By Karen L. Kier, Pharmacist on behalf of the ONU Healthwise Team 

In 2001, If I Didn’t Have You won the Academy Award for Best Original Song from the Disney movie Monsters, Inc. The song was written by Randy Newman and performed by Billy Crystal and John Goodman.   

Randy Newman became a Disney Legend in 2007 for his songwriting on 9 different Disney-Pixar movies. He has won 7 Grammy Awards and 3 Emmys.  

What if we didn’t have new drugs?

The FDA approves an average of 3-4 novel medications every month.  In 2023, the agency approved 55 novel drugs exceeding 37 in 2022.  This number reflects novel drugs approved and not the total number.  

What makes something novel?  According to the FDA, it is a medication with an active ingredient never before available in the US.  It is a drug working differently than anything else.  In the past 2 years, 92 unique drugs entered the market to help manage a diverse set of conditions.  Approximately 35% of these approvals are for the treatment and management of cancer.  

In addition to novel drug approvals, the FDA evaluates older drugs for new uses or new doses or dosage forms.  Anytime the FDA evaluates a drug, it reviews scientific evidence in the form of clinical studies to determine the drug’s effectiveness and safety.  The FDA only approves the drug for the indication or use as presented in the studies.  

For example, Merck submitted a new drug application for lovastatin (Mevacor®) to the FDA in 1986.  The FDA approved the drug for commercial use in September 1987.  The drug initially was only approved for the indication to lower blood cholesterol or lipids based on the clinical trials. 

Other statins followed lovastatin on the market including the most widely prescribed atorvastatin (Lipitor®) and rosuvastatin (Crestor®).  These 2 statins have an FDA-approved indication for lowering cholesterol, but they also have FDA-approved uses for preventing heart disease including preventing a second heart attack.  Why?  The companies supplied additional evidence to the FDA to prove this benefit and the FDA agreed. 

If a drug manufacturer wants to add other uses for the drug, it is required to submit more studies showing evidence before the FDA will approve.  A drug can be on the market for years before it gains additional FDA-approved indications.  

Can a drug be used for indications besides what is FDA-approved?  Yes!

Once a drug is on the market, a physician or prescriber can write for a drug not FDA-approved for a specific condition.  This is legal and referred to as off-label prescribing.  Off-label prescribing is very common in the United States with estimates indicating about 1 out of every 5 prescriptions filled are for off-label uses. 

Why would a prescriber write for a drug if the FDA has not approved the indication or use?  

Once a drug is on the market, the manufacturer is under no obligation to go back (and spend more money) to get new indications for a drug. The caveat is the manufacturer is restricted in its ability to promote or market the drug for off-label uses.  It must stay consistent with the FDA-approved indications. 

How do healthcare professionals know about off-label uses?  

Research and clinical trials for drugs are constantly being done around the globe.  This research will often evaluate a drug for other uses and publish the data in peer-reviewed medical journals.  

Healthcare professionals like physicians, nurse practitioners, nurses, physician assistants, and pharmacists are required to keep up with the literature and treatment guidelines to maintain their licenses.  They may attend meetings to gather information or read professional journals.  

These resources will provide information on best practices to manage conditions including both FDA-approved indications as well as off-label uses.  

For example, a December 15, 2023 study in JAMA Network Open found statins reduced the risk of blood clots in women who were prescribed hormone replacement therapy for menopause. In the study, women taking a statin had an 18% lower risk of developing blood clots.  

Likewise, additional evidence from 2 studies found women treated for breast cancer survived longer when taking a statin drug.  Another study found women in breast cancer remission were less likely to have a recurrence of breast cancer if they were prescribed a statin.  

It is the study evidence to support positive outcomes and improve health. The cautionary tale is when drugs are used for off-label uses not supported by studies or evidence.  This is why it is critical for healthcare professionals to be lifelong learners.  

If we didn’t have you (off-label uses for drugs), our therapies for managing conditions would be very limited.  

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