No uniformity in reporting patient experience in MM trials

There is a lack of uniformity in the way patient-reported outcomes (PROs) are collected in clinical trials of multiple myeloma (MM), a recent study has found. Such heterogeneity may interfere with the accurate characterization of patient experience in these trials.

Drawing from FDA databases, the researchers identified 25 trials assessing 20 MM indications, which were then reviewed for their PRO measures, including collection methods used, threshold definitions, instrumentation, and statistical analyses.

Of the included trials, only 17 (68 percent) included PRO data and were included in the analysis. The cumulative study sample included 569 patients, and a total of 40 PRO instruments were used. In 14 trials, ≥2 different instruments were employed. The timing of PRO assessments was likewise heterogeneous across studies and were generally conducted infrequently.

Aside from basic descriptive summaries, statistical methods applied varied greatly across studies. Ten studies performed longitudinal mixed model analyses, nine employed responder analyses, and two used a basic inferential test.

Similarly, considerable among-study differences were observed in terms of the definition of PRO population (ie, intention-to-treat population vs treatment completers), as well as what constitutes the baseline and completion. The clinical threshold to determine the minimal important difference was likewise heterogeneous across studies.

“These differences in PRO analyses within the same disease and therapeutic setting may hinder the ability to effectively capture and interpret patient experience in MM clinical trials, which are valuable information for patients and clinicians,” the researchers said.