Mitsubishi Tanabe Pharma America Announces Real-World Data Analysis of Survival with RADICAVA (edaravone)

On Aug 16, 2022

Mitsubishi Tanabe Pharma America, Inc. announced the publication of a paper entitled, “Intravenous edaravone treatment in ALS and survival: an exploratory, retrospective, administrative claims analysis,” in eClinicalMedicine, part of The Lancet Discovery Science. Results from the analysis suggest that continued treatment with RADICAVA (edaravone) in people with amyotrophic lateral sclerosis (ALS) was associated with prolonged survival compared to those not treated with the drug in a real-world setting based on U.S. administrative claims data.

“Real-world data may bridge gaps in knowledge that exist between clinical trials and everyday medical practice,” said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. “While a randomized clinical trial is required to support the findings from this analysis, these data provide important insights from a real-life setting and deepen our understanding of RADICAVA’s role in the treatment of ALS.”

The retrospective, observational study utilized Optum’s Clinformatics Data Mart (CDM), a real-world de-identified database of administrative health claims across the U.S. for members with commercial or Medicare Advantage health plans, to assess overall survival among commercially insured ALS patients (≥18 years) who were treated continuously with RADICAVA, compared to a control group of patients not prescribed RADICAVA. The median treatment duration with RADICAVA was 8.6 months. Implementing 1:1 propensity score matching, the analysis compared 318 non-RADICAVA-treated control patients with 318 patients who initiated RADICAVA treatment between August 8, 2017, and March 31, 2020.

“ALS has been a clinically challenging disease to evaluate due to its heterogeneity and average life expectancy,” said Benjamin Rix Brooks, M.D., a longtime leader in ALS research and lead author of the study. “The results from this analysis showed that, for this specific group, treatment with RADICAVA led to the observation of a lower number of deaths and risk of death as well as longer overall survival estimates compared to those not on therapy. These real-world findings are encouraging and will help inform future research for this devastating disease.”

Results from the analysis showed:

Treatment with RADICAVA was associated with a six-month longer median survival compared with the non-RADICAVA-treated patients. The median treatment duration with RADICAVA was 8.6 months.
- Median survival was 29.5 months (95% CI, 25.4-35.9) for the RADICAVA-treated patients and 23.5 months (95% CI, 20.0-28.0) for the non-RADICAVA-treated patients.
The risk of death during the study was 27% lower for the RADICAVA-treated patients than for non-RADICAVA-treated patients (hazard ratio [HR], 0.73; 95% CI, 0.59–0.91).
Between August 8, 2017, and March 31, 2021, 155 all-cause deaths (48.7%) were reported among RADICAVA-treated patients vs. 196 (61.6%) among the non-RADICAVA-treated patients.

It is important to note that results from this study are not generalizable and cannot be used to determine definitive conclusions about the effects of treatment. This study used real-world data, it was not randomized, it was observational, exploratory and retrospective in nature, and may be subject to unknown bias and confounding factors. Understanding the utility and limitations of real-world data is critical to proper application of insights.

This analysis was funded and conducted by MTPA.