Oral anticancer drugs

How limited dosing options and dose reductions may affect outcomes in comparative trials and efficacy in patients

Vinay Prasad, Paul R. Massey, Tito Fojo

Research output: Contribution to journalArticle

33 Citations (Scopus)

Abstract

Historically, cancer medicine has avoided the problem of unequal dosing by comparing maximumtolerated doses of intravenous regimens with proportionate dose reductions for toxicity. However, in recent years, with the development of numerous oral anticancer agents, dosing options are arbitrarily and increasingly limited by the size of pills. We contend that an underappreciated consequence of pill size is unequal dosing in comparative clinical trials and that this can have an impact on outcomes. We discuss how comparative effectiveness trials can be unbalanced and how the use of doses that are not sustainable might affect outcomes, especially marginal ones. We further argue that because of their poor tolerability and their limited dosing options, which often result in large dose adjustments in response to toxicity, the real-world clinical effectiveness of oral anticancer agents may be diminished and may not emulate results achieved in registration trials.

Original languageEnglish (US)
Pages (from-to)1620-1629
Number of pages10
JournalJournal of Clinical Oncology
Volume32
Issue number15
DOIs
StatePublished - May 20 2014
Externally publishedYes

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Antineoplastic Agents
Pharmaceutical Preparations
Medicine
Clinical Trials
Neoplasms

ASJC Scopus subject areas

  • Cancer Research
  • Oncology
  • Medicine(all)

Cite this

Oral anticancer drugs : How limited dosing options and dose reductions may affect outcomes in comparative trials and efficacy in patients. / Prasad, Vinay; Massey, Paul R.; Fojo, Tito.

In: Journal of Clinical Oncology, Vol. 32, No. 15, 20.05.2014, p. 1620-1629.

Research output: Contribution to journalArticle

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