Examining Off-Label Drug Use: What a Pharmacist Needs to Know
According to the FDA, good medical practice requires physicians to use all legally available drugs to treat their patients. It’s a reasonable demand when talking about drugs that have been well studied and approved for the treatment of a particular condition. But what happens when the physician recommends off-label drug use? Pharmacists are legally required to review each prescription to ensure that the medication is appropriate, necessary, and is unlikely to cause adverse events.1 Without knowing the safety and efficacy of how a drug performs off-label, however, pharmacists are put in a poor position to give their patients the best care possible.
What Specific Concerns Does Off-Label Drug Use Raise?
With any off-label drug, there is no intensive study of efficacy and safety in use-appropriate patient populations. That means that even if a drug has been deemed safe for treating one particular condition, its safety has not been evaluated in a patient population appropriate for a different, off-label condition. Studies have shown prescriptions given for off-label use can be as high as 21%, with a majority of those drugs being anti-seizure or cardiovascular medications.2 In fact, as the study authors noted, most off-label drug use has little or no scientific support.
Consider the case of verapamil, a commonly prescribed calcium channel blocker approved to treat angina, arrhythmia, and hypertension. Currently, verapamil is also prescribed off-label to treat migraines3 and, more controversially, as topical and injected treatment in the penis for resolving symptoms of Peyronie’s disease.4 Recent research further indicates that off-label drug use of verapamil may be successful in treating a number of other conditions — the key word here is “may.” And that’s where the pharmacist’s role gets complicated.
The off-label use of verapamil to treat Peyronie’s disease is a perfect case study that illustrates the dilemma pharmacists face when a drug is prescribed without sufficient research supporting its use in treating a particular condition. Verapamil is able to increase the rate at which fibroblasts remodel after a scar or lesion is reformed, enhance the growth of urothelial cells, decrease collagen production by urothelial and smooth muscle cells, and change cytokine expression in early phases of wound healing.5 So far, these effects have only been studied in small populations without a placebo group or in in vitro samples. However, many physicians still recommend using oral and/or injected verapamil, sometimes in conjunction with another drug, to resolve the penile scar tissue caused by Peyronie’s disease.6 Without controlled studies to evaluate safety and efficacy of this off-label drug use, how can pharmacists do their due diligence before treating their patients?
How Can Pharmacists Offer the Best Care for Patients Needing Off-Label Drugs?
The answer to this question is not clear. By examining crises and lawsuits that have arisen from off-label drug use, one study recommended that all pharmacists be given the following information for every off-label prescription:
- A definition of the off-label drug.
- The drug class and patient populations in which the off-label drug is used most often.
- A description of the risks and benefits of using the off-label drug.
- An explanation of the role the FDA plays in regulating promotion of the drug’s off-label use.
- Recognition of the pharmacist’s potentially increased counseling role.
The problem is that it remains unclear who should provide this information. One option is to require physicians to write out the rationale behind their off-label prescriptions, but that still doesn’t allow pharmacists to assess the off-label use of drugs independently. The burden alternatively could be placed on the drug manufacturer, but that would involve relaxing restrictions on manufacturer promotion of off-label drug use. Insurers can also be tasked with providing this information. As it stands now, though, the job falls to the pharmacist, and it can be very onerous to keep track of potential off-label use for a whole dispensary of medications.
Perhaps the best solution is to turn to a national electronic database that interested parties must use to justify off-label drug use. Currently, the most comprehensive information regarding what an approved drug is used for and why it’s used comes from periodic surveys of office-based physicians. However, one research group has developed a data mining algorithm that searches through electronic medical records to detect off-label drug use, measure risk and cost, and also identify novel uses for approved drugs that need further investigation.7 Using such a tool proactively, with the data kept in one central location, would be a powerful tool for pharmacists to review before they evaluate an off-label prescription.
Calls to regulate off-label drug use are becoming louder from patients, pharmacists, and lawmakers who are hearing persistent complaints about irregular medical care. As physicians increasingly switch from paper files to electronic records, the ability to add a tag for off-label drug use becomes more appealing. Records of substantial off-label use of a particular medication can allow other physicians and pharmacists to become aware of the treatment’s success rate, risks, and side effects in that patient population.8 Pharmaceutical manufacturers can use these data to maintain required pharmacovigilance information. Furthermore, looking at trends in off-label prescriptions can inspire other healthcare providers and researchers to investigate novel uses of an old drug, as well as can help insurers re-evaluate what drug should be covered for which condition in their policies. Patient care works best when information is shared and when all stakeholders involved with patient healthcare work together. In the end, everyone benefits.
Off-label drug use can provide patients much-needed treatment, but it should be done in a careful, studied manner. Pharmaceutica North America is committed to providing safe and high-quality compounding materials that can be used to offer the right treatment for each patient. Learn more by contacting us today.
- “ASHP Statement on the Use of Medications for Unlabeled Uses, http://www.ashp.org/DocLibrary/BestPractices/FormStUnlabeled.aspx ↩
- “Off-Label Drug Use and Promotion,” October 1, 2014, http://www.uspharmacist.com/continuing_education/ceviewtest/lessonid/110671/ ↩
- “Medication Profile – Verapamil Used Off Label for Migraine,” March 7, 2012, http://www.healthcentral.com/migraine/c/202/150945/medication-profile/ ↩
- “’A Critical Analysis of Nonsurgical Treatment of Peyronie’s Disease,” June 1, 2006, http://www.europeanurology.com/article/S0302-2838(06)00304-6/fulltext/a-critical-analysis-of-nonsurgical-treatment-of-peyronie-s-disease ↩
- “Intralesional verapamil for the treatment of Peyronie’s disease: a review,” October 2002, http://www.nature.com/ijir/journal/v14/n5/full/3900917a.html ↩
- “Efficacy of Intralesional Verapamil Injections for the Treatment of Severe Peyronie’s Disease,” http://www.issmsmsna2012.org/present/_pdf/395.pdf ↩
- “Automated Detection of Off-Label Drug Use,” February 19, 2009, http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3929699/ ↩
- “Monitoring the safety of medicines used off-label,” 2009, http://www.pharmaco-vigilance.eu/content/monitoring-safety-medicines-used-label ↩