Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review

摘要: Patients and their families have become more active in healthcare systems research. The value of patient involvement is particularly relevant the area rare diseases, where patients face delayed diagnoses limited access to effective therapies due high level uncertainty market approval reimbursement decisions. It has been suggested that may help reduce some these uncertainties. This review explored existing proposed roles for patients, families, organizations at each stage lifecycle diseases (i.e., orphan drug lifecycle). A scoping was conducted using methods outlined by Arksey O’Malley. To validate findings from literature identify any additional opportunities were missed, a consultative webinar with members Patient Caregiver Liaison Group Canadian research network. Existing involving reported throughout fell into 12 themes: outside clinical trials; outcomes measures; registries biorepositories; education; advocacy awareness; conferences workshops; care support; organization development; regulatory decision-making; decision-making. not described sufficient detail allow be assessed. Additionally, no information on impact within specific found. Based feedback documentation Canada poor. Opportunities patient, family, exist lifecycle. However, based found, it possible determine which would most stage.