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BMC Res Notes. 2017 Jul 28;10(1):350. doi: 10.1186/s13104-017-2687-5.

Characterizing expanded access and compassionate use programs for experimental drugs.

Author information

1
Division of Medical Ethics, Department of Population Health, NYU School of Medicine, 227 East 30th Street, Office 723, New York, NY, 10016, USA. Jennifer.miller@nyumc.org.
2
Bioethics International, New York, USA. Jennifer.miller@nyumc.org.
3
Section of General Internal Medicine and Robert Wood Johnson Foundation Clinical Scholars Program, Department of Medicine, Yale School of Medicine, New Haven, USA.
4
Department of Health Policy and Management, Yale School of Public Health, New Haven, USA.
5
Center for Outcomes Research and Evaluation, Yale-New Haven Health, 789 Howard Ave, New Haven, CT, 06519, USA.
6
Cognition Therapeutics, Inc., 2403 Sidney St # 261, Pittsburgh, PA, 15203, USA.
7
Division of Medical Ethics, Department of Population Health, NYU School of Medicine, 227 East 30th Street, Office 722, New York, NY, 10016, USA.

Abstract

OBJECTIVE:

We sought to determine the characteristics of "expanded access" and "compassionate use" programs registered in ClinicalTrials.gov and to determine the percentage of drugs provided through these programs that ultimately received FDA marketing approval.

RESULTS:

We identified 398 expanded access and compassionate use programs (hereafter referred to as expanded access programs) registered on ClinicalTrials.gov. Industry funded 61% (n = 241) of programs individually or collaboratively, while NIH and the US Federal Government rarely funded programs (3% [n = 11] and 2% [n = 6], respectively). Most programs provided access to drugs (71% [n = 282]), 11% to biologics (n = 43), and 10% to medical devices (n = 40). These programs covered 460 unique conditions, the most common being HIV (n = 26), leukemia (22), and multiple myeloma (n = 14). Only 2% of programs reported results in ClinicalTrials.gov. Most programs (82%) were open to enrolling adults and seniors (n = 326). These programs provided access to 210 unique experimental drugs, of which 76% have received FDA approval.

KEYWORDS:

21st Century Cures Act; Access to medicines; Bioethics; Compassionate use; Ethics; Expanded access; Experimental drugs; Pharmaceutical industry; Policy; Real-world evidence; Right to Try Laws

PMID:
28754150
PMCID:
PMC5534121
DOI:
10.1186/s13104-017-2687-5
[Indexed for MEDLINE]
Free PMC Article

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