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U.S. Food and Drug Administration anticancer drug approval trends from 2016 to 2018 for lung, colorectal, breast, and prostate cancer

Published online by Cambridge University Press:  28 November 2019

Tatiane Bomfim Ribeiro Open the ORCID record for Tatiane Bomfim Ribeiro [Opens in a new window] ,
Adalton Ribeiro Open the ORCID record for Adalton Ribeiro [Opens in a new window] ,
Luíza de Oliveira Rodrigues ,
Guilherme Harada  and
Moacyr Roberto Cuce Nobre
Tatiane Bomfim Ribeiro*
Affiliation:
Department of Preventive Medicine, Faculty of Medicine, University of São Paulo, São Paulo, Brazil
Adalton Ribeiro
Affiliation:
São Paulo Health Department, São Paulo, Brazil
Luíza de Oliveira Rodrigues
Affiliation:
UNIMED Belo Horizonte, Minas Gerais, Brazil
Guilherme Harada
Affiliation:
State of São Paulo Cancer Institute, University of Sao Paulo School of Medicine, Sao Paulo, Brazil Oncology Center, Hospital Sírio-Libanês, São Paulo, Brazil
Moacyr Roberto Cuce Nobre
Affiliation:
Department of Preventive Medicine, Faculty of Medicine, University of São Paulo, São Paulo, Brazil Clinical Epidemiology and Research Support Team, Heart Institute (InCor), São Paulo, Brazil
*
Author for correspondence: Tatiane Bomfim Ribeiro, E-mail: tatianeribeiro@usp.br
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Abstract

Objective

This paper aims to describe the clinical and regulatory aspects of new drugs and indications that were approved for lung, breast, prostate, and colorectal cancer, from 2016 to 2018, in order to provide health technology assessment trends in oncology.

Methods

Data were collected from the US Food and Drug Administration (FDA) online database for new medications and indications approved for the above-mentioned types of cancer. Data regarding clinical study characteristics and regulatory information were collected.

Results

From 2016 to 2018, 53 percent of the FDA approvals of new drugs and indications for the most incident cancers were for oral protein kinase inhibitor monotherapy for advanced lung cancer. Since 2018, four drugs were approved as tumor-agnostic therapies. A biomarker was included in 72 percent of indications, and 58 percent of approvals were for targeted therapies, potentially heralding an end to research into conventional cytotoxic agents. A special designation for faster approval was granted in 78 percent of new approvals. The majority of the studies were open label randomized controlled trials (RCTs) (44 percent), followed by blind RCTs, single-arm clinical trials, and cohort studies. Only 14 percent of studies used overall survival as the primary end point; the vast majority used surrogate end points, and did not use patient-important outcomes. Three biosimilars were approved in the period.

Conclusion

Advanced lung cancer therapy, mainly targeted drugs, accounted for 53 percent of approvals. Special designations for faster approval were used in 78 percent of FDA approvals, and four drugs were approved for tumor-agnostic treatment—a new form of approval.

Keywords

FDAUnited States Food and Drug AdministrationDrug approvalCancerLung cancerBreast cancerProstate cancerColorectal cancer
Type
Article Commentary
Information
International Journal of Technology Assessment in Health Care , Volume 36 , Issue 1 , 2020 , pp. 20 - 28
Copyright
Copyright © Cambridge University Press 2019

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