Putting It All Together
What goes into a patient reported outcome?
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...
"With cystic fibrosis, you're measuring improvements in respiratory symptoms after the use of a medication. This would include whether the patient is coughing less, being woken fewer times at night due to coughing, and less tightness in the chest," says Quittner. "But it's hard to get funding to do measurement studies. You need funding or partnering with a drug company or a foundation. It can be expensive. And what if you're questionnaire shows the product makes [symptoms] worse? It's a risk and, you know, pharmaceutical companies are risk-averse."
Stephen Joel Coons designed a patient-reported outcomes questionnaire for a drug maker that hoped to market a smoking-cessation medication, which was in Phase II testing at the time. The effort took nearly eight months but involved only 30 patients, and so it carried the modest price tag of less than $30,000. Citing confidentiality, Coons declined to name the company.
"It was a very structured process. We asked questions and received open-ended responses, which we used to develop a pool of items to measure. We questioned between six and 10 people at a time, and they fed off each other, which is a benefit of doing a focus group. You can do one-on-one interviews, but it's often better to develop your item pool from a group. It's more cost effective in terms of time and money you'll spend."
The gist of the questions involved asking participants about their reactions to using a smoking-cessation product to quit smoking versus going "cold turkey." Coons used their responses to fashion five different domains: social interactions, self-control, sleep, anxiety, and cognitive functions. From there, he took a draft questionnaire and went to still more focus groups to test the instrument for clarity and relevance. The questionnaire was deemed useful, "but the drug never made it to Phase III," he says, "so it was all a sunk cost."