Managing the Risks of Off-Label Dermatological Medications

In this episode, Steve Adubato, PhD, speaks with Asha Patel Shah, MD, MBA, FAAD, a board-certified dermatologist and a member of the medical faculty at MagMutual. Dr. Shah discusses a variety of medication-related topics in dermatology, including off-label medication use and its associated risks for patients and physicians. Dr. Shah shares her insights on the use of off-label medications, including the informed consent process, medical record documentation and payer reimbursement, and more. She also speaks about best practices for prescribing medications and communicating patient responsibilities.

Release Date: 8/2/2023; Recorded Date: 3/7/2023

Please note: This podcast was originally a production of MDAdvantage, which is now part of the MagMutual family of companies.

Podcast Transcript

ADUBATO:  Welcome to the MDAdvantage podcast. I'm Steve Adubato. MDAdvantage is proud to now be a part of the MagMutual family. MDAdvantage physicians and their practices will continue to receive exceptional protection and support, now backed by the strength and stability of MagMutual. We're honored to be joined by Dr. Asha Patel Shah, a board certified dermatologist and member of the medical faculty at MagMutual Insurance Company. Dr. Shah, it’s great to have you with us.

SHAH:  Thank you. Thank you, Steve, it's great to be here. 

ADUBATO:  Doctor, tell us a little bit about your background and also your experience more specifically as a dermatologist. 

SHAH:  Sure. Thanks for this opportunity, and, yes, of course I'm happy to briefly introduce myself. I graduated in a combined BS/MD program at the University of Miami in Miami, FL, and because of the combined aspect of that program, I had an opportunity to explore my research interests in between my third and fourth year of medical school. I spent some time at the University of Miami as a research fellow. Then I went on to the National Institutes of Health in Bethesda, MD, as a clinical research fellow in the dermatology branch, which was in the National Cancer Institutes at that time. Subsequently, I went on to match and then graduate from Columbia University's dermatology residency program as chief resident, and then moved from New York City to Atlanta, GA, for practice, which is where I currently still am today. 

I am a board-certified dermatologist by the American Board of Dermatology and I am currently an adjunct assistant professor of dermatology at Emory University in the Department of Dermatology. My clinical experiences in the last decade of practice span a health maintenance organization, private practice, locum tenens in underserved areas, academic practice and telehealth in the commercial and government sectors.

ADUBATO:  It’s really helpful for us to understand your very extensive background. Talk a little bit about your role as a member of the medical faculty at MagMutual.

SHAH:  My role is primarily helping with claims and, specifically, dermatology claims, and producing educational content for the learning center.

ADUBATO:  Let's talk about some of these claims, particularly in the area of off-label medication. What exactly are we talking about? What are we referring to when we talk about the risks and considerations of off-label use of medication? And, right off the bat, what is off label?

SHAH:  I think it's first important to define what FDA approved means, as the definition of off-label use generally comes from an FDA-approved drug for an unapproved use. So, for a drug to become FDA approved, the pharmaceutical company must submit pharmacology, clinical and safety data for the FDA to review. The company must show that the drug is safe and effective for its intended use, however, safe does not mean that the drug has no side effects. Instead, it means that the FDA has determined the benefits outweigh the potential risks of using the drug for a specific disease indication. Ultimately, the approved drug labeling is very specific to the dosage of treatment and the disease or diseases the drug is meant to treat.  

From the FDA regulatory point of view, a physician may prescribe a drug for an unapproved use when they deem it's medically appropriate for a specific patient. That is what we call off-label use. Examples of off-label use would be: 1) if there are no approved drugs to treat the disease or medical condition; 2) all approved treatments have been tried without seeing any benefit to the patient; or 3) there are medical contraindications to the approved treatments out there and the off-label usage of another medication would be the best option for the patient, in your medical decision-making. Other examples of off-label use include things like if you alter the dose on the labeling, such as using an adult medication for a pediatric case, or using a different vehicle to deliver the medication, which we often do in dermatology, such as compounding a topical cream from the contents of an oral capsule. 

ADUBATO:  I'm curious about this, and I know that everyone listening to this podcast would be curious as well. Cite the most specific factors that a healthcare provider should consider when evaluating the potential risk associated with off-label prescriptions. What specifically are those factors?

SHAH:  Great question. So most importantly, off the bat, is safety. Safety is the biggest factor.  Clinical trial safety data is derived from disease-specific indications, and if you'd like to prescribe off label, you must ensure that the benefits would outweigh the risks in your patient. Each clinical scenario is really unique to that specific encounter, and bringing the patient into this discussion and presenting them with all the options and insight into your medical decision process really helps make the physician/patient relationship into more of a true partnership. This will hopefully increase the compliance on the patient's part. 

Another factor that keeps coming up these days is cost. Many, if not all, health insurance companies heavily regulate and abide by their formularies with what is FDA approved only.  What I’m also finding out is that even for FDA-approved indications, there's still an arduous prior authorization in place for many standard medications. To try to get a payer to cover an off-label use medication can be a near impossible challenge, and thus these open communications with the patients on the front end, and frankly just getting the patient to be their own advocate, may help move the process along. If all else fails, there are out-of-pocket or cash payment options for medications, but that tends to be incredibly expensive and generally a non-starter for most patients. 

ADUBATO:  This is Steve Adubato with the MDAdvantage podcast with our colleagues and friends at MagMutual. We're joined by Dr. Shah, who is a board-certified dermatologist who serves on the medical faculty at MagMutual.  Dr. Shah, I want to follow up on this question. So you mentioned at MagMutual that you deal with medical liability claims.  Be specific about some of the most common and relevant claims you've seen.  

SHAH:  They are mostly unexpected outcomes, and can stem from things like off-label use. In terms of examples, there have been quite a few, but one that comes to mind that was quite devastating to the patient involved a board-certified dermatologist and hyaluronic acid gel injections. The injections were done to a combination of on-label sites, such as the nasolabial folds, but also an off-label site was done at the same time, which was the glabella (the area between the brows). Unfortunately, the next day there was pain and redness at the glabella site, and the patient was diagnosed with vascular occlusion, which is a medical emergency. Subsequently, the patient was put on an appropriate treatment regimen of hyaluronidase, vasodilators and antibiotics, but eventually required seven hyperbaric oxygen treatments to help with all the skin necrosis. Ultimately, when this case was reviewed, it was deemed that the consent was not thorough enough for the patient to make an informed decision to treat the off label site.

STEVE:  You know, it's interesting, you just mentioned informed consent. How does the informed consent process differ with off-label medications?  

SHAH:  So I can only really speak to dermatology-specific cases and, frankly, this is almost a daily occurrence within our field. If you think about how resource intensive a quality clinical trial can be, it's not feasible for the pharmaceutical companies to do this for every single dermatologic indication, especially if it's for an established medication that has been around for decades, even preceding the existence of these FDA regulations. Thus, dermatologists have become very comfortable treating off label, because there's just simply no choices for some of the diseases we treat.

Luckily, there has been a big boom in the last five years for diseases like atopic dermatitis, alopecia areata and vitiligo, but some of these newer medications are still out of financial reach for most patients. So off-label prescribing is still very much thriving in dermatology. 

The informed consent process is nearly always verbal with the patient, and generally we review the rationale of the decision. First, I'll discuss what the drug is actually approved for; then I'll get into counseling if there are any other drugs or therapies that are approved for the disease. But then I have to explain the rationale behind why those options won't work for this current situation. So then I go into reviewing any sort of evidence I might have to support the off-label use I'm presenting to them, like smaller anecdotal case reports experienced from colleagues or presented at a medical meeting - things I can show or print for them. Then I'll discuss if the off-label use is expected to reach a similar treatment outcome compared to those that are approved, so I can level their expectations. This is especially important nowadays because patients are bombarded with advertisements and social media, and they may expect different outcomes.  

As part of the traditional informed consent, of course, also review expected side effects, contraindications, any drug/drug interactions currently or anything that might be expected down the road. And then I'll counsel regarding required lab monitoring and follow-up visit intervals to keep the patient safely on the medication. It's very important that we're on the same page. Finally, I'll go briefly into the cost portion of the medication. Of course I'm not on the pharmacy side. I have no idea what's going to happen on that side, but I just want to warn the patient that there might be a prior authorization or a cash pay option, so there is less frustration once they leave my office. So, as you can tell, this is a super lengthy conversation, and it just needs to be documented in the plan portion of the note, not only to cover myself but to also document that the patient was an equal partner in all of this, and that they understood the decision making and that they had full awareness of their responsibilities.  

Lastly, I wanted to mention that I've actually heard of a few fellow dermatologists creating written contracts for patients to sign, which is essentially like a written informed consent for certain off-label use medications. I've not actually had to do that kind of consent in my years of practice, but I imagine for certain scenarios and in certain types of patients, it would actually be a really great document to have handy for medical-legal peace of mind. 

ADUBATO:  Final question on my end, Dr. Shah. What advice do you have for physicians, some of whom are calling in new prescriptions over the phone, versus putting it in writing?  

SHAH:  In my humble opinion, calling in a new prescription is not ideal, and pharmacy staff nationwide is pushing back on this practice. So if you think about this logically and even logistically, there are so many audio-technical issues that can happen with leaving a new prescription on a pharmacy's voice mail, which is generally what we're forced to do because the pharmacies are short staffed and can't take your call in real time. In addition, calling in a new prescription this way may not be properly documented in the written record, and thus that just leaves more error to grow. If a medication must be called in, it's best to actually get a live pharmacy staff person on the phone, verbally relay the information back and forth, make sure both parties have understood the details correctly, and then the next step would be for that person who called in the prescription to document this as a pharmacy call-in, like a telephone encounter, in the chart, and then on top of that they have to add the new medication to the patient's active medication list. So as you can already tell, this is way too many steps in the process, and, of course, the more steps you add in any workflow, the more room for error. Ideally, we live in the electronic age, and nearly everything has an electronic trail. It is just optimal to use your electronic health record to e-prescribe, so documentation is present in real time, and then the patient's medication list is also auto-populated. 

If you're still paper charting, of course that's okay, but copies of paper prescriptions, telephone notes and pharmacy calls would be really important for appropriate record keeping. The reason this is just very important is because there are a lot of claims right now with wrong dosages, wrong drug names, wrong amount dispensed, due to miscommunication between the office and the pharmacy. Getting into a better documentation practice and workflow rhythm between you and your staff is really important to protect yourself. 

ADUBATO:  Dr. Shah, I cannot thank you enough for providing such important valuable information on the podcast. Thank you so much. 

SHAH:  Thank you, it is my pleasure. 

ADUBATO:  On behalf of the teams at MDAdvantage and MagMutual, this has been Steve Adubato. You've been listening to Dr. Shah talk about a whole range of important issues. We thank you so much for listening. Check us out next time.