Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:


Off-label use: Oft not evidence based

Physicians lack knowledge of off-label drug use and FDA approval status

In a recent national survey, a substantial minority of physicians erroneously believed that certain off-label uses of prescription drugs were approved by the Food and Drug Administration. This mistaken belief could encourage them to prescribe these drugs, despite the lack of scientific evidence supporting such use.

"Off-label prescribing is common, but researchers have not always known why. Our research shows that some off-label prescribing might be driven by mistaken beliefs about FDA approval and the level of evidence supporting off-label drug use," said G. Caleb Alexander, MD, MS, Assistant Professor of Medicine at the University of Chicago Medical Center and corresponding author of the research, which will be published under an embargo in Pharmacoepidemiology and Drug Safety on August 21, 2009. "The results indicate an urgent need for more effective methods of informing physicians about the level of evidence supporting off-label drug use—especially for common off-label uses that are ineffective or carry unacceptable risks of harm."

Overall, physicians were able to correctly identify the FDA-approval status of just over half (mean 55%) of the 22 drug-indication pairs (i.e., a particular drug prescribed for a particular condition) that were included in the survey.

In many cases, the proportion of physicians who erroneously believed a particular drug was FDA approved for a specific indication was higher among physicians who had prescribed the drug for that indication.

For example, 26% of all physician respondents erroneously believed that lorazepam was FDA approved for chronic anxiety. That figure rose to 33% for physicians who had prescribed lorazepam for chronic anxiety.

And 13% of all physician respondents erroneously believed that quetiapine (Seroquel®) was FDA approved for dementia with agitation. That figure rose to 19% for physicians who had prescribed quetiapine for dementia with agitation. At the time of the study there was even an FDA-advisory specifically urging caution regarding the off-label use of quetiapine in patients with dementia.

The survey of 1,199 physicians (599 primary care physicians and 600 psychiatrists) was conducted in 2007-2008 and included 22 drug-indication pairs. The indications varied in their FDA approval status from on-label use to off-label use supported by medical evidence to off-label use deemed to be ineffective. (The level of evidence supporting or not supporting each specific use was based on Drugdex, an independent drug compendium.)

The FDA makes it clear that they regulate the marketing of prescription drugs, not prescribing. The agency approves drugs for marketing with an official "label" that stipulates an indication, dose, intended population, duration of use, and other specifications. However, physicians and other licensed prescribers are free to prescribe any approved drug for any indication, whether or not the indication is included on the drug's FDA-approved label.

"Some physicians and health care experts maintain that physicians should know the evidence, not the FDA labeling. However, knowledge about FDA labeling can be important because FDA approval of a drug for a specific indication indicates a clear threshold of evidence supporting that use," said Donna Chen, MD, Assistant Professor of Biomedical Ethics, Public Health Sciences, and Psychiatry at the University of Virginia. Dr. Chen is the lead author of the research, entitled "U.S. physician knowledge of the FDA-approved indications and evidence base of commonly prescribed drugs: results of a national survey".

A study published in the Archives of Internal Medicine by other investigators in 2006 indicated that approximately 21% of drug uses in the United States occur for off-label purposes, with 73% of those cases lacking scientific evidence of the drug's effectiveness. The highest rates of off-label use were for anticonvulsants (74%), antipsychotics (60%) and antibiotics (41%).

"We hope our research will increase awareness of off-label prescribing and highlight the pressing need for more evidence-based use of prescription drugs," Alexander said. "Although some off-label uses are well-supported, many are not. New ways are needed to help physicians tap the scientific evidence supporting various prescription drug uses."

Some disadvantages of off-label use

May diminish public expectation that drugs will be evaluated for safety and efficacy before use

Blunts industry incentives to perform studies required for FDA label changes

Drugs used off label may have unrecognized safety and efficacy problems

Promotes use of drugs in populations (e.g., children, the elderly) for which they have not been tested

Source: G. Caleb Alexander, MD, MS, Assistant Professor of Medicine at the University of Chicago Medical Center

Some advantages of off-label use

Allows for clinical innovation, especially for patients who do not respond to standard treatments

May be only available option for uncommon conditions or for patient populations that have not been studied

Allows physicians to anticipate growing evidence of efficacy prior to formal evaluation

Increases return on investment for pharmaceutical firms

Source: G. Caleb Alexander, MD, MS, Assistant Professor of Medicine at the University of Chicago Medical Center

Greg Borzo | EurekAlert!
Further information:

More articles from Life Sciences:

nachricht ‘Farming’ bacteria to boost growth in the oceans
24.10.2016 | Max-Planck-Institut für marine Mikrobiologie

nachricht Calcium Induces Chronic Lung Infections
24.10.2016 | Universität Basel

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Light-driven atomic rotations excite magnetic waves

Terahertz excitation of selected crystal vibrations leads to an effective magnetic field that drives coherent spin motion

Controlling functional properties by light is one of the grand goals in modern condensed matter physics and materials science. A new study now demonstrates how...

Im Focus: New 3-D wiring technique brings scalable quantum computers closer to reality

Researchers from the Institute for Quantum Computing (IQC) at the University of Waterloo led the development of a new extensible wiring technique capable of controlling superconducting quantum bits, representing a significant step towards to the realization of a scalable quantum computer.

"The quantum socket is a wiring method that uses three-dimensional wires based on spring-loaded pins to address individual qubits," said Jeremy Béjanin, a PhD...

Im Focus: Scientists develop a semiconductor nanocomposite material that moves in response to light

In a paper in Scientific Reports, a research team at Worcester Polytechnic Institute describes a novel light-activated phenomenon that could become the basis for applications as diverse as microscopic robotic grippers and more efficient solar cells.

A research team at Worcester Polytechnic Institute (WPI) has developed a revolutionary, light-activated semiconductor nanocomposite material that can be used...

Im Focus: Diamonds aren't forever: Sandia, Harvard team create first quantum computer bridge

By forcefully embedding two silicon atoms in a diamond matrix, Sandia researchers have demonstrated for the first time on a single chip all the components needed to create a quantum bridge to link quantum computers together.

"People have already built small quantum computers," says Sandia researcher Ryan Camacho. "Maybe the first useful one won't be a single giant quantum computer...

Im Focus: New Products - Highlights of COMPAMED 2016

COMPAMED has become the leading international marketplace for suppliers of medical manufacturing. The trade fair, which takes place every November and is co-located to MEDICA in Dusseldorf, has been steadily growing over the past years and shows that medical technology remains a rapidly growing market.

In 2016, the joint pavilion by the IVAM Microtechnology Network, the Product Market “High-tech for Medical Devices”, will be located in Hall 8a again and will...

All Focus news of the innovation-report >>>



Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

Agricultural Trade Developments and Potentials in Central Asia and the South Caucasus

14.10.2016 | Event News

World Health Summit – Day Three: A Call to Action

12.10.2016 | Event News

Latest News

Oasis of life in the ice-covered central Arctic

24.10.2016 | Earth Sciences

‘Farming’ bacteria to boost growth in the oceans

24.10.2016 | Life Sciences

Light-driven atomic rotations excite magnetic waves

24.10.2016 | Physics and Astronomy

More VideoLinks >>>