Designing a patient-reported outcome requires months of painstaking research and interaction with patients, says Alexandra Quittner, a professor of psychology and pediatrics at the University of Miami, who eight years ago began designing a questionnaire for Genentech and the Cystic Fibrosis Foundation that eventually spanned 18 study cites around the United States.
Focus groups involving interviews with patients, caregivers, and healthcare providers must be conducted, and these take time. "Every phase is difficult. You start by trying to get some representative samples in the appropriate age range that you're targeting," says Quittner, who also consulted with Gilead Sciences on its questionnaire. "You make sure you talk to the kids, their parents, their nurses.
"So you have to have a lot patience and persistence. You have to know the domains [the concepts being measured]; you have to write a series of questions, and you ...
