An assessment of the proposed rule to change US FDA’s interpretation of Intended Use

The US Food and Drug Administration published a proposed rule in September 2020 to update the language used to define intended use in relationship to both medical devices and drugs. This should be carefully reviewed by manufacturers, and action is recommended.

Overview of Intended Use

Traditionally, the intended use of a medical device or drug has been considered the general purpose of the product or its function, including the relevant medical disease or condition for which the product is intended to be used, and has been evaluated by FDA through the review of proposed labeling for products which require a premarket submission.

Recent history of FDA’s description of Intended Use

Starting in 2015, the FDA began an effort to update the language describing intended use to expand the understanding from specific labeling to include additional sources of evidence. They issued a proposed rule in 2015 and a final rule in 2017 to make the intended use inclusive of additional information. In both cases, there were significant comments from industry, leading to FDA delaying the effective date related to the intended use description indefinitely, as previously reported by Emergo by UL.. 

One of the primary industry concerns was that the language suggested that off-label use would be considered part of the intended use of the product. Off-label use happens widely and, in some cases, is even considered the standard of care. Off-label use of drugs represents approximately 50-75% of pediatric medication use and off-label use of medicinal products for rare diseases is estimated to be up to 90%.

The 2020 proposed rule language has been modified from previous versions in an attempt to address industry concerns, and is:

“…the objective intent of the persons legally responsible for the labeling… show by such persons’ expressions, the design or composition of the article, or the circumstances surrounding the distribution of the article. The objective intent may, for example, be shown by labeling claims, advertising matter, or oral or written statements by such persons or their representatives… [or] by circumstances in which the article is, with the knowledge of such persons or their representatives, offered or used for a purpose for which it is neither labeled nor advertised; provided, however, that a firm would not be regarded as intending an unapproved new use… based solely on that firm’s knowledge that such drug was being prescribed or used by health care providers for such use…”

The 2020 proposed rule provides improved clarity from the 2015 and 2017 rules. However, there are still significant questions related to the language of “solely.”

Application of the 2020 proposed rule regarding Intended Use

The FDA states that updating the traditional interpretation of intended use is an important priority for the FDA. Manufacturers of medical devices and drugs need to prepare themselves for implementation of this rule.

Emergo recommends the following actions for manufacturers:

1. Review your current labeling, processes, and practices to determine if this interpretation could suggest that any off-label use would be considered by the FDA and/or courts of law to be part of the intended use of the product.
2. If any concerns are discovered, implement a plan to address these concerns in a timely manner.
3. Consider commenting on the FDA proposed rule. Comments are actively considered by the FDA and led to changes to the 2015 and 2017 interpretations, each providing improvements from the previous version. By partnering with the FDA, we can address concerns on both sides to provide true clarity for all involved.

Sarah Fitzgerald is Senior Consultant, Quality and Regulatory Affairs at Emergo by UL.

Learn more about US FDA medical device regulations at Emergo by UL:

• US FDA medical device and IVD regulatory strategy
• US FDA medical device classification consulting
• Whitepaper: Preparing a US FDA 510(k) submission