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Availability of experimental therapy outside oncology randomized clinical trials in the United States.

Publication ,  Journal Article
Hamilton, EP; Lyman, GH; Peppercorn, J
Published in: J Clin Oncol
December 1, 2010

PURPOSE: Investigational cancer therapies may be available outside trials as "off-protocol therapy" (OPRx), with implications for patient safety, trial accrual, and access to care. We conducted a literature-based analysis of recent randomized trials to evaluate the potential scope and impact of OPRx in the United States. METHODS: A MEDLINE search identified all English-language phase III medical oncology randomized clinical trials (RCTs) published over a 2-year period ending April 17, 2008. Determination of OPRx availability was based on US Food and Drug Administration approval for any indication. We limited assessment of accrual to studies with US sites. Data from articles were extracted independently by two investigators. RESULTS: Among 172 eligible RCTs, the majority (108; 63%) evaluated drugs that were available OPRx in the United States at trial initiation, while an additional 19 (11%) evaluated interventions that became available during the trial. Among trials with US sites, time to accrual was slower (41 vs 22 months; P = .002) and less efficient (8.8 v 22.7 patients per month; P = .001) when OPRx was available. Sixty-six percent of RCTs reported at least one increased grade 3 to 4 toxicity in the experimental arm, 47% reported superior efficacy for at least one major clinical outcome in the experimental arm, and 27% reported improvement in overall survival. These outcomes did not vary on the basis of OPRx availability. CONCLUSION: The majority of recent oncology RCTs involve experimental interventions that are available outside trials in the United States with potential impact on trial accrual. The safety and efficacy of novel interventions must be determined by clinical trials.

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Published In

J Clin Oncol

DOI

EISSN

1527-7755

Publication Date

December 1, 2010

Volume

28

Issue

34

Start / End Page

5067 / 5073

Location

United States

Related Subject Headings

  • United States
  • Therapies, Investigational
  • Randomized Controlled Trials as Topic
  • Oncology & Carcinogenesis
  • Off-Label Use
  • Neoplasms
  • Humans
  • 1112 Oncology and Carcinogenesis
  • 1103 Clinical Sciences
 

Citation

APA
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Hamilton, E. P., Lyman, G. H., & Peppercorn, J. (2010). Availability of experimental therapy outside oncology randomized clinical trials in the United States. J Clin Oncol, 28(34), 5067–5073. https://doi.org/10.1200/JCO.2010.28.6567
Hamilton, Erika P., Gary H. Lyman, and Jeffrey Peppercorn. “Availability of experimental therapy outside oncology randomized clinical trials in the United States.J Clin Oncol 28, no. 34 (December 1, 2010): 5067–73. https://doi.org/10.1200/JCO.2010.28.6567.
Hamilton EP, Lyman GH, Peppercorn J. Availability of experimental therapy outside oncology randomized clinical trials in the United States. J Clin Oncol. 2010 Dec 1;28(34):5067–73.
Hamilton, Erika P., et al. “Availability of experimental therapy outside oncology randomized clinical trials in the United States.J Clin Oncol, vol. 28, no. 34, Dec. 2010, pp. 5067–73. Pubmed, doi:10.1200/JCO.2010.28.6567.
Hamilton EP, Lyman GH, Peppercorn J. Availability of experimental therapy outside oncology randomized clinical trials in the United States. J Clin Oncol. 2010 Dec 1;28(34):5067–5073.

Published In

J Clin Oncol

DOI

EISSN

1527-7755

Publication Date

December 1, 2010

Volume

28

Issue

34

Start / End Page

5067 / 5073

Location

United States

Related Subject Headings

  • United States
  • Therapies, Investigational
  • Randomized Controlled Trials as Topic
  • Oncology & Carcinogenesis
  • Off-Label Use
  • Neoplasms
  • Humans
  • 1112 Oncology and Carcinogenesis
  • 1103 Clinical Sciences