Studying Patient Reported Outcomes on Off-label use of approved medications:

Off-label use allows doctors to also prescribe approved medicines for unapproved use if

they think a medicine can help their patient. The decision to offer medicine to a patient

in an off-label way is made by doctors based on clinical trials and other research that’s

been done on the medicine.

From AHRQ:

“The United States Food and Drug Administration (FDA) drug approval process requires

clinical trials that demonstrate efficacy for a specific indication prior to market

availability. Once approval for an indication is given, however, the FDA has a minimal

role in determining whether drugs are prescribed for their approved uses or for other

“off-label” indications not part of the FDA-approved product labeling.”

“Prior studies of off-label prescribing have typically been conducted in narrowly defined

clinical contexts, including psychiatric disorders,1,2 bacterial infections,3 in children2,4

as well as in “orphan” clinical conditions not well served by FDA approved drugs.5,6

These studies have demonstrated that off-label use is frequent in these clinical areas,

that such uses arise from diverse circumstances, and that there is substantial variation

in the available evidence supporting particular off-label uses. Notably, while raising

some concerns about off-label use, the studies also identify disease- and patient-

specific situations where off-label use may be beneficial.”

 

1. AHRQ, ”Developing Evidence-Based Research Priorities for Off-Label Drug Use,”

AHRQ Research Report Jun 29, 2009.

https://effectivehealthcare.ahrq.gov/products/off-label-use-research-

priorities/research.

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2. Zain Mithani, MD, “Informed Consent for Off-Label Use of Prescription

Medications,” Virtual Mentor. 2012;14(7):576-581. doi:

10.1001/virtualmentor.2012.14.7.oped1-1207.