Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus...

Area of Science:

• Hematology
• Genetics
• Public Health

Background:

• Sickle cell disease (SCD) is the most prevalent inherited blood disorder globally.
• Gene therapy and genome editing represent a significant advancement in SCD treatment.
• Historic exploitation necessitates careful consideration of readiness for genetic therapies in SCD patients.

Purpose of the Study:

• To address the need for patient readiness resources for gene therapy in sickle cell disease.
• To develop consensus recommendations and practical resources for pre-gene therapy patient readiness assessments.

Main Methods:

• Convened the Patient Readiness and Resilience Working Group, including experts, patients, and caregivers.
• Developed consensus recommendations over two years.
• Created resources to guide the implementation of readiness assessments.

Main Results:

• Identified five key goals for pre-gene therapy patient readiness assessments.
• Recommendations focus on enhancing education delivery and assessing knowledge, motivation, and psychosocial factors.
• Highlighted the importance of assessing patient understanding, interest, motivation, and psychosocial risks and resilience.

Conclusions:

• Patient readiness and psychosocial factors significantly impact individual and collective gene therapy success in SCD.
• Healthcare institutions, industry, payers, policymakers, and clinicians share responsibility for optimizing patient readiness and access.
• Developed resources can guide the implementation of pre-gene therapy readiness and resilience assessments for SCD.