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Center for International Blood and Marrow Transplant Research

CGT Access Model for Sickle Cell Disease

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CIBMTR PRO Data Collection Protocol for Sickle Cell Disease

CIBMTR is collecting patient-reported outcome (PRO) data from recipients of cellular and gene therapies with sickle cell disease to learn about and improve patient quality of life. By participating in the PRO Data Collection Protocol, participants can provide data to help researchers understand long-term impacts of cellular and gene therapies.

The CGT Access Model for Sickle Cell Disease (SCD)

The Cell and Gene Therapy (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. This model aims to increase Medicaid beneficiaries’ access to innovative gene therapy treatments, improve their health outcomes, and reduce health care costs and burden to state Medicaid programs.

On this page:

 

What are Patient Reported Outcomes (PROs)?

A PRO is any report on the status of a patient’s health condition that comes directly from the patient, without interpretation by a doctor or anyone else. 

Here is an example of the type of question a patient may be asked:

In the last 7 days, how OFTEN did you have a HEADACHE?
1-    Never
2-    Rarely
3-    Occasionally
4-    Frequently
5-    Almost constantly

This unique type of data has been used to improve engagement and facilitate patient-physician discussion among cancer patients. PROs add value to understanding survival and quality of life outcomes of cellular therapies.

 

How has PRO data been used at CIBMTR so far?

Click this link for summaries of the studies that have used PRO data.

 

How does CIBMTR collect PRO data?

CIBMTR asks participants in the CGT Access Model for SCD PRO Data Collection Protocol to answer surveys electronically or on a paper form. The surveys typically take 15–25-minutes to complete. 

Surveys are sent at the following timepoints: 

  • Pre-infusion
  • Day 30
  • Day 100
  • Day 180
  • Year 1
  • Annually

The surveys include questions about the below topics: 

TOPIC ADULT PEDIATRIC PARENT / GUARDIAN
Anxiety X X  
Depression X X  
Pain Interference X X  
Fatigue X X  
Cognitive Function X X  
Sleep Disturbance X X  
Sickle Cell Disease Pain Episodes X X  
Global Health X X  
Social Roles and Activities X    
Physical Function X    
Mobility   X  
Peer Relationships   X  
Family Relationships   X  
Financial Well-Being X   X
Demographic Information X   X

Participants are free to skip any question or stop the surveys at any time. For children under the age of 18, a parent or guardian will have to sign a consent form allowing their child to participate and complete a short survey regarding financial and demographic information at most timepoints. 

 

Who is eligible for the CGT Access Model?

  • Gene therapy recipients, age 12 and older at the time of infusion
  • Medicaid and Medicaid-expansion Children’s Health Insurance Program (CHIP) beneficiaries
  • Consented to participation in the CIBMTR Research Database Protocol and have selected “yes” to be contacted by the CIBMTR for future research
  • English or Spanish speaking

Cell and gene therapy treatment centers report and provide contact information of eligible patients to the CIBMTR.

 

PRO Reimbursement 

Enrolled participants will receive a $25 gift card for their first completed survey and a $10 gift card for every survey completed thereafter.

 

Reference Materials

English Speaking Patient Flyers

Spanish Speaking Patient Flyers

 

Questions?