Rare disease ethics: what do patients and doctors really think about personalized trials?

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Group 1 (Parental Caregiver and Patient Participants) * Parental/primary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with a suspected genetic etiology. * Child is under 21 years of age at the time of enrollment. * Child has an expected survival of at least one year following study enrollment. * Patients (age ≤ 25 years) with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic etiology. * Willingness to provide verbal informed consent (or assent, as appropriate) to participate Group 2 (Other Family) * Family member of a Group 1 participant who plays an active role in the child's life or care. * Includes siblings (≥ 13 years of age), grandparents, or other non-primary caregivers directly affected by the child's diagnosis. * Demonstrated familiarity with the child's medical and family experience. * Willingness to provide verbal informed consent (or assent, as appropriate) to participate. Group 3 (Non-Family Stakeholders) * Individuals currently engaged, or recently active, in clinical care, research, advocacy or policy work related to pediatric-onset rare genetic disorders. * May include clinicians (e.g., neurologists, genetic counselors, nurses, child-life specialists, home-health staff), members of patient-advocacy organizations, institutional-review-board (IRB) members, payers, sponsors, funders, or representatives of hospital systems or regulatory agencies. * Willingness to provide verbal informed consent to participate in semi-structured interviews or focus groups Exclusion Criteria: * Limited English proficiency * Unable to complete the survey materials or complete the interviews in English. * Inability or unwillingness of research participant to give verbal informed consent (in English) * Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).