Evidence Based Medicine – Where's the Beef?

Blood in the Eye: You Make the Call
January 8, 2010
Standard of Care vs. FDA Approved – How We Choose a Treatment
January 13, 2010
Show all

Evidence Based Medicine – Where's the Beef?

Evidence based medicine (EBM) gives us the proof that a new drug or treatment is valid and effective. The FDA provides a sound process by which treatments in the U.S. become evidence based.

I believe that doctors should base their medical decisions and recommendations based upon evidence based medicine.  Evidence based medicine is the “proof” that a given treatment is worthwhile.  It is the process through which a new procedure, or drug, must pass to gain acceptance.

In the U.S., in order for a new drug to acceptable, it must be proven to be safe and effective.  If you are a regular reader, many times I’ll write about new drugs entering a particular phase of clinical testing.  These clinical trials, as defined by the FDA (U.S. Food and Drug Administration), are the “beef” of evidence based medicine.

Clinical trials have 5 phases.

Phase o – determines a drugs behavior in humans
Phase I –  first testing in humans for safety
Phase II – confirms safety info from phase I,  additional toxicity information and efficacy
Phase III – multicenter trials, test against “gold standard”
Phase IV – if needed, tests for long-term safety

When developing a new drug, a pharmaceutical company will test small amounts of the drug on human subjects to get a feeling of how the drug is processed (metabolized) and the rate at which this is completed.  This is Phase 0.

Phase I clinical trials now begin testing for human safety utilizing larger “real-life” doses of the developing medication.  Phase II and III are both focused on establishing a safety profile differing only by the numbers of patients tested and the number of testing centers.  Safety profile testing also includes the determination of side-effects.

Phase III also tests for efficacy, meaning, does the drug do what it is intended to do and how does it stack up against the established treatment.  For instance, does Drug X, a medicine designed to cure headache, do a better job than acetaminophen. It is during this stage that clinical trials often use the words randomized, prospective and double-blind.  These are testing methods to really evaluate a new treatment versus an older, established treatment.  It is the best way to test a new drug.

If a drug has an acceptable safety profile and completes Phase III clinical trials, then it may be ready for introduction into the “market.”  A company then will file a New Drug Application asking for approval.

In a nutshell, that’s how a drug gets to market.

What Does This Mean? In other words, the FDA, acting as an objective non-biased source, determines if there is enough “evidence” to provide “approval” for a new drug.  The FDA reviews the evidence.  If the evidence is lowsy, the product doesn’t get developed.

Evidence based medicine provides objective evidence that therapies are valid and work.  It is the docs job to be aware of the advantages and disadvantages of a particular treatment. EBM provides a safety net for our consumers, the patients.

As I continue to urge everyone to read critically, look and see if a particular drug is FDA approved.  It will also list an indication.  For example, Drug X is FDA approved for the treatment of itchy teeth.  Remember too, that supplements (vitamins, remedies) do NOT have to have the FDA approval for them to be sold in stores.  Safety data for supplements, at present, is also not needed.

There are too many remedies and treatments, on or off the internet, that have no evidence whatsoever.  Treatments, especially for diabetes, are rampant.  Too many believe that vitamins cure macular degeneration, they don’t.

Remember, read critically.

“Randy”

Randall V. Wong, M.D.
Ophthalmologist, Retina Specialist
Fairfax Virginia