Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

"The following three groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases:~(1) patients and families of children with rare genetic diseases who have received gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy), (2) patients and families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or ultimately did not qualify for a trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met), and (3) healthcare workers who provide care to patients receiving gene therapy (to assess their perspective regarding patient/family needs for gene therapy education and communication)."