Evaluation of Representation of Women As Authors in Pivotal Trials Supporting US Food and Drug Administration Approval of Novel Hematological Drugs

Introduction Pivotal trials evaluating the efficacy and safety of novel drugs provide the basis for their US Food and Drug Administration (FDA) approvals. Reportedly, women were underrepresented as authors in pivotal trials supporting approval of novel cardiometabolic drugs . However, gender inequity in authorship across pivotal trials of FDA-approved novel hematological drugs remains unknown. Therefore, in the present study, we sought to investigate whether gender disparity exists in the representation of women as authors of pivotal trials supporting approval of novel hematological drugs.

Methods Drugs@FDA portal was searched for novel hematological drugs approved between 2008 and 2022. Drug approval labels were searched to identify all pivotal trials listed in Section 14 under "Clinical Studies.” Only pivotal trials with corresponding trial publications were included in this study. The gender of trial authors was captured as binary (women and men) and was identified by using the Genderize software (Demografix ApS) or by verifying personal pronouns on authors’ institutional profiles or other sources. Study-level estimates for the proportion of women authors were pooled using the DerSimonian and Laird random-effects meta-analysis of proportions model. Analyses were conducted with OpenMeta [Analyst] software version 10.12.

Results From 2008 to 2022, the FDA approved a total of 45 novel hematological drugs, corresponding to 60 trials. Of these, 57 trial publications (21,527 participants) were included in this study (Table 1). Three publications included the results of two trials each. A total of 1203 authors were screened, and 15 were excluded because of the inability to assign author gender. Genderize was used to identify the gender of 1137 authors, which was further confirmed by checking personal pronouns listed in institutional profiles or other sources. Of the 66 authors with a prediction probability estimate less than 90.0% on Genderize, author gender was identified for 51 by using personal pronouns listed in institutional profiles or other sources.

The median number of men and women authors of trial publications was 12 (IQR, 9-20) and 6 (IQR, 4-8), respectively. The overall proportion of women authors was 28.6% (95% CI, 25.3%-31.9%). The proportion of women as first and senior authors was the same, estimating about 22.8% (95% CI, 11.9%-33.7%).

Conclusion Our study demonstrates gender inequity in authorship of pivotal efficacy trials supporting approval of novel Hematological drugs by the FDA. Institutions should devise targeted policies addressing unique challenges faced by women in medicine and develop a workplace environment conducive to women's academic success.

Jaglal:Sanofi: Consultancy; Novartis: Consultancy.