Standard of Care vs. FDA Approved – How We Choose a Treatment

13 Jan Standard of Care vs. FDA Approved – How We Choose a Treatment

Avastin® is not FDA (Food and Drug Administration) approved for treating wet macular degeneration, but it is the standard of care for treating the disease.   The FDA has given approval for Avastin to be used to treat a variety of cancers, but nothing about eyes.

What is Standard of Care? Standard of care is the treatment that another prudent healthcare professional, of similar backgrounds and training,  would give to the same patient.  As an example, if I choose to treat you with Lucentis for wet macular degeneration and I am complying with the local standard of care, another retina specialist practicing across the street, would choose the same treatment for you.

What About FDA Approval? Last post, we spoke about evidenced based medicine and the need for a new drug to gain FDA approval.  Once a drug gains FDA approval, it may be used “off-label.”  Off-label indicates that healthcare providers have found other uses for the drug, in addition to the FDA indication.

Avastin is used “off-label” for the treatment of wet macular degeneration.  Avastin has FDA approval for the treatment of certain colon, breast, lung, kidney and brain tumors.

What About Lucentis? Lucentis® is made by the same manufacturer as Avastin (Genentech/Roche).  That manufacturer did go through clinical trials, and costs, of getting FDA approved for wet macular degeneration.

Avastin and Lucentis are chemically very similar, they are cousins.  One is much more expensive than the other.  Both are anti-VEGF treatments.

On Becoming the Standard of Care In this case, several people receiving chemotherapy for colon cancer started reporting that their vision was improving.  Some smart docs paid attention to these comments.  In short, because of the similarities, Avastin was soon used for intraocular injection.

The doctors that pioneered this approach designed a few “randomized” studies and reported their results at our industry meetings.  Others repeated similar studies and were finding the same results.

This then becomes intriguing for community physicians (like yours truly) and we try it ourselves.  If it works, we embrace the new “off-label” treatment.

What Does This Mean? In the case of Lucentis and Avastin, this means that we use a similar drug, get similar (or perhaps even better) results, but significant cost savings.  From a healthcare point of view, while Avastin was never FDA approved, the industry provides its own proof.

This is still evidenced based medicine.  While the traditional FDA clinical trials were not performed, similar trials are usually performed by institutions or groups of doctors that feel a new use, or indication, for a treatment is possible.  The trials are usually shorter and focused on efficacy of the new treatment.  The best “studies” are those that are prospective, randomized and double blinded.  These ensure the accuracy of any results.  The results are presented at meetings and in so-called “peer reviewed” journals.  It is in this arena, that we decide, outside of government (i.e. FDA) sanction, if new treatments are worthwhile.

It is at this level, we read a plethora of “studies” making claims.  There are good and bad studies.  This is where we need to “read critically.”

“Randy”

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