Publication statuses of clinical trials supporting FDA-approved immune checkpoint inhibitors: a meta-epidemiological investigation.


Journal

BMC cancer
ISSN: 1471-2407
Titre abrégé: BMC Cancer
Pays: England
ID NLM: 100967800

Informations de publication

Date de publication:
24 Oct 2019
Historique:
received: 15 02 2019
accepted: 02 10 2019
entrez: 26 10 2019
pubmed: 28 10 2019
medline: 27 2 2020
Statut: epublish

Résumé

The low data publication rate for Food and Drug Administration (FDA)-approved drugs, and discrepancies between FDA-submitted versus published data, remain a concern. We investigated the publication statuses of sponsor-submitted clinical trials supporting recent anticancer drugs approved by the FDA, with a focus on immune checkpoint inhibitors (ICPis). We identified all ICPis approved between 2011 and 2014, thereby obtaining 3 years of follow-up data. We assessed the clinical trials performed for each drug indication and matched each trial with publications in the literature. The primary benchmark was the publication status 2 years post-approval. We examined the association between time to publication and drug type using a multilevel Cox regression model that was adjusted for clustering within drug indications and individual covariates. Between 2011 and 2014, 36 anticancer drugs including 3 ICPis were newly approved by the FDA. Of 19 trials investigating the 3 ICPis, 11 (58%) were published within 2 years post-approval. We randomly selected 10 of the 33 remaining anticancer drugs; 68 of 101 trials investigating these drugs (67%) were published. Overall, the publication rate was 66% at 2 years post-approval with a median time to publication of 2.3 years. There was no significant difference in the time to trial publication between ICPis and other anticancer drugs (adjusted hazard ratio [HR], 1.1; 95% confidence interval [CI], 0.8-1.7; P = 0.55). However, findings related to non-ICPis investigated specifically in randomized phase 2 or phase 3 trials were significantly more likely to be published earlier than those related to ICPis (adjusted HR, 7.4; 95% CI, 1.8-29.5; P = 0.005). One in 3 sponsor-submitted trials of the most recently approved anticancer drugs remained unpublished 2 years post-FDA approval. We found no evidence that the drug type was associated with the time to overall trial publication.

Sections du résumé

BACKGROUND BACKGROUND
The low data publication rate for Food and Drug Administration (FDA)-approved drugs, and discrepancies between FDA-submitted versus published data, remain a concern. We investigated the publication statuses of sponsor-submitted clinical trials supporting recent anticancer drugs approved by the FDA, with a focus on immune checkpoint inhibitors (ICPis).
METHODS METHODS
We identified all ICPis approved between 2011 and 2014, thereby obtaining 3 years of follow-up data. We assessed the clinical trials performed for each drug indication and matched each trial with publications in the literature. The primary benchmark was the publication status 2 years post-approval. We examined the association between time to publication and drug type using a multilevel Cox regression model that was adjusted for clustering within drug indications and individual covariates.
RESULTS RESULTS
Between 2011 and 2014, 36 anticancer drugs including 3 ICPis were newly approved by the FDA. Of 19 trials investigating the 3 ICPis, 11 (58%) were published within 2 years post-approval. We randomly selected 10 of the 33 remaining anticancer drugs; 68 of 101 trials investigating these drugs (67%) were published. Overall, the publication rate was 66% at 2 years post-approval with a median time to publication of 2.3 years. There was no significant difference in the time to trial publication between ICPis and other anticancer drugs (adjusted hazard ratio [HR], 1.1; 95% confidence interval [CI], 0.8-1.7; P = 0.55). However, findings related to non-ICPis investigated specifically in randomized phase 2 or phase 3 trials were significantly more likely to be published earlier than those related to ICPis (adjusted HR, 7.4; 95% CI, 1.8-29.5; P = 0.005).
CONCLUSION CONCLUSIONS
One in 3 sponsor-submitted trials of the most recently approved anticancer drugs remained unpublished 2 years post-FDA approval. We found no evidence that the drug type was associated with the time to overall trial publication.

Identifiants

pubmed: 31651263
doi: 10.1186/s12885-019-6232-x
pii: 10.1186/s12885-019-6232-x
pmc: PMC6814120
doi:

Substances chimiques

Antineoplastic Agents 0
Immunologic Factors 0

Types de publication

Journal Article Meta-Analysis

Langues

eng

Sous-ensembles de citation

IM

Pagination

998

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Auteurs

Kenji Omae (K)

Department of Innovative Research and Education for Clinicians and Trainees (DiRECT), Fukushima Medical University Hospital, 1 Hikarigaoka, Fukushima city, Fukushima, 960-1295, Japan. omae416@fmu.ac.jp.
Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan. omae416@fmu.ac.jp.
Department of Urology, Tokyo Women's Medical University, Tokyo, Japan. omae416@fmu.ac.jp.

Yuki Kataoka (Y)

Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.
Hospital Care Research Unit, Hyogo Prefectural Amagasaki General Medical Center, Hyogo, Japan.

Yasushi Tsujimoto (Y)

Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.
Department of Nephrology and Dialysis, Kyoritsu Hospital, Hyogo, Japan.

Yusuke Tsutsumi (Y)

Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.
Department of Emergency Medicine, National Hospital Organization Mito Medical Center, Ibaraki, Japan.

Yosuke Yamamoto (Y)

Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.

Shunichi Fukuhara (S)

Department of Healthcare Epidemiology, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.

Toshi A Furukawa (TA)

Department of Health Promotion and Human Behavior, Kyoto University School of Public Health in the Graduate School of Medicine, Kyoto, Japan.

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Classifications MeSH