Phase 1 Activities (Pre-Gene Therapy)
Initial efforts of the PRRWG focused on developing recommendations for assessing and optimizing readiness for the gene therapy process. The recommendations were established based on scientific evidence, expert clinical guidance, review of strategic planning documents, and consultation with CureSCi subcommittees and working groups.
The goals of the Pre-Gene Therapy Patient Readiness and Resilience Assessment were to:
- Gather information about a patient's understanding of and perceived readiness for a gene therapy clinical trial.
- Encourage open dialogue regarding any concerns about gene therapy.
- Provide a conceptualization of psychosocial factors likely to influence participation in a gene therapy clinical trial and affect relevant outcomes.
- Identify patient strengths that can be leveraged to promote psychosocial well-being before, during, and after a gene therapy clinical trial.
- Identify psychosocial risks to be considered and addressed through tailored education, psychosocial support, and community resources.
Phase 2 Activities (During and Post-Gene Therapy)
Building on the earlier recommendations, during Phase 2, the PRRWG focused on developing recommendations to guide assessing, monitoring, and supporting psychosocial health during and after gene therapy.
PRRWG Members:
- Steven Hardy, PhD-Children’s National Hospital (Chair)
- Jerlym Porter, PhD, MPH-St. Jude Children’s Research Hospital (Chair)
- Cecelia Valrie, PhD-Virginia Commonwealth University (Chair)
- Ugonna Anyadike-CureSCi Community Input Panel Representative
- Heather Avant-CureSCi Community Input Panel Representative
- Victoria Coleman-Cowger, PhD-The Emmes Company, LLC
- Katrina Dalton-Virginia Commonwealth University
- Clevetta Drew-Patient Advocate
- Melissa Gordon-Pitts, MSW, LCSW-C-Duke University
- Charles Jonassaint, PhD, MHS-Emory University
- Phillip Okwo, MBA-CureSCi Community Input Panel Representative
- Beth Riley-The Emmes Company, LLC
- Juan Salomon, MS, PMP-National Heart, Lung, and Blood Institute
- Beth Thompson, PsyD-Emory University
- Teonna Woolford-Sickle Cell Reproductive Health Education Directive (SC RED)
Published Resources:
- Pre-Gene Therapy Assessment of Patient Readiness and Resilience: Best Practices and Recommendations for Implementation in Sickle Cell Disease (White Paper)
- Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement (Article in JAMA Network Open)
- Guidelines for Assessing, Monitoring, and Supporting Psychosocial Health During and After Gene Therapy for Sickle Cell Disease (White Paper)