CMS Innovation Center's Cell and Gene Therapy (CGT) Access Model for Sickle Cell Disease (SCD)

The CMS Innovation Center (CMMI) CGT Access Model for sickle cell disease aims to improve the lives of people living with rare and severe diseases by increasing access to potentially transformative treatments. The multi-year model for states and manufacturers to test whether a CMS-led approach to developing and administering outcomes-based agreements (OBAs) for cell and gene therapies increases Medicaid beneficiaries’ access to innovative treatment, improves their health outcomes, and reduces health care costs and burden to state Medicaid programs.

The initial focus of the model is on access to gene therapy treatments for people living with sickle cell disease, a genetic blood disorder. To date, CMS has negotiated the key terms of an outcomes-based agreement (OBA) with the two manufacturers of gene therapies for sickle cell disease (SCD), Vertex Pharmaceuticals, Incorporated for CASGEVY™ and/or Genetix Biotherapeutics (formerly bluebird bio, Inc.) for LYFGENIA™.

As part of the model, CIBMTR was approved to implement an Investigational Review Board-approved study plan which includes collection of clinical data and patient-reported outcomes data for people with Medicaid who receive gene therapies for SCD.

On this page:

• Study Objectives
• Eligibility
• Protocols and Consents
• Patient-Reported Outcomes
• CIBMTR Center Requirements
• Study Contacts
• Reference Materials

Open for Patient Enrollment: January 2025 - present

Study Objectives

• To support the CMS CGT Access Model patient outcomes data requirements through use of CIBMTR’s data infrastructure, including completeness and validation methods, data source linking capabilities, and data protection, and governance;
• To develop a CMS CGT Access Model Study Plan to describe project-specific data extracts that support statistical analytics along with a validated, repeatable, protected data exchange process; and
• To establish standard CMS CGT Access Model PRO measures and to expand PRO electronic data collection and follow-up techniques for patients with SCD.

Eligibilitiy

Center:

• Existing or eligible CIBMTR Network Center
• Center uses or plans to use gene therapy treatments including Vertex Pharmaceuticals, Incorporated for CASGEVY™ and/or Genetix Biotherapeutics (formerly bluebird bio, Inc.) for LYFGENIA™ to treat sickle cell disease

Patient:

• Inclusion
  • Sickle cell disease gene therapy recipients, age 12 and older at the time of infusion
  • Medicaid and Medicaid-expansion Children’s Health Insurance Program (CHIP) beneficiaries
  • Sickle cell disease gene therapy recipients who consent to participation in the CIBMTR Research Database Protocol
  • PRO-specific eligibility includes:
    • Sickle cell disease patient aged 12 and older, or any parent/guardian who has a child aged 12 or older, who has received or will receive a gene therapy infusion
    • Speaks and/or writes in English and/or Spanish
    • Has consented to participation in the CIBMTR Research Database Protocol and has agreed to be contracted by the CIBMTR for future patient-reported outcomes (PRO) research
• Exclusion
  • Sickle cell disease gene therapy recipients not receiving Medicaid or not Medicaid-expansion CHIP beneficiaries

CIBMTR Center Requirements

If a study center plans to use a gene therapy product, including Vertex Pharmaceuticals, Incorporated for CASGEVY™ and/or Genetix Biotherapeutics (formerly bluebird bio, Inc.) for LYFGENIA™, the center will be expected to complete the following actions:

1. Notify CIBMTR of plans and/or timeline for use
2. Obtain Patient Consent for CIBMTR Research Participation
   • Approach applicable participating SCD patients to request their consent to participate in the CIBMTR Research Database Protocol, under which this study is governed, and to allow submission of their data to CIBMTR in accordance with the CGT Access Model study requirements. Inform patients that to participate in the paid PRO portion of this study, they must also consent to future contact by the CIBMTR. 
3. Inform Patients and/or Parent/Guardian of PRO Participation and Reimbursement
   • Notify applicable participating SCD patients and/or parent/guardian that CIBMTR will contact them separately to obtain consent for participation in patient-reported outcomes (PRO) data collection for this study and provide each patient and/or parent/guardian with a study flyer at the time of consent to the Research Database. 
     • To be approached by the CIBMTR for enrollment in the PRO section of this study, participants must agree to be contacted by CIBMTR for future research on the CIBMTR Research Database consent form.
   • Patients will receive a $25 gift card for completing the initial PRO survey prior to receiving their gene therapy infusion, and a $10 gift card for each subsequent survey completed during the study period.
4. Submit CIBMTR Comprehensive Report Form (CRF) level data for all applicable SCD gene therapy recipients for the duration of the study.
   • Upon enrollment of a center’s first applicable participating SCD patient, the center will receive a one-time participation incentive of $1,000. Additionally, the center will receive form reimbursement in accordance with the CIBMTR fee schedule.

For further information and reporting directions, CIBMTR centers participating in the study should refer to the CIBMTR Portal. 

Study Contacts

• For PRO-specific questions regarding this study, contact: CGTmodel@mcw.edu  
• All other questions, CIBMTR Centers should submit a ticket via CIBMTR Center Support > CIBMTR Corporate Studies, Registries, and Trials > CMS CGT Access Model for SCD. Learn more about CIBMTR Center Support.
• For Medicaid claims questions, contact: the Participating State Medicaid Agency, CGTModel@cms.hhs.gov

Reference Materials

• Cell and Gene Therapy Access Model
• Improved Access to Gene Therapy for Sickle Cell Disease: Maya’s Care Journey
• Study Synopsis

English Speaking Patient Flyers

• Informational Flyer for Adult Patients
• Informational Flyer for Pediatric Patients
• Consent to share health info brochure

Spanish Speaking Patient Flyers

• Informational Flyer for Adult Patients
• Informational Flyer for Pediatric Patients
• Consent to share health brochure