Ventricular Assist Devices as Destination Therapy

Ventricular assist devices (VADs) are mechanical pumps used to assist a damaged or weakened heart in pumping blood. These devices support a patient’s weakened native heart but do not replace it, unlike heart transplantation. VADs are approved by the Food and Drug Administration (FDA) when used for temporary support until the native heart recovers function or a heart transplant is performed. § 65-15 of the Medicare Coverage Issues Manual (CIM) covers these devices when used in accordance with FDA labeling, including support of circulation after cardiotomy and as a bridge to transplant for patients approved and listed for heart transplantation. Medicare does not presently cover this device as an alternative to medical management in end-stage heart failure (ESHF) patients who are not eligible for a heart transplant.

CMS has received a request to revise and update CIM § 65-15 to expand coverage for use of these devices as destination (permanent) therapy for ESHF patients who are not candidates for heart transplantation. Destination therapy means that the VAD will be used by the patient for the remainder of life. Long-term use of this device will require continuous follow-up by a multi-disciplinary medical team and extensive support systems. It will also present patients, caregivers, and families with unique environmental challenges and responsibilities. We specifically request evidence to assist us in developing parameters to ensure the most positive outcomes possible.

This request is based on evidence presented in The Randomized Evaluation of Mechanical Assistance for the Treatment of Congestive Heart Failure (REMATCH). The findings of that trial are currently under review by the FDA as the result of a manufacturer’s request to expand labeling to include use of the device as destination therapy.

Harold C. Sox, MD, Chairperson
Steve E. Phurrough, CMS Liaison
Kimberly A. Long, Executive Secretary

7:30 - 8:00 AM

8:00 - 8:15 AM

8:15 - 8:35 AM

8:35 - 9:35 AM

9:35 - 10:15 AM

10:15 - 10:30 AM

10:30 - 12:00 PM

12:00 1:00 PM

1:00 - 1:20 PM

1:20 - 3:15 PM

3:15 - 3:30 PM

3:30 PM

The Medicare Coverage Advisory Committee met on March 12, 2003, to hear and discuss evidence and testimony regarding the use of a ventricular assist device as destination therapy in end-stage heart failure patients who are not eligible for a heart transplant. The meeting began with the introduction of the Committee, a reading of the conflict of interest statement, and opening remarks by the CMS Liaison.

Perry Bridger described the impact of congestive heart failure on the Medicare population, presented the panel with the history and time line of Medicare coverage for VAD devices, gave an overview of the current coverage request, and presented the panel with the following voting question:

Is the quality of the evidence adequate to draw conclusions about the net health outcomes in Medicare beneficiaries meeting the REMATCH trial criteria who undergo LVAD implantation?

Mr. Bridger also informed the panel of the following statements concerning the REMATCH trial, and related discussion questions that CMS would like the panel’s comments on:

One: REMATCH showed increased survival in device recipients, but the survival advantage diminished over time and was associated with severe complications and increased hospitalization. Do the demonstrated extension of life and the limited improvement in the quality of life justify the risks of LVAD implantation?

Two: One REMATCH inclusion criterion is that candidates for LVAD implantation for destination therapy could not be a heart transplant candidate. Should the evaluation to determine transplant candidacy be performed only by a heart transplant center that has been approved for Medicare reimbursement?

Three: Initially, should treatment centers using LVAD meet specific facility and personnel requirements in order to provide the patient with an optimal chance of successful outcomes following LVAD implantation?

Four: REMATCH results are based on LVAD implantation in 68 patients. Complete, timely, and accurate LVAD implant and outcomes data for destination therapy patients is critical for future Medicare coverage review and policy refinements. Should data reporting be required as a condition for Medicare reimbursement?

Five: There have been improvements in both LVAD design and medicine management of end-stage heart failure patients since the start of the REMATCH trial. Have these improvements affected the applicability of the REMATCH results?

Requestor’s Presentation.

Dr. Eric Rose, Dr. James Long, and Dr. Leslie Miller addressed the panel. Their presentations included summaries of the trial process and results; videos showing some recipients of an LVAD implant; a history of the development of the LVAD device and changes that have been made to the device since the REMATCH trial commenced. They also discussed the issues of infection, adverse events, survival, nutrition, and patient management for LVAD recipients.

The requestors stated that REMATCH was a well designed trial with high quality evidence that can be used to answer the voting question and that the net health outcome of device patients is substantially more effective than medically managed patients. They also commented positively on the five discussion questions posed by CMS to the panel.

Scheduled Public Comments.

The panel heard from five speakers who had requested the opportunity to address the panel. These speakers included representatives of the International Society for Heart and Lung Transplantation, American College of Cardiology, the Society of Thoracic Surgeons and American Association for Thoracic Surgery, the American Society of Transplant Surgeons, and the American Heart Association.

Collectively, these speakers strongly endorsed the use of LVADs for certain patients ineligible for cardiac transplantation, but stressed the importance of ensuring that these patients met REMATCH criteria, and that providers be required to submit outcomes data to a centralized registry.

Open Panel Deliberations.

As the primary reviewer on the MCAC LVAD panel, Dr. Aubry gave his evaluation of the methodology in the REMATCH trial. As a second designated reviewer for the MCAC LVAD panel, Dr. Slaughter described several clinical issues for the panel’s consideration. Following these presentations, the panel asked questions of the REMATCH investigators.

Open Public Comments.

Two members of the public had signed up at the meeting to address the panel. However, when called upon, they both stated that they would defer their comments and contribute their time to the panel deliberations and discussions with the REMATCH investigators.

Open Panel Deliberations and Voting.

The panel conducted extensive discussions, including asking many additional questions of the REMATCH investigators. Following the discussion, it was moved and seconded that the panel change the voting question to read as follows:

"Is the quality of the evidence adequate to draw conclusions about the net health outcomes in Medicare beneficiaries comparable to the patients enrolled in the REMATCH trial who undergo LVAD implantation?"

Prior to taking a vote, Dr. Sox asked each voting member, as well as the consumer and industry representatives, to summarize their conclusions based on the evidence and the panel deliberations. Following these statements, a vote was taken. The results of the vote were six to one in favor of the question.

The panel then discussed the appropriate effect size for the improvement of net health outcomes of LVADs compared to optimal medical management for these patients. A motion was made and seconded that the procedure is effective and falls into the “substantially more effective” category on magnitude of net health outcomes. Following discussion, the panel voted five to two on this motion. The two panelists who cast a negative vote commented that they considered the effect size to fall in the “more effective” category.

Discussion Questions.

Dr. Phurrough asked the panelists, including the consumer, industry representatives, and the non-voting guest panelists, to comment on each of the discussion questions. He noted that the panel’s recommendation on the voting question is just one of many criteria that CMS considers in arriving at a coverage decision, and that all comments about the discussion questions would be in the record of the meeting and would be considered by CMS. After the panelists had made their comments on discussion questions one through four, it was decided that discussion question five had been adequately addressed by earlier comments and further discussion was not needed.

Adjournment.

The meeting adjourned at 3:35 p.m.

Voting Question (After discussion, each panelist will be asked to comment prior to voting)

Is the quality of the evidence adequate to draw conclusions about the net health outcomes in Medicare beneficiaries meeting the Randomized Evaluation of Mechanical Assistance for the Treatment of Heart Failure (REMATCH) trial criteria who undergo LVAD implantation?

Please consider the following in your assessment of the quality of the evidence:

• Are the study endpoints and patient selection criteria appropriate?
• Are the management and extent of complications adequately described?
• Do the follow-up survival data for the REMATCH trial suggest any meaningful difference in patient survival compared to the data at the time the study reached its primary endpoint?

If the quality of the evidence is adequate, does it demonstrate any positive net health outcomes and if so what is the size of the improvement in net health outcomes of LVADs compared to optimal medical management for these patients? (See MCAC document “Recommendations for Evaluating Effectiveness” for categories of effectiveness).

Discussion Questions (Each panelist will be asked to comment on each issue)

REMATCH showed increased survival in device recipients, but the survival advantage diminished over time and was associated with severe complications and increased hospitalization. Does the demonstrated extension of life and limited improvement in the quality of life justify the risks of LVAD implantation?

One REMATCH inclusion criterion was that a candidate for LVAD implantation for destination therapy could not be a heart transplant candidate. Should the evaluation to determine transplant candidacy be performed only by a heart transplant center that has been approved for Medicare reimbursement?

Initially, should there be specific facility (e.g. Medicare approved transplant center only or other transplant center) and personnel requirements (surgeon and team experience) that must be met to provide the patient with an optimal chance of successful LVAD implantation (e.g., adequate pre/post operative care, follow-up care, psychological support for patient/family, and end-of-life care)?

REMATCH results are based on LVAD implantation in 68 patients. Complete, timely, and accurate LVAD implant and outcomes data for destination therapy patients is critical to future Medicare coverage review and policy refinements. Should mandatory data reporting be required as a condition for Medicare reimbursement?

There have been improvements in both LVAD design and medical management of end-stage heart failure patients since the start of the REMATCH trial. Have these improvements affected the applicability of the REMATCH results?

"MCAC Categories of Effectiveness"

• Breakthrough technology: The improvement in health outcomes is so large that the intervention becomes standard of care.

• Substantially more effective (approved 9/25/02): The new intervention improves health outcomes by a substantial margin as compared with established services or medical items.

• More effective: The new intervention improves health outcomes by a significant, albeit small, margin as compared with established services or medical items.

• As effective but with advantages: The intervention has the same effect on health outcomes as established services or medical items but has some advantages (convenience, rapidity of effect, fewer side effects, other advantages) that some patients will prefer.

• As effective and with no advantages: The intervention has the same effect on health outcomes as established alternatives but with no advantages.

• Less effective but with advantages: Although the intervention is less effective than established alternatives (but more effective than doing nothing), it has some advantages (such as convenience, tolerability).

• Less effective and with no advantages: The intervention is less effective than established alternatives (but more effective than doing nothing) and has no significant advantages.

• Not effective: The intervention has no effect or has deleterious effects on health outcomes when compared with "doing nothing," (e.g., treatment with placebo or patient management without the use of a diagnostic test).