Health Outcomes in Heart Failure Treatment Technology Studies

On March 22, 2017, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes in studies for heart failure treatment technologies should be of interest to CMS.

Given the increased focus on the need of patients for new and innovative medical products, medical technologies are receiving market authorization based on less long-term data with greater reliance upon intermediate and surrogate outcomes. As a result, assessments of medical technologies are being made with more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries. The MEDCAC panel will examine the growing challenges associated with the decreased level of evidence generated prior to market authorization of new and innovative technology. By voting on specific questions, and by their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of heart failure treatment technologies and appropriate follow-up duration to ensure transparency of National Coverage Analyses (NCA) and requirements under Coverage with Evidence Development (CED). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

January 18, 2017

January 23, 2017

March 16, 2017

March 21, 2017

March 27, 2017

May 25, 2017

• Webcast of meeting
• Registration
• Information about visiting CMS
• Driving directions to CMS
• Recommended hotels

Rita Redberg, MD, Committee Chair
Alan Hirsch, MD, Acting Committee Vice Chair
Joseph Chin, MD, Deputy Director, Coverage and Analysis Group
Maria Ellis, Executive Secretary

7:30 - 8:00 AM   

8:00 - 8:15 AM

8:15 - 8:35 AM

8:35 - 9:05 AM

9:05 - 9:35 AM

9:35 - 10:05 AM

10:05 - 10:35 AM

10:35 - 10:45 AM

10:45 - 11:45 AM

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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.

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11:45 - 12:00 AM

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

12:00 - 1:00 PM

1:00 - 2:00 PM

2:00 - 3:00 PM

3:00 - 4:00 PM

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

4:00 - 4:20 PM

4:20 - 4:30 PM

On March 22, 2017, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes in studies for heart failure treatment technologies should be of interest to CMS.

Given the increased focus on the need of patients for new and innovative medical products, medical technologies are receiving market authorization based on less long-term data with greater reliance upon intermediate and surrogate outcomes. As a result, assessments of medical technologies are being made with more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries.

The MEDCAC panel will examine the growing challenges associated with the decreased level of evidence generated prior to market authorization of new and innovative technology. By voting on specific questions, and by their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of heart failure treatment technologies and appropriate follow-up duration to ensure transparency of National Coverage Analyses (NCA) and requirements under Coverage with Evidence Development (CED). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

For each voting question, please use the following scale identifying your level of confidence - with a score of 1 being low or no confidence and 5 representing high confidence.

1. * How confident are you that the following are standalone, meaningful primary health outcomes in research studies of heart failure treatment technologies:
   1. Heart failure hospitalization;
   2. Heart failure hospitalization or heart failure hospitalization equivalent events (i.e., outpatient IV therapy for heart failure);
   3. Total Hospitalizations?

Discussion:

• For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post-heart failure intervention for assessing this outcome;
• Please discuss important considerations when assessing the merits of composite outcomes in research studies of heart failure treatment technologies which include the combination of mortality, heart failure hospitalization, or heart failure hospitalization equivalent events.

2. How confident are you that surrogate and intermediate endpoints are predictive of standalone, meaningful primary health outcomes (e.g., reduction in mitral regurgitation, cardiac remodeling, ejection fraction, or biomarkers) in clinical research studies of heart failure treatment technologies for:

   1. Heart failure with preserved ejection fraction;
   2. Heart failure secondary to mitral regurgitation where the focus of therapy is mitral valve repair/ replacement;
   3. Heart failure with reduced ejection fraction (e.g., cardiac remodeling, ejection fraction)?

Discussion:

• If greater than or equal to intermediate confidence (≥ 2.5), please identify the specific surrogate or intermediate endpoints and associated disease or therapy which you believe are sufficiently predictive of meaningful health outcomes.
• Please discuss how these intermediate and surrogate endpoints meaningfully contribute towards the evidence base for heart failure treatment technologies.
• Please discuss important factors to consider when assessing the utility of surrogate and intermediate endpoints.

3. How confident are you that quality of life measures [e.g., Kansas City Cardiomyopathy Questionnaire (KCCQ), Minnesota Living With Heart Failure Questionnaire (MLWHFQ):

   1. Are adequate measures which reflect the patient experience;
   2. Should be included as the standalone, meaningful primary health outcomes in research studies;
   3. Should be included as a composite standalone, meaningful primary health outcomes in research studies?

4. How confident are you that functional assessments [e.g., 6 min walk test (6MWT), VO2max, ventilator threshold]:

   1. Are adequate measures which reflect the patient experience;
   2. Should be included as the standalone, meaningful primary health outcomes in research studies;
   3. Should be included as a composite standalone, meaningful primary health outcomes in research studies?

Discussion:

• Please discuss whether additional patient-reported measurement [e.g., Short Form-36 (SF-36), EuroQol five dimensions questionnaire (EQ5D)] should be considered to capture burdens associated with the heart failure therapy under study.
• Please discuss the appropriate length of follow-up post-heart failure intervention for assessing patient-reported measurements.
• For some studies of heart failure treatment technologies it may not be practical for patients to be blinded. Please discuss the impact of unblinded study participants on patient-reported measurements and functional assessments.
• Please discuss how to best consider the impact of adverse events associated with heart failure technologies while balancing the potential for improvements to meaningful health outcomes.
• Please discuss how to balance the benefits and harms of therapies which may improve near-term patient-reported health outcome assessments or clinical measurements (e.g., 6 MWT or symptoms) but may decrease length of life.

Additional Discussion Topics:

• Please discuss health outcomes of interest and appropriate follow-up duration in studies of technologies designed for diagnosis of acute decompensation of heart failure.
• With the health outcomes and information that we have discussed today, how confident are you that there will there be enough accurate information provided to patients for them to make informed decisions?
• Please discuss how studies can be designed to accurately capture patient preferences and how their preferences can best be considered and operationalized once the study has concluded.

* CMS recognizes the importance of mortality as a meaningful primary health outcome of interest in research studies. We are seeking input on what additional outcomes should be considered.