Chimeric Antigen Receptor (CAR) T-Cell Therapy and Patient Reported Outcomes

On May 16, 2018, the Centers for Medicare & Medicaid Services (CMS) opened a national coverage determination (NCD) analysis on Chimeric Antigen Receptor (CAR) T-cell therapy for Medicare beneficiaries with advanced cancer. As part of this NCD analysis, the Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) will review the evidence specific to patient reported outcomes (PROs). We are seeking recommendations from the MEDCAC panel regarding how existing PRO assessment tools should be incorporated into future clinical studies, including future clinical studies on CAR T-cell therapy. The MEDCAC will focus on specific PRO assessment tools and important characteristics of a PRO assessment tool.

The MEDCAC panel will assess whether scientific evidence supports a specific number of outcome assessments, study design characteristics, study duration, and suitable controls for applying PROs to health outcomes research. This meeting will also explore the challenges regarding the validity, reliability, and generalizability of existing PRO assessments. MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

June 14, 2018

July 31, 2018

August 16, 2018

August 23, 2018

December 12, 2018

• Webcast of meeting **(registration is not required to view the webcast)**
• Foreign Visitors
• Visitor Screening Procedures
• Registration
• Information about visiting CMS
• Driving directions to CMS
• Recommended hotels

Joseph Ross, MD, MHS, Acting Committee Chair
Aloysius Cuyjet, MD, MPH, Committee Vice-Chair
Tamara Syrek Jensen, JD, Director, Coverage and Analysis Group
Maria Ellis, Executive Secretary

7:30 – 8:00 AM

8:00 – 8:15 AM

8:15 - 8:30 AM

8:30 – 8:45 AM

8:45 – 9:00 AM

9:00 – 9:30 AM

9:30 – 10:10 AM 

10:10 – 10:40 AM

10:40 – 10:55 AM

10:55 – 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 PM

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 

Download scoresheet.

On May 16, 2018, the Centers for Medicare & Medicaid Services (CMS) opened a national coverage determination (NCD) analysis on Chimeric Antigen Receptor (CAR) T-cell therapy for Medicare beneficiaries with advanced cancer. As part of this NCD analysis, the Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) will review the evidence specific to patient reported outcomes (PROs). We are seeking recommendations from the MEDCAC panel regarding how existing PRO assessment tools should be incorporated into future clinical studies, including future clinical studies on CAR T-cell therapy. The MEDCAC will focus on specific PRO assessment tools and important characteristics of a PRO assessment tool.

The MEDCAC panel will assess whether scientific evidence supports a specific number of outcome assessments, study design characteristics, study duration, and suitable controls for applying PROs to health outcomes research. This meeting will also explore the challenges regarding the validity, reliability, and generalizability of existing PRO assessments. MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

1. How confident are you that each of the following PRO assessments are valid and generalizable to the Medicare population?

1. Patient-Reported Outcomes-Common Terminology Criteria for Adverse Events (PRO-CTCAE);
2. MD Anderson Symptom Inventory (MDASI);
3. European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC—QLQ-C30 (core questionnaire)
4. University of Washington Quality of Life (UW-QOL);
5. Patient-Reported Outcome Measurement Information System (PROMIS);
6. Electronic Self-Report–Cancer (ESRA-C);
7. Functional Living Index–Cancer (FLIC).

2. Considering all PRO assessments in question 1 with greater than or equal to score 2.5, please vote whether or not those PRO assessments combined have available supporting evidence on each of the following desired characteristics.

Discussion Questions

• Are there PRO assessments other than those listed in question 1 that have adequately stated evidence-based criteria and processes?


• Are there additional desired characteristics other than those listed in question 2?

3. How confident are you that each of the following assessment intervals are appropriate measurement periods for a valid PRO assessment?

1. Variable event-dependent frequency interval (i.e. upon admission and after discharge)
2. Fixed time-dependent frequency interval (i.e. weekly, monthly, or yearly)

4. How confident are you that a PRO assessment over the course of the following study durations identifies a meaningful durable treatment effect with a valid PRO?

1. 6 months
2. 12 months
3. 24 months

5. How confident are you that PRO assessments can provide meaningful results when studied with each of the following control populations?

1. patient him/herself, before and after intervention
2. usual care versus protocol-driven intervention
3. historical control

Acting Committee Chair

Joseph Ross, MD, MHS
Associate Professor
Medicine and Public Health
Section of General Internal Medicine
Department of Medicine
Yale University School of Medicine

Committee Vice-Chair

Aloysius B. Cuyjet, MD, MPH
Medical Director
HealthCare Partners, IPA

MEDCAC Members

Joseph S. Cheng, MD, MS, FACS, FAANS
Frank H. Mayfield Professor and Chair
Department of Neurosurgery
University of Cincinnati College of Medicine

Diane Civic, PhD, MPH, MSW
Medical Policy Analyst Sr.
Office of Medical Policy and
Technology Assessment
Anthem, Inc.

Naftali Z. Frankel, MS
Patient and Consumer Advocate

Melissa M. Garrido, PhD, BS
Research Associate Professor
Department of Health Law, Policy & Management
Boston University School of Public Health
Research Health Science Specialist
Partnered Evidence-Based Policy Resource Center (PEPReC)
Boston VA Healthcare System

Thomas F. Goss, PharmD
Senior Vice President
Boston Healthcare Associates, Inc.

Thomas James III, MD, FACP, FAAP
Senior Medical Director, MM&Q
Highmark, Inc.

Joel Lamon, MD, FACP
Medical Oncologist/Hematologist
California Cancer Associates for Research and Excellence, Inc.

Carla Perissinotto, MD, MPH
Associate Professor of Medicine
School of Medicine
University of California, San Francisco

Industry Representative

Shamiram Feinglass, MD, MBA
Vice President
Global Government and Medical Affairs
Danaher Corporation

Guest Panel Members

Stephen Gottschalk, MD
Member, St. Jude Faculty
Chair, Department of Bone Marrow Transplantation & Cellular Therapy
Endowed Chair in Bone Marrow Transplantation & Cellular Therapy
St. Jude Children's Research Hospital

Doug Olson, MD
CEO
BUHLMANN Diagnostics Corporation

James C. Yang, MD
Senior Investigator
Surgery Branch
National Cancer Institute
Center for Cancer Research

Invited Guest Speakers

Elissa Bantug, MHS
Manager
Communication, Education, and Survivorship Johns Hopkins Breast Cancer Program
Johns Hopkins University

Ethan Basch, MD, MSc
Professor
Health Policy and Management
Director
Cancer Outcomes Research Program
Professor
Hematology and Oncology
School of Medicine
UNC Lineberger Comprehensive Cancer Center
University of North Carolina at Chapel Hill

Ilia Ferrusi, PhD
Associate Director
HEOR (CAR-T Therapy)
US Oncology
Novartis Pharmaceuticals Corporation

William Go, MD, PhD
Vice President
Clinical Development
Kite, a Gilead Company

Paul Kluetz, MD
Associate Director for Clinical Science
Office of Hematology and Oncology Products
Food and Drug Administration
Center for Drug Evaluation and Research
Office of Oncology Drug Products

Claire Snyder, PhD
Professor of Medicine
Division of General Internal Medicine
Program Director
BLOCS: Building Lifestyle, Outcomes, & Care Services Research in Cancer
Johns Hopkins School of Medicine

CMS Liaison

Tamara Syrek Jensen, JD
Director
Coverage and Analysis Group

Executive Secretary

Maria Ellis
Coverage and Analysis Group

• Kathryn E. Flynn, PhD, Associate Professor of Medicine, Senior Scientific Director for Patient-Reported Outcomes CIBMTR®, Milwaukee Campus, Medical College of Wisconsin – Representing:  Center for International Blood and Marrow Transplant Research (CIBMTR)
  
  
• Karen Chung, PharmD, MS, Senior Director, Health Economics and Outcomes Research, Juno Therapeutics, a Celgene Company
  
  
• Surbhi Sidana, MD, Assistant Professor of Medicine and Oncology, Fellow, Advanced Hematology, Mayo Clinic, Rochester, MN
  
  
• Cori Abikoff, MD, Medical Director CAR-T, Novartis Pharmaceuticals Corporation
  
• Merav Bar, MD, Assistant Member, Fred Hutchinson Cancer Research Center
  
  
• Heather Jim, PhD, Associate Member, Department of Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, FL
  
  
• Gunjan L. Shah, MD, MS, Hematologic Oncologist, Adult Bone Marrow Transplant Service, Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center – Representing: American Society for Blood and Marrow Transplantation

Screening procedures will require visitors to empty pockets, remove belts, hats, outerwear jackets (not suit jackets nor sports coats), and large pieces of jewelry and place in provided bins for x-ray screening. Small and loose items (e.g., purses, laptop computers, cell phones) will be placed in bins and larger items (e.g. computer bags) may be placed directly onto the conveyer. All items being carried into the building will require x-ray screening.

After all items are placed on the conveyer, visitors will proceed through the walk-through metal detector. If the detector alarms, a security guard will use a wand to clear the alarm. If that clears the alarm, screening is complete and the visitor may enter the facility. In the event that the wand does not clear the alarm, the security guard will physically check the area of the body where the alarm is located. Anytime there is an alarm over a sensitive area of a person's body, the officer will use the back of his/her hands to clear the area. If footwear alarms, then footwear will need to be removed and placed in a bin for x-ray screening. Disabled visitors using a mobility aid (e.g. wheel chair, motorized scooter) that cannot pass through the walk-through metal detector will bypass the device and undergo screening by the officer using a wand and/or pat down.

Foreign nationals are not authorized unfettered access to CMS facilities. All foreign nationals visiting any CMS facility are required to have prior approval and are assigned a “Hosting Official” who will be responsible for the visit. The request must be submitted no later than 13 business days prior to the scheduled visit, as processing and approval times can be as much as 10 days. Please contact Ruth McKesson via email at Ruth.McKesson@cms.hhs.gov to submit your request by 5:00 pm EDT on Friday, August 3, 2018.