Health Outcomes in Cerebrovascular Disease Treatment Studies

On September 22, 2021, 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 should be of interest to CMS in studies for cerebrovascular disease treatment with a focus on new technologies.

Given the increased emphasis on new and innovative medical products for treating diseases that have few proven therapies, studies of cerebrovascular disease treatment technologies submitted through the Investigational Device Exemption (IDE) pathway have focused on shorter term data with greater reliance upon intermediate and surrogate outcomes. Although such foci may help in answering Food and Drug Administration (FDA) questions on safety and efficacy, they are generally less helpful for CMS whose focus is on health outcomes associated with the technologies. As a result, there are more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical technologies. The MEDCAC panel will examine the growing challenges associated with these gaps in the evidence used to support requests for CMS coverage of new and innovative cerebrovascular disease treatment technologies. By voting on specific questions, and through their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of cerebrovascular disease treatment technologies, appropriate measurement instruments and follow-up durations to help to provide clarity and transparency of IDE analyses and National Coverage Analyses (NCAs). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

Although there is general agreement on the importance of using mortality as an outcome measure in cerebrovascular disease clinical research, there is little or no consensus on which other outcomes measures to include, such as stroke status and recurrence, hospitalization and healthcare resource utilization, clinician-reported patient functioning, as well as patient-reported outcome measures (PROMs). CMS is seeking input on whether, how, and when to include these additional outcomes.

August 9, 2021

September 16, 2021

September 30, 2021

November 2, 2021

Peter Bach, MD, Committee Chair
Joseph Ross, MD, Committee Vice-Chair
Joseph Chin, MD, Deputy Director, Coverage and Analysis Group
Tara Hall, MEDCAC Coordinator

8:00 - 8:15 AM

8:15 - 8:25 AM

8:25 - 8:50 AM

8:50 - 9:15AM

9:15 - 10:05 AM

10:05 - 10:20 AM

10:20 - 11:15 AM

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Public attendees, who have contacted the MEDCAC Coordinator 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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11:15 - 11:45 PM

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

11:45 - 12:45 PM

12:45 - 1:45 PM

1:45 - 2:45 PM

2:45 - 3:00

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 

Download meeting minutes.

Download transcript from meeting.

Download scoresheet

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 each of the following are standalone, meaningful primary health outcomes in research studies of cerebrovascular disease treatment technologies:
   1. Major disabling stroke: defined as stroke in the treated vascular territory that results in a Modified Rankin Scale (mRS) ≥ 3;
   2. Decrease in mRS of ≥ 2 points compared to baseline;
   3. mRS of ≤ 2 or equal to pre-stroke mRS (if the pre-stroke mRS was > 2);
   4. Other kinds of stroke, such as major ipsilateral stroke or morbid stroke.

Discussion:

• For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome;
• For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate cutoff points of mRS and NIHSS for assessing this outcome;
• Please discuss important considerations when using composite outcomes in research studies of cerebrovascular disease treatment technologies, which may include the combination of mortality, stroke, hospitalization/hospitalization equivalent events, and neurologic functional evaluations.


2. How confident are you that each of the following are standalone, meaningful primary health outcomes in research studies of cerebrovascular disease treatment technologies:
   1. Hospitalization length of stay for index procedure;
   2. Number of unscheduled re-admissions that are related to cerebrovascular disease;
   3. Discharge disposition to rehabilitation (home vs. inpatient facility).

   Discussion:

   • For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome (applies to “b.” only);
   • Please discuss important considerations when assessing the merits of composite outcomes in research studies of cerebrovascular disease treatment technologies, which include the combination of mortality, stroke, healthcare resource utilization for index procedure, post-procedure and re-hospitalizations, and neurologic functional evaluation.


3. How confident are you that each of the following functional assessments are standalone, meaningful primary health outcome measures in clinical research studies of cerebrovascular disease treatment technologies:
   1. The Modified Rankin Scale (mRS);
   2. The National Institutes of Health Stroke Scale (NIHSS).

   Discussion:

   • For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate length of follow-up post intervention for assessing this outcome;
   • For each health outcome with greater than or equal to intermediate confidence (≥ 2.5), please discuss the appropriate cutoff points for assessing this outcome;
   • Please discuss important considerations when assessing the merits of composite outcomes in research studies of cerebrovascular disease treatment technologies, which include the combination of mortality, stroke, hospitalization/ hospitalization equivalent events, and neurologic functional evaluation;
   • Are there any other functional assessments (e.g. the Barthel Index (BI), the Fugl-Meyer (FM) Upper and Lower Extremity scales) that we have not discussed whose use you believe would result in important information pertaining to meaningful primary health outcomes in clinical research studies of cerebrovascular disease treatment technologies?


4. How confident are you that using EQ-5D to measure quality of life:
   1. Is adequate measure which reflects the patient experience in the context of cerebrovascular disease studies;
   2. Should be included as standalone, meaningful primary health outcome measure in research studies;
   3. Should be included as part of a composite, meaningful primary health outcome measure in research studies;
   4. Should be included as secondary health outcome measure in research studies.

   Discussion:

   • Please discuss whether additional patient-reported measurement [e.g., Short Form-36 (SF-36), Stroke Impact Scale-16 (SIS-16)] should be considered to capture quality of life and burdens associated with the cerebrovascular disease treatment under study;
   • Please discuss the minimal clinically important differences (MCIDs) for the instruments;
   • Please discuss the appropriate length of follow-up post intervention for assessing patient-reported measurements.