclusions above generalizable to the management of other forms of diabetic retinal vascular disease beyond DME? Are there significant gaps in the evidence base on the management of diabetic macula edema? What study designs would support the narrowing or closure of these gaps? Download scoresheet from meeting [PDF, 38KB] Roster: Clifford Goodman, PhD CHAIR Senior Vice President The Lewin Group Steve E. Phurrough, MD Vice Chair Chief Operating Officer/Senior Clincal Director Center for Medical Technology Policy Wendolyn S. Gozansky, MD, MPH Investigator, Institute for Health Research Physician, Continuing Care Department Kaiser Permanente Colorado Willard K. Harms, MD, MBA Medical Policy and Adjudication Blue Cross Blue Shield of Illinois Peter Heseltine, MD Professor of Clinical Medicine University of California, Irvine Susan A. Levine, DVM, MS, PhD Vice President Technology Assessment/Editor-in Chief Hayes, Inc. Pamela R. Massey, Pt, MS Physical Therapist Robert McDonough, MD, JD Head Clinical Policy Research & Development Medical Policy and Program Administration National Medical Services Aetna, Inc. Prabashni Reddy, PharmD Director Center for Drug Policy Partners Healthcare Art Sedrakyan, MD, PhD Associate Professor & Director Patient Centered Comparative Outcomes Research Program Weill Cornell Medical School Robert L. Steinbrook, MD Professor Adjunct of Internal Medicine Yale University School of Medicine Industry Representative Robert W. Dubois, MD, PhD Chief Science Officer National Pharmaceutical Council Guest Panel Member James E. Puklin, MD, FACS Professor of Ophthalmology Department of Ophthalmology Kresge Eye Institute Wayne State University School of Medicine Invited Guest Speaker Robert N. Frank, MD The Robert S. Jampel, MD, PhD Professor of Ophthalmology Professor of Anatomy & Cell Biology Wayne State University School of Medicine Kresge Eye Institute CMS Liaison James Rollins, MD Director Division of Items and Devices Coverage and Analysis Group Executive Secretary Maria Ellis Coverage and Analysis Group Speakers List: Medicare Evidence Development & Coverage Advisory Committee March 21, 2012 SPEAKER LIST *7 MINUTES PER SPEAKER* Jason S. Ehrlich, MD, PhD , Associate Medical Director for Ophthalmology, Genentech, Inc. Helen D. Nickerson, PhD , Senior Scientific Program Manager, JDRF - No Powerpoint Presentation Victor Gonzalez, MD, Representing : The American Diabetes Association Trex Topping, MD , Chair, Health Policy Committee, American Academy of Ophthalmology, Representing: American Academy of Ophthalmology John Thompson, MD , President, American Society of Retina Specialists, Representing: American Academy of Ophthalmology Neil M. Bressler, MD , Chief of Retina Services, Wilmer Eye Institute, Johns Hopkins Hospital, Representing: American Academy of Ophthalmology