Asthma biologic trial eligibility and real-world outcomes in the United States

Abstract

Objective

To compare the outcomes of real-world patients who would have been eligible for asthma biologics to those who would not have been eligible.

Methods

We used data from the OptumLabs Data Warehouse (OLDW) to categorize patients into eligible and ineligible groups based on clinical trials (n = 19 trials) used for Food and Drug Administration (FDA) approval. We then compared the change in the number of asthma exacerbations before and after biological initiation between the two groups.

Results

The percentage of people who would have been eligible for asthma biologic clinical trials ranged from 0–10.2%. The eligible group had a greater reduction in number of asthma exacerbations compared to the ineligible group based on eligibility criteria from 1 omalizumab trial (1.52, 95% CI 1.25, 1.8 in eligible vs. 0.47, 95% CI 0.43, 0.52 in ineligible) and from 1 dupilumab trial (1.6, 95% CI 0.92, 2.28 in eligible vs. 0.52, 95% CI 0.38, 0.65 ineligible). Notably, 15 of the 19 trials had fewer than 11 eligible people, limiting additional comparisons.

Conclusions

Fewer than 1 in 10 people in the United States treated with asthma biologics would have been eligible to participate in the trial for the biologic they used. Where comparisons could be made, trial eligible people have a greater reduction in exacerbations.

Supplemental data for this article is available online at https://doi.org/10.1080/02770903.2021.2010749 .

Keywords:

• Asthma
• biologics
• trials
• eligibility
• external validity

Additional information

Funding

This work was supported by National Institutes of Health, National Heart, Lung, and Blood Institute (NIH R21 HL140287) and the Mayo Clinic Robert D. and Patricia E. Kern Center for the Science of Health Care Delivery.