CMS Studies – An Update CIBMTR Data Managers Meeting February 21, 2018 Sue Logan Clinical Research Coordinator CIBMTR Data Operations
Conflict of interest • There are no conflicts of interest to disclose TRAINING & DEVELOPMENT | 2 . 2
Agenda • What are CMS CED studies? • Current status of CIBMTR’s CMS CED studies • Medicare reimbursement for CED studies TRAINING & DEVELOPMENT | 3 . 3
Background
What is CMS? • Centers for Medicare & Medicaid Services (CMS) is a federal agency within the U.S. Department of Health and Human Services (HHS). • Administers many programs including Medicare. TRAINING & DEVELOPMENT | 5 . 5
Objective of all CMS studies • To provide a mechanism to Medicare beneficiaries for claims coverage for allogeneic HCT. • To provide data requested by Medicare under its CED to make payment on claims for HCT using the existing research observational database of the CIBMTR TRAINING & DEVELOPMENT | 6 . 6
What is CED? • Coverage with Evidence Development (CED) is the means by which CMS can provide coverage and encourage clinical studies that will lead to solid evidence for future decision making. • National Coverage Determination (NCD) • Relatively rare mechanism used when: – Safety has been assured – Service has high potential of benefit – Significant barriers to conduct of trials exist TRAINING & DEVELOPMENT | 7 . 7
Why is CED needed? • Transplants in the older population continues to increase for a variety of diseases • BMT community continued to work with CMS to consider other CED/NCD • Decisions have been made for: – MDS (Active Dec 2010) – Myelofibrosis (Active Nov 2016) – Multiple Myeloma (Active July 2017) – Sickle Cell (Active Oct 2017) TRAINING & DEVELOPMENT | 8 . 8
Requirements • Centers must designate willingness to participate in CRF submission for CED • A separate participation agreement is required for each study • Participating recipients must sign CMS consent form TRAINING & DEVELOPMENT | 9 . 9
CMS Consent • CMS consent form – Feb 2017 • Patients should be invited to participate in both the CMS CED study and the Research Database protocol. • Patient participation in the CMS CED study is not dependent on their participation in the Research Database Protocol. TRAINING & DEVELOPMENT | 10 . 10
CMS Consent ver 3.0 • Revised consent form was approved by NMDP IRB Jan 2018. • Language was added in Section IV regarding the NIH Certificate of Confidentiality. • Language was added in Section IV regarding submitting health information to scientific databases. • https://www.cibmtr.org/DataManagement/Protoc olConsent/ObservationalData/pages/index.aspx TRAINING & DEVELOPMENT | 11 . 11
Instructions for Transplant Centers That Use the NMDP/Be The Match IRB • Transplant centers that have an IRB Authorization Agreement with the NMDP IRB may begin using the new consent form immediately for enrolling patients on the CMS CED studies. No additional local review or approval is necessary. TRAINING & DEVELOPMENT | 12 . 12
Instructions for Transplant Centers That Use Their Own Local IRB • If transplant centers use their own local IRB to enroll patients on the CMS CED studies, they should submit the revised protocol and new consent form to their IRB as soon as possible. • It may be submitted as an amendment so they don’t have to wait until their local IRB continuing review TRAINING & DEVELOPMENT | 13 . 13
Instructions for Transplant Centers That Use Their Own Local IRB • Centers are allowed to continue using their current consent form while they are obtaining IRB approval of the revised form. • Renewals must be approved before their local IRB expiration date TRAINING & DEVELOPMENT | 14 . 14
Instructions for Transplant Centers That Use Their Own Local IRB • Once IRB approval is received, the IRB approval letter and IRB-approved consent form must be sent to Sue Logan at slogan@nmdp.org. TRAINING & DEVELOPMENT | 15 . 15
Reporting Requirements for all CMS CED studies • CRID assignment form 2804/2814 • Enrollment form 2554 TRAINING & DEVELOPMENT | 16 . 16
Reporting Requirements for all CMS CED studies • Pre-TED form 2400 – Indicate CMS clinical trial 16-CMS-MF • Comprehensive report forms - necessary for study objectives . TRAINING & DEVELOPMENT | 17 . 17
Assessment of Allogeneic Hematopoietic Stem Cell Transplantation in Medicare Beneficiaries with Myelodysplastic Syndrome and Related disorders - Part I. (10-CMSMDS)
10-CMSMDS • Primary Objective : – To prospectively examine outcomes of allogeneic HCT in adults >= 65 years of age with MDS to determine whether their outcomes are similar to those in younger patients. • Secondary Objectives: – To prospectively determine whether there are disease- or patient-related factors that predict outcomes of HCT for MDS and related disorders in patients >= 65 years of age – To prospectively evaluate what transplant characteristics are associated with outcomes of HCT for MDS and related disorders in patients >= 65 years of age TRAINING & DEVELOPMENT | 19 . 19
10-CMSMDS • CMS decision in Summer of 2010 • Opened for enrollment in December 2010 • 134 participating centers • Current Enrollment 3466 – >= 65 y/o Arm = 2181 – <65 Arm = 1285 TRAINING & DEVELOPMENT | 20 . 20
10-CMSMDS • Patients do not have to have Medicare to be enrolled in the study. – 25% of patients with MDS who gave consent to the research database are enrolled – 100% of patients over age 65 who gave consent to the research database are enrolled • CMS consent and F2554 only required if CMS will be paying for the transplant TRAINING & DEVELOPMENT | 21 . 21
10-CMSMDS • As of the January 30th, 2018 release of FormsNet3, centers will be required to complete Comprehensive Report Forms (CRF) for about half of the patients participating in the 10- CMSMDS-1 study. • Further study information is located on the CIBMTR website at the following location: https://www.cibmtr.org/Studies/ClinicalTrials/mct/HCT- MDS/Pages/index.aspx TRAINING & DEVELOPMENT | 22 . 22
BMT CTN 1102 Hypometh vs RIC for MDS • A Multi-Center Biologic Assignment Trial Comparing Reduced Intensity Allogeneic Hematopoietic Cell Transplant to Hypomethylating Therapy or Best Supportive Care in Patients Aged 50-75 with Intermediate-2 and High Risk Myelodysplastic Syndrome • CTN 1102 is also a CMS CED study • Co-enrollment in 10-CMSMDS and CTN 1102 is not allowed TRAINING & DEVELOPMENT | 23 . 23
Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis (16-CMS-MF)
16-CMS-MF HLA-Matched Donor HCT Study • Primary objective: – Compare the five-year survival probabilities from DIPSS assessment between the two study arms: alloHCT recipients and non-HCT therapies (ruxolitinib / best supportive care) recipients. • Secondary objectives: – Compare leukemia-free survival at five years from DIPSS assessment. – Identify patient-, disease-, and HCT-related factors associated with poor HCT outcomes in the alloHCT arm. – Estimate the cumulative incidences of acute and chronic graft- versus-host disease, transplant related mortality, and relapse starting at HCT in the alloHCT arm. TRAINING & DEVELOPMENT | 25 . 25
16-CMS-MF Haploidentical Donor Study • Primary objective: – Estimate the five-year overall survival probabilities with haploidentical HCT from DIPSS assessment. • Secondary objectives: – Estimate five-year leukemia-free survival with haploidentical HCT from DIPSS assessment. – Identify patient-, disease-, and HCT-related factors associated with poor outcomes post haploidentical HCT, starting at HCT. – Estimate the cumulative incidences of acute and chronic GVHD, relapse, and transplant related mortality, starting at HCT. TRAINING & DEVELOPMENT | 26 . 26
16-CMS-MF • CMS decision in October 2016 • 104 participating centers • Opened for enrollment in December 2016 – Target Enrollment = 650 (225 MAC) – Current Enrollment = 52 • Non-HCT Arm = 2400 TRAINING & DEVELOPMENT | 27 . 27
16-CMS-MF • Eligibility Criteria – Primary myelofibrosis, post- essential thrombocythemia myelofibrosis, or post- polycythemia vera myelofibrosis. – Int-2 or high-risk disease as determined by the DIPSS. – Age ≥55 at the time of DIPSS. – For the patients receiving an allogeneic transplant: • 6/6 HLA-matched related donors • 8/8 HLA-matched unrelated donor • Haploidentical donor. TRAINING & DEVELOPMENT | 28 . 28
16-CMS-MF • Eligibility Criteria – Both peripheral blood stem cells and bone marrow grafts are allowed. – All conditioning regimen intensities are allowed – All GVHD prophylaxis regimens are allowed TRAINING & DEVELOPMENT | 29 . 29
16-CMS-MF • Ineligible if – AlloHCT using umbilical cord blood unit(s) – HLA-mismatched adult donors (< 6/6 HLA alleles for related and < 8/8 HLA alleles for unrelated) – Overlap syndromes (e.g., CMML, JMML) – Prior allo HCT TRAINING & DEVELOPMENT | 30 . 30
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