By Gregory Stephens, Windrose Partners, and Sheila Campbell, PhD, RD01.19.21
The Food and Drug Administration (FDA) approves drugs for certain health conditions. When physicians prescribe drugs for an off-label use, they are prescribing it for a different condition or at a different dosage than what the FDA has approved. Off-label use is not unique to the pharmaceutical industry. Medical foods, which are intended for the management of a specific disease or medical condition, are often used for other health issues. Additionally, dietary supplements, which are by FDA standards to be used by healthy individuals, may be recommended by healthcare professionals to assist in management of a disease.
Common Practice
It is important to understand that FDA regulates drug approval, not drug prescribing, and doctors are free to prescribe to patients drugs they feel are medically appropriate. Actually, the practice is legal and very common. More than one in five outpatient prescriptions written in the U.S. are for off-label therapies. A physician can, however, get into trouble if he or she falls below appropriate standards of care. Since the FDA does not regulate doctors (the states do that), it simply cannot prevent off-label use.
For most dietary supplements, adequate clinical substantiation does not exist to qualify them for FDA-approved health claims. Thus, supplements are relegated to the use of structure-function claims. Despite all the attention given to proper use of these claims and other labeling requirements, it is common knowledge (supported by survey data) that many dietary supplements are being taken to address specific medical conditions.
The COVID Factor
The coronavirus pandemic has actually increased off-label uses for Rx medications and dietary supplements. Sweeping around the world, the virus reached the most remote and isolated regions by the end of 2020, generating a tremendous scientific effort to find effective treatment, interventions and vaccines to inhibit its spread.
Given how disastrous and frightening COVID-19 is, it is not surprising that reports of unproven cures and remedies have proliferated. Products have been inappropriately touted for use against the virus or for unfounded off-label claims—for instance, ingested bleach and hydroxychloroquine. On the other hand, some off-label claims are not completely outlandish, such as the claim that lactoferrin, an antiviral and immune modulator, may protect against coronavirus.
Lactoferrin is a protein found in high concentrations in human breast milk, and as a niche product is typically marketed as an immune booster for babies. But in Italy last summer, after a video went viral that reported the results of a small study that suggested lactoferrin might protect against coronavirus, Italian pharmacies saw product sales spike as much as 50%. Most pharmacists were surprised by the claim, but one commented, “When people are scared, they will believe anything.” Another said, “When someone buys [lactoferrin] in bulk, you know it’s because of COVID.” Subsequently, one firm branded lactoferrin as “CovAlt” and incorporated it into a variety of virus-related products such as hand gel. (“Italians snap up baby-milk supplements after virus claim,” Joseph Boyle, Giuliana Ricozzi. AFP. Yahoo! News, October 2020.)
Off Label Case Studies
Our careers with the Nutritional Division of Abbott Laboratories ended long before the coronavirus pandemic reached our shores. But a recent conversation about the virus and potential and purported cures kicked off a discussion about off-label uses of nutritional products. The first product that came to mind was Pedialyte.
A water and electrolyte solution, Pedialyte was first marketed for children who experience vomiting and diarrhea, causing dehydration and electrolyte-depletion. It became clear that the product could help adults similarly afflicted. Its marketing reach broadened accordingly. And in 2015, the Wall Street Journal reported that adult use of Pedialyte had grown 60% over the previous three years. Of particular interest was the improbable and huge sales increase in Mexico, where the rehydration product was not heavily marketed.
Upon more in-depth exploration it was determined that this growth was due to the fact that adults had discovered that Pedialyte could ease the symptoms of a particular adult malady, the hangover. Interestingly, when the marketing team proposed a strategy to enhance this “off-label” use, wise leaders suggested a hands-off approach—an early example of viral marketing.
So, until recently, adults simply consumed the kids’ product to quell the aftermath of a night on the town. Years later they now they have a Pedialyte product labeled of their own need—Sparkling Rush. This very adult-sounding product is an effervescent electrolyte powder that was launched in 2019, just in time to ease all the coronavirus lockdown hangovers. (“Pedialyte Finally Made a Beverage Just for Adults,” Sarah Rense. Jan 2, 2019. Esquire.)
The flagship adult nutritional product from Abbott is Ensure, a “complete, balanced” nutritional beverage. Ensure has been used for many off-label purposes. Many are not surprising such as for weight loss and as an alternative to bicarbonate to reduce gastric pH. Ensure Plus, the product with more calories and protein per serving, is used by athletes as a source of concentrated nutrition and protein to protect and build muscle mass. As with the rehydration solution, this new use spawned a line extension branded Ensure Max with higher protein levels for the sports nutrition market.
Companion Cases
But some uses may be surprising. Ensure has been used to feed many species of non-human animals—dogs, cats, horses, primates, and others—that need nutritional support. At this point in our conversation, we turned to an expert on non-human consumers of Abbott products. Debbie Rooney, PhD, has a degree in animal science. During our tenure at Abbott she was the point person who fielded questions about whether, for instance, a giraffe could drink Ensure, or whether a lion cub could benefit from Pedialyte. She told us several mostly happy stories.
Seaworld asked if any of Abbott’s nutritional products could be used to feed an ailing pilot whale. She counseled that Pulmocare, a high-fat, low-carbohydrate product for people with COPD, was best suited for meeting the sea mammal’s nutritional needs. The handlers ultimately used a concoction of Pulmocare and pureed fish as a tube feeding product for the animal.
A hurricane hit a primate facility in Florida, causing severe stress to several young cotton-top tamarins. The tiny monkeys became dehydrated and weren’t thriving. Following Rooney’s advice, the facility fed the tamarins Vital HN, a low-fat highly digested (elemental) product formulated for people with a compromised GI tract. The monkeys’ condition quickly stabilized and they began to eat normally.
Expected Growth
Make no mistake, off-label drug use can put people at risk of receiving ineffective or even harmful treatment. Though the majority of dietary supplements have a higher safety profile than pharmaceuticals, it is prudent to suggest that healthcare professionals only recommend supplements “off-label” when they have sound evidence of the product efficacy and safety for the patient population. There are clearly benefits to off-label product usage and with the expanding base of clinical research supporting nutritional interventions, we feel the practice will grow accordingly.
Gregory Stephens
Windrose Partners
Greg Stephens, RD, is president of Windrose Partners, a company serving clients in the the dietary supplement, functional food and natural product industries. Formerly vice president of strategic consulting with The Natural Marketing Institute (NMI) and Vice President of Sales and Marketing for Nurture, Inc (OatVantage), he has 25 years of specialized expertise in the nutritional and pharmaceutical industries. His prior experience includes a progressive series of senior management positions with Abbott Nutrition (Ross Products Division of Abbott Laboratories), including development of global nutrition strategies for disease-specific growth platforms and business development for Abbott’s medical foods portfolio. He can be reached at 267-432-2696; E-mail: gregstephens@windrosepartners.com.
Sheila Campbell, PhD, RD
Sheila Campbell, PhD, RD, has practiced in the field of clinical nutrition for more than 30 years, including 17 years with Ross Products Division of Abbott Laboratories. She has authored more than 70 publications on scientific, clinical and medical topics and has presented 60 domestic and international lectures on health-related topics. She can be reached at smcampbellphdrd@gmail.com.
Common Practice
It is important to understand that FDA regulates drug approval, not drug prescribing, and doctors are free to prescribe to patients drugs they feel are medically appropriate. Actually, the practice is legal and very common. More than one in five outpatient prescriptions written in the U.S. are for off-label therapies. A physician can, however, get into trouble if he or she falls below appropriate standards of care. Since the FDA does not regulate doctors (the states do that), it simply cannot prevent off-label use.
For most dietary supplements, adequate clinical substantiation does not exist to qualify them for FDA-approved health claims. Thus, supplements are relegated to the use of structure-function claims. Despite all the attention given to proper use of these claims and other labeling requirements, it is common knowledge (supported by survey data) that many dietary supplements are being taken to address specific medical conditions.
The COVID Factor
The coronavirus pandemic has actually increased off-label uses for Rx medications and dietary supplements. Sweeping around the world, the virus reached the most remote and isolated regions by the end of 2020, generating a tremendous scientific effort to find effective treatment, interventions and vaccines to inhibit its spread.
Given how disastrous and frightening COVID-19 is, it is not surprising that reports of unproven cures and remedies have proliferated. Products have been inappropriately touted for use against the virus or for unfounded off-label claims—for instance, ingested bleach and hydroxychloroquine. On the other hand, some off-label claims are not completely outlandish, such as the claim that lactoferrin, an antiviral and immune modulator, may protect against coronavirus.
Lactoferrin is a protein found in high concentrations in human breast milk, and as a niche product is typically marketed as an immune booster for babies. But in Italy last summer, after a video went viral that reported the results of a small study that suggested lactoferrin might protect against coronavirus, Italian pharmacies saw product sales spike as much as 50%. Most pharmacists were surprised by the claim, but one commented, “When people are scared, they will believe anything.” Another said, “When someone buys [lactoferrin] in bulk, you know it’s because of COVID.” Subsequently, one firm branded lactoferrin as “CovAlt” and incorporated it into a variety of virus-related products such as hand gel. (“Italians snap up baby-milk supplements after virus claim,” Joseph Boyle, Giuliana Ricozzi. AFP. Yahoo! News, October 2020.)
Off Label Case Studies
Our careers with the Nutritional Division of Abbott Laboratories ended long before the coronavirus pandemic reached our shores. But a recent conversation about the virus and potential and purported cures kicked off a discussion about off-label uses of nutritional products. The first product that came to mind was Pedialyte.
A water and electrolyte solution, Pedialyte was first marketed for children who experience vomiting and diarrhea, causing dehydration and electrolyte-depletion. It became clear that the product could help adults similarly afflicted. Its marketing reach broadened accordingly. And in 2015, the Wall Street Journal reported that adult use of Pedialyte had grown 60% over the previous three years. Of particular interest was the improbable and huge sales increase in Mexico, where the rehydration product was not heavily marketed.
Upon more in-depth exploration it was determined that this growth was due to the fact that adults had discovered that Pedialyte could ease the symptoms of a particular adult malady, the hangover. Interestingly, when the marketing team proposed a strategy to enhance this “off-label” use, wise leaders suggested a hands-off approach—an early example of viral marketing.
So, until recently, adults simply consumed the kids’ product to quell the aftermath of a night on the town. Years later they now they have a Pedialyte product labeled of their own need—Sparkling Rush. This very adult-sounding product is an effervescent electrolyte powder that was launched in 2019, just in time to ease all the coronavirus lockdown hangovers. (“Pedialyte Finally Made a Beverage Just for Adults,” Sarah Rense. Jan 2, 2019. Esquire.)
The flagship adult nutritional product from Abbott is Ensure, a “complete, balanced” nutritional beverage. Ensure has been used for many off-label purposes. Many are not surprising such as for weight loss and as an alternative to bicarbonate to reduce gastric pH. Ensure Plus, the product with more calories and protein per serving, is used by athletes as a source of concentrated nutrition and protein to protect and build muscle mass. As with the rehydration solution, this new use spawned a line extension branded Ensure Max with higher protein levels for the sports nutrition market.
Companion Cases
But some uses may be surprising. Ensure has been used to feed many species of non-human animals—dogs, cats, horses, primates, and others—that need nutritional support. At this point in our conversation, we turned to an expert on non-human consumers of Abbott products. Debbie Rooney, PhD, has a degree in animal science. During our tenure at Abbott she was the point person who fielded questions about whether, for instance, a giraffe could drink Ensure, or whether a lion cub could benefit from Pedialyte. She told us several mostly happy stories.
Seaworld asked if any of Abbott’s nutritional products could be used to feed an ailing pilot whale. She counseled that Pulmocare, a high-fat, low-carbohydrate product for people with COPD, was best suited for meeting the sea mammal’s nutritional needs. The handlers ultimately used a concoction of Pulmocare and pureed fish as a tube feeding product for the animal.
A hurricane hit a primate facility in Florida, causing severe stress to several young cotton-top tamarins. The tiny monkeys became dehydrated and weren’t thriving. Following Rooney’s advice, the facility fed the tamarins Vital HN, a low-fat highly digested (elemental) product formulated for people with a compromised GI tract. The monkeys’ condition quickly stabilized and they began to eat normally.
Expected Growth
Make no mistake, off-label drug use can put people at risk of receiving ineffective or even harmful treatment. Though the majority of dietary supplements have a higher safety profile than pharmaceuticals, it is prudent to suggest that healthcare professionals only recommend supplements “off-label” when they have sound evidence of the product efficacy and safety for the patient population. There are clearly benefits to off-label product usage and with the expanding base of clinical research supporting nutritional interventions, we feel the practice will grow accordingly.
Gregory Stephens
Windrose Partners
Greg Stephens, RD, is president of Windrose Partners, a company serving clients in the the dietary supplement, functional food and natural product industries. Formerly vice president of strategic consulting with The Natural Marketing Institute (NMI) and Vice President of Sales and Marketing for Nurture, Inc (OatVantage), he has 25 years of specialized expertise in the nutritional and pharmaceutical industries. His prior experience includes a progressive series of senior management positions with Abbott Nutrition (Ross Products Division of Abbott Laboratories), including development of global nutrition strategies for disease-specific growth platforms and business development for Abbott’s medical foods portfolio. He can be reached at 267-432-2696; E-mail: gregstephens@windrosepartners.com.
Sheila Campbell, PhD, RD
Sheila Campbell, PhD, RD, has practiced in the field of clinical nutrition for more than 30 years, including 17 years with Ross Products Division of Abbott Laboratories. She has authored more than 70 publications on scientific, clinical and medical topics and has presented 60 domestic and international lectures on health-related topics. She can be reached at smcampbellphdrd@gmail.com.
