Evidence-based medicine (EBM) aims to apply evidence gained from the scientific method to certain parts of medical practice. It seeks to assess the quality of evidence relevant to the risks and benefits of treatment (including lack of treatment). According to the Centre for Evidence-Based Medicine, "Evidence-based medicine is the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients."
The key for evidence-based medicine is the quality of evidence. Obviously the regulatory such as FDA applied very strict efficacy standard. According to a slide on FDA's website, FDA does not permit Sponsors To Promote Off-Label Uses because such behaviour
- Would diminish or eliminate incentive to study the use and obtain definitive data.
- Could result in harm to patients from unstudied uses that actually lead to bad results, or that are merely ineffective.
- Would diminish the use of evidence-based medicine.
- Could ultimately erode the efficacy standard.
However, there are also different voices.