Real-world data control arm provides new look into effectiveness of lymphoma therapies

Real-world data (RWD) is having an impact on cancer care in a number of ways, from supporting diverse and representative clinical trials to offering critical insights into the post-market performance of novel therapies.

By using rich and varied datasets to generate comprehensive, longitudinal portraits of patients and their health experiences, researchers can leverage RWD to answer important questions of viability, safety, and efficacy in an environment of rapid breakthroughs and new discoveries.

A recent study from an international team of oncologists and Bristol Myers Squibb (BMS) illustrates exactly how valuable RWD can be for uncovering important efficacy information.

Using real-world data from COTA and other sources, the team developed an external control arm to provide additional context around the efficacy of lisocabtagene maraleucel (liso-cel) compared with conventional (noncellular) therapies in patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL).

This type of lymphoma is aggressive, and patients tend to have a poor prognosis with few available treatments. To provide more options for patients, BMS has introduced Breyanzi, a CAR-T therapy with the potential to increase response rates and extend overall survival time.

Liso-cel has demonstrated favorable efficacy to prior standards of care, according to literature reviews. However, the authors acknowledge that these attempts to contextualize the results had limitations due to a lack of patient-level data to serve as matched comparators.

An external control arm helped to overcome these obstacles by allowing researchers to evaluate the comparative efficacy of Breyanzi with conventional therapies in the real-world at the patient level. By matching patients from the real-world cohort to patients from TRANSCEND, the team could put the initial results in context and better evaluate the efficacy of both approaches.

“[Real-world] data from each source were integrated to conform to a standardized, common study data model, thus allowing variables of comparable constructs from disparate data sources to be harmonized into a pooled dataset,” the BMS study authors write. “Consistent operational definitions were systematically applied during harmonization. Probabilistic methods were used to address the potential duplication of patients across the disparate de-identified data sources. All real-world patient-level data collection was retrospective and did not influence clinical practice or patient visit schedule.”

The results favored the use of the novel therapy over the existing standard of care, showing that the liso-cel therapy produced a better overall response rate (74% versus 39%; p<0.0001) and improved median overall survival (23.5 months vs. 6.8 months; p<0.0001).

The data is valuable for helping oncologists decide on treatment options for their patients. But the study also serves another purpose: providing evidence that external control arms are a viable and useful way to gain deeper, more detailed insights into standards of care.

“COTA is proud to work with partners across the clinical, academic, and pharmaceutical communities as an integral part of this ongoing discovery process,” said C.K. Wang, MD, the Chief Medical Officer at COTA. “We believe strongly in the promises of RWD to expand our shared knowledge of how to safely and effectively treat cancers, and we are eager to continue collaborating on similar projects to inform the future of cancer care for patients.”

To read the full study, please click here