European Journal of Clinical Pharmacology

, Volume 75, Issue 9, pp 1193–1200 | Cite as

Regulatory characteristics and pivotal study design of US Food and Drug Administration approval of drugs for major vs. minor cancer

  • Kenji YamashitaEmail author
  • Masayuki Kaneko
  • Mamoru Narukawa
Clinical Trial



We aimed to investigate the regulatory approval of drugs for cancers by the US Food and Drug Administration based on the cancer type (major vs. minor), including the use of expedited development programs and duration from Investigational New Drug application (IND) to marketing approval.


From publicly available records and through a Freedom of Information Act request, we gathered data to evaluate regulatory characteristics and pivotal study design for 115 anticancer drug approvals between 2012 and 2017 and the data were analyzed based on cancer incidence (major vs. minor cancers) and how expedited programs, orphan drug designation, and pivotal study design contribute to expedited approval was studied.


Drugs targeting minor cancers more frequently (67%; P = 0.0155) utilized breakthrough therapy designation and/or accelerated approval, both of which significantly contributed to expedited drug approval (median time from IND to approval, 6.4 years; P = 0.0008, 6.2 years; P < 0.0001). Drug approvals for pivotal study design without a comparator arm took significantly less time from IND to approval (median time from IND to approval, 6.2 years; P < 0.0001).


Drugs targeting minor cancers have frequently utilized the expedited development programs; thus, efficiently shortening time to approval. As many of such drugs are approved based on non-comparative pivotal studies, meticulous evaluation and follow-up should be performed for such drugs after their approval.


Drug approval Investigational new drug application Cancer U.S. Food and Drug Administration Expedited development programs 


Compliance with ethical standards

Conflict of interest

Kenji Yamashita is an employee of MSD K.K. (a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA). Masayuki Kaneko declares that he has no conflict of interest. Mamoru Narukawa declares that he has no conflict of interest.

Ethical approval

This article does not contain any studies with human participants or animals.

Informed consent

For this study, a formal consent is not required.


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Copyright information

© Springer-Verlag GmbH Germany, part of Springer Nature 2019

Authors and Affiliations

  1. 1.Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical SciencesKitasato UniversityMinato-kuJapan

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