Greg Stephens, RD, & Sheila Campbell, PhD, RD, Windrose Partners09.08.15
Medical foods are products specifically formulated and intended for the dietary management of a disorder, disease or condition having distinctive nutritional needs not met by modification of a regular diet alone. A key marketing benefit of medical foods is their ability to refer to the “nutritional or dietary management of a specific disease or its metabolic processes” on the product label. Though medical foods do not require FDA pre-market approval or registration, there are specific criteria to qualify. FDA oversight and enforcement has escalated over the past two years.
Global sales for medical foods have been estimated to exceed $9 billion. This includes similar regulatory categories outside the U.S. (e.g., foods for special medical purposes). The U.S. market for medical foods easily exceeds $2 billion. The category is experiencing significant growth and the number of new product launches is escalating. During the past 5 years there has been an unprecedented number of medical food launches, primarily from companies entering the category.
Recently, the medical food market has seen surprising growth in the health practitioner channel. Most medical foods entering this channel are not “legacy” products (e.g., Pulmocare, Abbott Nutrition; Vivonex, Nestlé Health Science) but rather are new formulations launched by start-up companies and companies established in the practitioner channel. Category growth is driven by an increasing number of practitioners, including allopathic physicians, recommending nutritional interventions.
Disease management claims are appealing to dietary supplement companies and a compelling reason many choose to venture into the category. Substantiating these claims, however, differs significantly from substantiating structure-function claims.
For example, an intriguing and compelling story can certainly create interest in new products. These benefits are often voiced by celebrity spokespersons. We all appreciate the talents of celebrities like Gwyneth Paltrow, Neil Young and Jim Carrey. However, when they publically state their opinions about food, nutrition and health as scientific fact, their statements are not relevant in supporting medical food claims. This is because often times such claims do not provide the scientific substantiation FDA requires; therefore, they are often viewed by the agency as pseudoscience, as they are lacking fact and scientific evidence. Of course, it is not just celebrities making claims. Social and public media abounds with pseudoscience. Though psuedoscience may be effective in marketing, FDA will not accept it as support for medical food claims.
FDA Guidance About Medical Food Product Claims
According to Sandy Bigelow, PhD, principal, Vanguard Global Associates LLC, in the U.S., FDA regulates product claims made on product labeling. Product labeling includes product labels as well as other materials accompanying and supporting the sale of products. These include brochures, shelf talkers and other materials where claims are made. The Federal Trade Commission (FTC) regulates claims made in advertising materials.
FDA requires that claims, statements and representations about products must be truthful, not misleading and substantiated for dietary supplements and for foods generally (21 U.S.C. 343). Claims must reveal facts material to the representations made in context with the claims (21 U.S.C. 321(n)). Claims for medical foods cover those about the product’s capability to affect structure and function of the human body, and as such, are excluded from claims made for drugs (21 U.S.C. 321(g)). Therefore, these structure-function claims must not include claims to cure or treat disease. Only drugs are allowed to make such statements.
Medical food claims must be substantiated. The quality and quantity of evidence supporting product claims must match the nature and specificity of the claim.
FDA Guidance on Evidence Required to Substantiate Claims
It may surprise you to learn that FDA leaves it to the manufacturer to determine how to generate evidence to support product claims. However, bear in mind, the agency expects that generally accepted scientific and statistical principles are used to determine the quality of the evidentiary studies used to substantiate a claim.
Further, claims should be supported by results of medical evaluation (i.e., clinical research). This evidence must be based on recognized scientific principles (e.g., appropriate study design using appropriate clinical measures and statistical analysis, credible and accurate data interpretation and the reliability and reproducibility of findings).
The “gold standard” for claims support is the prospective, randomized, controlled clinical trial (PRCT). However, trials of this type may not always be possible, practical or ethical. Other evidence, including in vitro and animal studies, as well as expert consensus opinion, may be helpful to augment the clinical studies used to support claims.
Guidance About Evidence Required to Substantiate Claims
The strongest evidence comes from systematic reviews, meta-analyses and PRCT (see Figure 1). Descriptive, observational reports such as case studies and anecdotal reports provide weaker evidence because their design is not controlled and data are not generalizable to broader populations. Expert opinion provides the weakest support.
Systematic reviews are reviews of the scientific literature focused on a particular question (e.g., “Do omega-3 fatty acids effectively treat heart disease?”). The goal of such a review is to identify, appraise, select and interpret all high quality research evidence relevant to that question. The operative phrase is “high quality research evidence.” If you are using systematic reviews to support claims, you must be sure the studies used in the review are the result of high quality scientific research.
A meta-analysis uses statistics to combine and analyze data from two or more independent PRCTs. Usually the PRCTs used in a meta-analysis come from the results of a systematic review. As with a systematic review, the validity of the meta-analysis depends on the quality of the studies included in the systematic review on which it is based.
PRCTs are considered the gold-standard of study design. PRCT study design controls bias by randomly assigning subjects to receive the intervention or control treatment, and observed outcomes are due to the intervention under study rather than being due to chance.
In comparison, descriptive (versus analytical) reports and expert opinion provide the weakest evidence to support claims. The results of these reports cannot be analyzed statistically and are not designed to eliminate bias. However, if you have numerous case reports and expert opinion, their combined weight may be useful.
When creating your dossier of evidence, you need to evaluate the studies you plan to use by answering the following questions for each study and report:
Medical foods do not require pre-market approval from FDA. However, it is critical to bear in mind, the agency has developed clear guidelines regarding substantiation of claims. Following these guidelines, with consultation from qualified experts if necessary, can eliminate bumps down the road, and support a successful product launch.
Gregory Stephens, RD
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 215-860-5186; 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.
Global sales for medical foods have been estimated to exceed $9 billion. This includes similar regulatory categories outside the U.S. (e.g., foods for special medical purposes). The U.S. market for medical foods easily exceeds $2 billion. The category is experiencing significant growth and the number of new product launches is escalating. During the past 5 years there has been an unprecedented number of medical food launches, primarily from companies entering the category.
Recently, the medical food market has seen surprising growth in the health practitioner channel. Most medical foods entering this channel are not “legacy” products (e.g., Pulmocare, Abbott Nutrition; Vivonex, Nestlé Health Science) but rather are new formulations launched by start-up companies and companies established in the practitioner channel. Category growth is driven by an increasing number of practitioners, including allopathic physicians, recommending nutritional interventions.
Disease management claims are appealing to dietary supplement companies and a compelling reason many choose to venture into the category. Substantiating these claims, however, differs significantly from substantiating structure-function claims.
For example, an intriguing and compelling story can certainly create interest in new products. These benefits are often voiced by celebrity spokespersons. We all appreciate the talents of celebrities like Gwyneth Paltrow, Neil Young and Jim Carrey. However, when they publically state their opinions about food, nutrition and health as scientific fact, their statements are not relevant in supporting medical food claims. This is because often times such claims do not provide the scientific substantiation FDA requires; therefore, they are often viewed by the agency as pseudoscience, as they are lacking fact and scientific evidence. Of course, it is not just celebrities making claims. Social and public media abounds with pseudoscience. Though psuedoscience may be effective in marketing, FDA will not accept it as support for medical food claims.
FDA Guidance About Medical Food Product Claims
According to Sandy Bigelow, PhD, principal, Vanguard Global Associates LLC, in the U.S., FDA regulates product claims made on product labeling. Product labeling includes product labels as well as other materials accompanying and supporting the sale of products. These include brochures, shelf talkers and other materials where claims are made. The Federal Trade Commission (FTC) regulates claims made in advertising materials.
FDA requires that claims, statements and representations about products must be truthful, not misleading and substantiated for dietary supplements and for foods generally (21 U.S.C. 343). Claims must reveal facts material to the representations made in context with the claims (21 U.S.C. 321(n)). Claims for medical foods cover those about the product’s capability to affect structure and function of the human body, and as such, are excluded from claims made for drugs (21 U.S.C. 321(g)). Therefore, these structure-function claims must not include claims to cure or treat disease. Only drugs are allowed to make such statements.
Medical food claims must be substantiated. The quality and quantity of evidence supporting product claims must match the nature and specificity of the claim.
FDA Guidance on Evidence Required to Substantiate Claims
It may surprise you to learn that FDA leaves it to the manufacturer to determine how to generate evidence to support product claims. However, bear in mind, the agency expects that generally accepted scientific and statistical principles are used to determine the quality of the evidentiary studies used to substantiate a claim.
Further, claims should be supported by results of medical evaluation (i.e., clinical research). This evidence must be based on recognized scientific principles (e.g., appropriate study design using appropriate clinical measures and statistical analysis, credible and accurate data interpretation and the reliability and reproducibility of findings).
The “gold standard” for claims support is the prospective, randomized, controlled clinical trial (PRCT). However, trials of this type may not always be possible, practical or ethical. Other evidence, including in vitro and animal studies, as well as expert consensus opinion, may be helpful to augment the clinical studies used to support claims.
Guidance About Evidence Required to Substantiate Claims
The strongest evidence comes from systematic reviews, meta-analyses and PRCT (see Figure 1). Descriptive, observational reports such as case studies and anecdotal reports provide weaker evidence because their design is not controlled and data are not generalizable to broader populations. Expert opinion provides the weakest support.
Systematic reviews are reviews of the scientific literature focused on a particular question (e.g., “Do omega-3 fatty acids effectively treat heart disease?”). The goal of such a review is to identify, appraise, select and interpret all high quality research evidence relevant to that question. The operative phrase is “high quality research evidence.” If you are using systematic reviews to support claims, you must be sure the studies used in the review are the result of high quality scientific research.
A meta-analysis uses statistics to combine and analyze data from two or more independent PRCTs. Usually the PRCTs used in a meta-analysis come from the results of a systematic review. As with a systematic review, the validity of the meta-analysis depends on the quality of the studies included in the systematic review on which it is based.
PRCTs are considered the gold-standard of study design. PRCT study design controls bias by randomly assigning subjects to receive the intervention or control treatment, and observed outcomes are due to the intervention under study rather than being due to chance.
In comparison, descriptive (versus analytical) reports and expert opinion provide the weakest evidence to support claims. The results of these reports cannot be analyzed statistically and are not designed to eliminate bias. However, if you have numerous case reports and expert opinion, their combined weight may be useful.
When creating your dossier of evidence, you need to evaluate the studies you plan to use by answering the following questions for each study and report:
- Is the research question clearly and succinctly stated so it applies to your product and desired claims?
- Are appropriate endpoints and their measures clearly and appropriately defined?
- Is the study correctly designed to address the research question and to control for bias?
- Can the study findings be generalized to your population of interest?
- Are the statistical tests appropriate to the study design?
- Are the statistical results adequately analyzed and interpreted, and any potential threats to validity and reliability identified and explained?
- Are the conclusions drawn supported by the data?
Medical foods do not require pre-market approval from FDA. However, it is critical to bear in mind, the agency has developed clear guidelines regarding substantiation of claims. Following these guidelines, with consultation from qualified experts if necessary, can eliminate bumps down the road, and support a successful product launch.
Gregory Stephens, RD
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 215-860-5186; 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.
