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Off-Label Indications for Medication Use and the Published Literature

Rebecca Dresser, JD
JAMA. 2008;300(12):1411-1412. doi:10.1001/jama.300.12.1411-b
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To the Editor: In their Commentary criticizing the guidelines proposed by the Food and Drug Administration (FDA) on off-label promotion, Drs Psaty and Ray1 point to shortcomings in the peer-reviewed literature about unapproved uses of approved drugs and devices. However, in focusing on sponsors' distribution of such literature, the authors fail to recognize the full extent of the problem.

As Psaty and Ray observe, the published peer-reviewed literature on off-label uses may present an inaccurate picture of a product's risks and benefits. Studies with unfavorable results may not be published, and published studies may be designed and presented in ways that exaggerate benefits and minimize risks. Prohibiting sponsors from distributing peer-reviewed literature on off-label use will not rectify the situation, however, for physicians rely on that very same literature to guide their off-label prescribing.

Without rigorous studies and independent evaluation of a product's off-label effects, physicians lack solid support for off-label prescribing. This puts patients at risk of receiving harmful or ineffective treatments. The real challenge to regulators and industry and medical professionals is to develop an oversight approach that produces a stronger basis for off-label prescribing.2 4

AUTHOR INFORMATION

Financial Disclosures: None reported.

REFERENCES

Psaty BM, Ray W. FDA guidance on off-label promotion and the state of the literature from sponsors.  JAMA. 2008;299(16):1949-1952
PubMedCrossRef
Gottlieb S. Opening Pandora's pillbox: using modern information tools to improve drug safety.  Health Aff (Millwood). 2005;24(4):938-958
PubMedCrossRef
Oates M. Facilitating informed medical treatment through production and disclosure of research into off-label uses of pharmaceuticals.  NYU Law Rev. 2005;801272-1308
Dresser RS. The curious case of off-label use.  Hastings Cent Rep. 2007;37(3):9-11
CrossRef

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Psaty BM, Ray W. FDA guidance on off-label promotion and the state of the literature from sponsors.  JAMA. 2008;299(16):1949-1952
PubMedCrossRef
Gottlieb S. Opening Pandora's pillbox: using modern information tools to improve drug safety.  Health Aff (Millwood). 2005;24(4):938-958
PubMedCrossRef
Oates M. Facilitating informed medical treatment through production and disclosure of research into off-label uses of pharmaceuticals.  NYU Law Rev. 2005;801272-1308
Dresser RS. The curious case of off-label use.  Hastings Cent Rep. 2007;37(3):9-11
CrossRef
September 24, 2008
Bruce M. Psaty, MD, PhD; Wayne Ray, PhD
JAMA. 2008;300(12):1411-1412.
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