Posted: Tuesday, February 21, 2023
Real-world data to assess the efficacy of therapies for patients with diffuse large B-cell lymphoma (DLBCL) may be used to supplement randomized controlled trials, according to an article published in the American Journal of Hematology. Despite these studies having an increased susceptibility to bias, there is potential for them to provide complementary evidence of a treatment’s efficacy, suggested Grzegorz Nowakowski, MD, of the Mayo Clinic, Rochester, Minnesota, and colleagues.
Recently, real-world data have been used as the control arm for single-arm clinical trials in randomized controlled trials. Studies such as SCHOLAR-1, RE-MIND, and RE-MIND2 have focused on a comparison of the efficacy of this approach in DLBCL and have demonstrated the need to balance covariates to improve confidence in the study’s endpoints. However, this analysis also highlights the lack of inclusion of patients requiring urgent treatment, which may consequently negatively impact the individual study’s generalizability.
Furthermore, to account for the baseline differences in patient characteristics, propensity scores have been implemented to ensure a balance between groups. The effectiveness of the propensity score in accounting for differences between the study’s populations determines the extent of the study’s validity. Other studies have addressed the differences in patient characteristics by using matching-adjusted indirect comparisons. However, there have been issues with this approach’s comparison of the enrolled and evaluable populations.
Moreover, in studies using real-world data, there is an increased chance of bias given that treatment selection is commonly influenced by a patient’s characteristics. To mitigate this bias, it has been suggested that the use of similar eligibility and noneligibility criteria may reduce the differences in patient profiles between randomized controlled trials and studies using real-world data.
Disclosure: For full disclosures of the study authors, visit onlinelibrary.wiley.com.
American Journal of Hematology