At the 26th Annual Conference of the International Society for Quality of Life Research, 2 groups of US Food and Drug Administration (FDA)-affiliated researchers presented abstracts for studies aiming to test the limits of patient-reported outcomes (PROs) as a useful metric in the assessment of the efficacy of certain treatment approaches in cancer. One study investigated whether patient awareness of treatment administration, or unblinding, impacted reporting of PROs,1 and another questioned whether PROs are truly a stable metric for a patient’s deteriorating condition.2
That second study took a closer look at how PROs are currently used (and could be used in the future) in cancer drug development. While these data could be harnessed in the pursuit of regulatory approval for a drug, or as surrogate end points in the evaluation of the effect of a treatment — both seemingly positive uses of the information — the use of PROs as a metric to gauge quality of life may also be susceptible to gaming.
The evaluation of PROs, then, must be standardized — especially because PRO data are increasingly finding their way into pharmaceutical companies’ drug trials and across presentations of pivotal cancer trial results.
“These days, an industry sponsor is putting itself at a disadvantage if it does not have expertise on PROs or include PROs in development programs,” said Ethan Basch, MD, MSc, director of cancer outcomes research at the University of North Carolina-Chapel Hill’s Lineberger Cancer Center, who wasn’t involved in either study. “This is a fast-growing field of increasing emphasis … Many patients have baseline impairments that should be measured by PROs so as not to be misattributed to products.”
The first of the 2 aforementioned abstracts presented at the conference aimed to suss out whether bias is detectable in PROs across open-label trials in oncology compared with other trial designs.1
Part of the impetus for the research was that, as the abstract noted, open-label cancer trials — where researchers and participants are both aware of the parameters of the trial, and treatment assignment is unblinded — are used more often in cancer to bolster research than in other clinical settings. If bias were detectable, it could have a marked impact on health outcomes for cancer patients specifically.
The study enrolled patients with multiple myeloma and sorted them into 2 groups. One group received treatment by way of an open-label design, while the other received the same treatment through a double-blind trial. PROs were assessed twice: at baseline, or before patients were randomly assigned to 1 of these 2 treatment groups, and again after treatment was assigned (but before treatment actually began).