Medicare Coverage with Evidence Development (CED) Studies

The US Centers for Medicare & Medicaid Services (CMS) expanded coverage to hematopoietic cell transplantation (HCT) for myelodysplastic syndromes (MDS), sickle cell disease, myelofibrosis, and multiple myeloma under CED studies. This means transplants for these indications need to take place within a CMS-approved clinical study that meets federal guidelines.

We are facilitating national clinical studies for each indication that you can enroll your patients in to meet the CED requirements.

In March 2024, CMS released its National Coverage Determination for allogeneic HCT for patients with MDS. The CED study CIBMTR performed generated important evidence supporting CMS’ decision, which represents a substantial improvement in payor policy and an important increase in access to therapy for CMS patients. Read CIBMTR’s formal news release for more information.

Current Status of CMS CED Studies

MDS CED (10-CMSMDS) - accrual closed
Multiple Myeloma CED (17-CMS-MM) - open for enrollment
Myelofibrosis CED (16-CMS-MF) - open for enrollment
Sickle Cell Disease CED (17-CMS-SCD) - open for enrollment
Sickle Cell Disease CED (BMT CTN 1503) - accrual closed

HCT for MDS Medicare Study (CMS) - CLOSED

Patients with Medicare will be eligible for claims coverage from CMS if they participate in this study. View clinical, billing, and reimbursement information below.

Study Title: Assessment of Allogeneic Hematopoietic Stem Cell Transplantation in Medicare Beneficiaries with Myelodysplastic Syndrome and Related disorders - Part I. (10-CMSMDS-1)

Clinical trial number: NCT #01166009

This study is a sub-study under the CIBMTR Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries NCT01166009

Opened: December 15, 2010

Closed: March 6, 2024

Study Objectives:

Primary objective: To prospectively examine outcomes of allogeneic HCT in adults at least 65 years of age with MDS to determine whether their outcomes are similar to those in younger patients.
To provide a mechanism to Medicare beneficiaries with MDS 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 CIBMTR.

Participating Transplant Centers are Required to:

Create a CRID in FormsNet following standard CIBMTR process.
Complete Form 2554 (CMS Registration Form, an on-demand form) in FormsNet3 for each subject to be enrolled. Select disease option for Myelodysplastic Syndrome.
Complete Form 2400 (Pre-TED). Select CIBMTR CRO Services as the study sponsor, and then select 10-CMSMDS-1 from the list of studies.
Complete Form 2402 indicating the disease is Myelodysplastic Syndrome.
Submit Comprehensive Report Forms (CRF) or Transplant Essential Data Forms (TED) to CIBMTR for all registered study patients.

Patient Eligibility:

Diagnosis of MDS-related disorders, including RA; RARS; RAEB-1; RAEB-2; RCMD; RCMD-RS; isolated 5q– syndrome; CMML; MDS-EB-1; MDS-EB-2; MDS-RS-SLD; MDS-RS-MLD; MDS/MPN; unclassifiable; and therapy-related MDS.
Age at least 65 or age <65 and Medicare beneficiary.
Being treated in a US transplant center.
Provide informed consent to participate in the study.

Exclusion Criteria:

Transformation to AML prior to HCT (Medicare will pay for AML)

Consent Forms:
This study uses the existing research observational database of CIBMTR to collect data requested by CMS as part of its payment policy. Patients enrolled on CIBMTR studies for indications under CED will sign a separate CMS consent form under the Research Database Protocol for participation in a CMS CED-approved study. These patients will 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. Additional protocol or consent forms are not required for this study.

The Research Database protocol, CMS consent form and CIBMTR / NMDP IRB approvals, can be found on the Observational Database Protocol page.

Study Contacts:
For clinical research questions, contact:
Beginning May 22, 2019, all questions from centers must be submitted via Center Support in the ServiceNow application. Please include the study ID# 10-CMSMDS in your ticket.
https://nmdp.service-now.com/csm

Learn more about Center Support.

For Medicare claims questions, contact:
NMDP Payer Policy team, payerpolicy@nmdp.org

Learn more about Medicare Coding and Billing.

Study Reference Materials:
10-CMSMDS Study Plan

Multiple Myeloma Medicare Study

Patients with Medicare are eligible for coverage of HCT for multiple myeloma if they participate in a CED clinical trial.

Study Title: Assessment of Allogeneic Hematopoietic Cell Transplantation in Medicare Beneficiaries With Multiple Myeloma (17-CMS-MM)

Clinical trial number: NCT03127761

This study is a sub-study under the CIBMTR Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries NCT01166009

Opened: July 25, 2017

Study Objectives:

Primary outcome: To compare five-year overall survival probabilities between the allogeneic HCT cohort and an age and disease-risk-matched cohort of autologous HCT patients. Overall survival is calculated for all patients from the date of transplant until death from any cause.
To provide a mechanism to Medicare Beneficiaries with multiple myeloma 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 CIBMTR.

Participating Transplant Centers are required to:

Create a CRID in FormsNet following standard CIBMTR process.
Complete Form 2554 (CMS Registration Form, an on-demand form) in FormsNet3.
1. Select disease option for Multiple Myeloma
Complete Form 2400 (Pre-TED). Select CIBMTR CRO Services as the study sponsor, and then select 17-CMS-MM from the list of studies.
Complete Form 2402 indicating the disease is multiple myeloma.
1. Eligible classifications include:
  1. MM-IgG
  2. MM-IgA
  3. MM-IgD
  4. MM-IgE
  5. MM-IgM (not Waldenstrom)
  6. MM-light chain only
  7. MM-non secretory
Submission of Comprehensive Report Forms will be required for all registered study patients.

Patient Eligibility:

Medicare beneficiaries
Have Stage II or III multiple myeloma
Are eligible to receive an allogeneic HCT from any suitable allogeneic donor (as determined by the transplant center), including umbilical cord blood
Being treated in a US transplant center
Provide informed consent to participate in the study

Consent Forms:
This study uses the existing research observational database of CIBMTR to collect data requested by CMS as part of its payment policy. Patients enrolled on CIBMTR studies for indications under CED will sign a separate CMS consent form under the Research Database Protocol for participation in a CMS CED-approved study. These patients will 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. Additional protocol or consent forms are not required for this study.

The Research Database protocol, CMS consent form, and CIBMTR / NMDP IRB approvals, can be found on the Observational Database Protocol page.

Study Contacts:
For clinical research questions, contact:
Beginning May 22, 2019, all questions from centers must be submitted via Center Support in the ServiceNow application. Please include the study ID# 17-CMS-MM in your ticket.
https://nmdp.service-now.com/csm

Learn more about Center Support.

For Medicare claims questions, contact:
NMDP Payer Policy team, payerpolicy@nmdp.org

Learn more about Medicare Coding and Billing.

Study Reference Materials:
17-CMS-MM Study Plan

Myelofibrosis Medicare Study

Patients with Medicare are eligible for coverage of HCT for myelofibrosis if they participate in a CED clinical trial.

Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis (16-CMS-MF)

Clinical trial number: NCT02934477

This study is a sub-study under the CIBMTR Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries NCT01166009

Opened: December 1, 2016

Study Objectives:

Primary objective: To compare the five-year survival probabilities from Dynamic International Prognostic Scoring System (DIPSS) assessment between the two study arms: allogeneic HCT recipients and non-HCT therapies (ruxolitinib / best supportive care) recipients. In addition, to estimate the five-year overall survival probabilities with haploidentical HCT from DIPSS assessment.
To provide a mechanism to Medicare Beneficiaries with myelofibrosis 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 CIBMTR.

Participating Transplant Centers are required to:

Create a CRID in FormsNet following standard CIBMTR process.
Complete Form 2554 (CMS Registration Form, an on-demand form) in FormsNet3.
1. Select disease option for myelofibrosis.
Complete Form 2555 (CMS - MF Eligibility).
Complete Form 2400 (Pre-TED) Select CIBMTR CRO Services as the study sponsor, and then select 16-CMS-MF from the list of studies.
Complete Form 2402 indicating the disease is myelofibrosis.
Submit Comprehensive Report Forms for all myelofibrosis recipients regardless of age at transplant. Myelofibrosis data has high research value, and it is critical to have CRF data for this study.

Patient Eligibility:

Medicare beneficiaries.
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 assessment.
For patients receiving an allogeneic transplant:
- Donors must be a 6/6 HLA-matched related donors, defined by Class I (HLA-A and -B) intermediate resolution or high-resolution DNA-based typing and Class II (HLA-DRBI) at high-resolution DNA-based typing (but not monozygotic twins) OR an 8/8 HLA-A, -B, -C, and -DRB1 at high-resolution DNA-based typing matched unrelated donor. Donors must meet institutional or NMDP selection criteria; there is no age restriction for sibling donors.
- The planned product must not be umbilical cord blood unit(s) or HLA-mismatched adult donors (< 6/6 HLA alleles for related and < 8/8 HLA alleles for unrelated).
If a matched related or unrelated donor cannot be identified, patients may proceed to allogeneic HCT with a suitable haploidentical donor.
The patient does not have MDS/MPN overlap syndrome.
Being treated in a US transplant center.
Provide informed consent to participate in the study.

Consent Forms:
This study uses the existing research observational database of CIBMTR to collect data requested by CMS as part of its payment policy. Patients enrolled on CIBMTR studies for indications under CED will sign a separate CMS consent form under the Research Database Protocol for participation in a CMS CED-approved study. These patients will 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. Additional protocol or consent forms are not required for this study.

The Research Database protocol, consent form, and CIBMTR / NMDP IRB approvals, can be found on the Observational Database Protocol page.

Study Contacts:
For clinical research questions, contact:
Beginning May 22, 2019, all questions from centers must be submitted via Center Support in the ServiceNow application. Please include the study ID# 16-CMS-MF in your ticket.
https://nmdp.service-now.com/csm

Learn more about Center Support.

For Medicare claims questions, contact:
NMDP Payer Policy team, payerpolicy@nmdp.org

Learn more about Medicare Coding and Billing.

Study Reference Materials:
16-CMS-MF Study Plan

Sickle Cell Disease Medicare Study

Patients with Medicare are eligible for coverage of HCT for sickle cell disease (SCD) if they participate in aCED clinical trial.

Study Title: Prospective Assessment of Allogeneic Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease (17-CMS-SCD)

Clinical trial number: NCT01166009

This study is a sub-study under the CIBMTR Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries NCT01166009

Opened: November 28, 2017

Study Objectives:

Primary objective: To prospectively compare survival of patients 15 to 50 years of age with sickle cell disease who receive allogeneic transplantation compared to those receiving standard of care treatments.
To study transplant-related complications and disease-free survival in patients who received an allogeneic transplant.
To provide a mechanism to Medicare Beneficiaries with sickle cell disease 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 CIBMTR.

Participating Transplant Centers are required to:

Create a CRID in FormsNet following standard CIBMTR process.
Complete Form 2554 (CMS Registration Form, an on-demand form) in FormsNet3.
- Select disease option for sickle cell disease.
Complete Form 2558 (CMS - SCD Eligibility).
Complete Form 2400 (Pre-TED). Select CIBMTR CRO Services as the study sponsor. Then select 17-CMS-SCD from the list of studies.
Complete Form 2402 indicating the disease is sickle cell disease.
Submit Comprehensive Report Forms for all registered study patients.

Patient Eligibility:

Sickle cell disease (HB SS, HB S beta thalassemia or HB SC) with severe signs or symptoms indicating poor prognosis and warranting transplantation. These could include one or more of include the following:
- Stroke
- Neurological deficit lasting >24 hours
- Recurrent acute chest syndrome (ACS)
- Recurrent pain crises
- Red blood cell (RBC) transfusions to prevent vaso-occlusive clinical complications (i.e. pain, stroke, or ACS)
- High tricuspid valve regurgitant jet velocity (TRJV).
Donors may be HLA-matched siblings, mismatched siblings, or other relatives (parent, offspring, or another relative mismatched to the recipient at one or more HLA locus), HLA-matched or mismatched unrelated donors. Donors may donate bone marrow, peripheral blood, or umbilical cord blood.
Donors with sickle cell trait are eligible to donate.
Age 15 – 50 years.
Being treated in a US transplant center.
Provide informed consent to participate in the study.

Consent Forms:
This study uses the existing research observational database of CIBMTR to collect data requested by CMS as part of its payment policy. Patients enrolled on CIBMTR studies for indications under CED will sign a separate CMS consent form under the Research Database Protocol for participation in a CMS CED-approved study. These patients will 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. Additional protocol or consent forms are not required for this study.

The Research Database protocol, CMS consent form, and CIBMTR / NMDP IRB approvals, can be found on the Observational Database Protocol page.

Study Contacts:
For clinical research questions, contact:
Beginning May 22, 2019, all questions from centers must be submitted via Center Support in the ServiceNow application. Please include the study ID# 17-CMS-SCD in your ticket.
https://nmdp.service-now.com/csm

Learn more about Center Support.

For Medicare claims questions, contact:
NMDP Payer Policy team, payerpolicy@nmdp.org

Learn more about Medicare Coding and Billing.

Study Reference Materials:
17-CMS-SCD Study Plan

Sickle Cell Disease CED (BMT CTN 1503) - CLOSED

Accrual closed. Please visit the BMT CTN website for more information.