Researchers Show How RWD on Racial Outcomes in MM Can Complement Clinical Trial Data

Real-world findings are adding insight into multiple myeloma outcomes among Black patients, a group typically underrepresented in clinical trials for the disease.

Black patients in the retrospective observational study had more favorable survival compared with White patients. In unadjusted analyses, Black patients had longer median survival during both first line (64.6 months vs 54.5 months) and second line treatment (53.2 months vs 41.7 months). However, these differences lost prominence when analyses adjusted for age, sex, and practice type.

“These findings are consistent with previous analyses suggesting that younger age at diagnosis and different cytogenetic profile may contribute to similar or more favorable survival outcomes in Black patients,” wrote the group.

They continued, “most recently, results from the CoMMpass registry showed superior survival for White patients when compared to Black patients, a difference seemingly driven, to considerable extent, by different patterns of care (with less utilization of triplet therapy and ASCT in Black patients), as also suggested by a recent SEER-based report.”

Of the 5800 patients included in study, 20% were Black, which the researchers say adequately represents disease incidence among the population. According to the authors, race was self-reported, which has been shown to increase accuracy of race reporting compared with clinician-reported race. In comparison, among new drug applications for multiple myeloma drugs submitted between 2003 and 2017, Black patients represented less than 5% of patients in the clinical data.

Unadjusted analyses for derived response rates (dRR), which included 1400 White patients and 360 Black patients, also showed racial differences, with favorable response rates for White patients. During first-line treatment, the dRR was 84.8% for Black patients and 86.9% for White patients, and during second-line treatment, dRR was 67.2% for Black patients and 72.4% for White patients.

The researchers emphasized how the inclusion of a derivation of a response endpoint based on their real-world data (RWD) source—a longitudinal de-identified electronic health record-derived database—was a strength of the study. They also noted that data like theirs offer insights into how data from randomized controlled trials translate into real-world practice and offer indications of how treatments work in more diverse patients than typically seen in clinical trials.

“Currently, no consensus exists on the clinical utility and adequacy of certain RWD endpoints (ie, dRR and rwOS), as they inherently differ from clinical trial endpoints considered the gold standard in evaluating outcomes for drug approval, and the association between the two is unclear,” highlighted the researchers.

“Potential gaps include: (1) RWD are not collected systematically and patients may have extended periods without assessments or may not have adequate follow-up time; and (2) laboratory assessments and response criteria may differ by physician or practice settings,” they said.

The researchers also recognized the potential bias of their findings due to the nonrandomization of their patients. They note that both observable factors, such as practice setting, and nonobservable factors, such as cytogenetics, could have had impacts on the populations.

Reference

Maignan K, Fashoyin-Aje L, Torres A, et al. Exploring racial disparities in treatment patterns and outcomes for patients with multiple myeloma using real-world data. Blood Cancer J. Published online April 19, 2022. doi:10.1038/s41408-022-00665-x