Four Ways FDA’s New Intended Use Regulations Affect Drug and Medical Device Companies | Knowledge


The final rule seeks to amend the consolidated regulatory text in important respects. The final rule changes the language of the regulation in an extremely consequent way: it abandons the general rule that the intended use means “objective intention”, which “is determined by” the “expressions” of the manufacturer. Under the revised regulations, intended use continues to mean “objective intention”, but such intention now “”may be demonstrated ”by such expressions and many other types of evidence. As a result, the manufacturer’s promotional claims – “phrases” – are no longer the backbone of intended use determinations.

According to the preamble accompanying the Final Rule, promotional claims are not necessary to establish a new intended use for a legally marketed product and the FDA “may seek to all relevant source of evidence, including a variety of direct and circumstantial evidence.3 The FDA does not discuss (or even recognize) all of the final rule’s specific changes to codified regulations, but rather asserts that the action only aligns the regulations so that they “better reflect” “current practices.” From the agency.4

Under the amended regulations, FDA asserts that many activities long considered protected, such as responding to unsolicited requests, are no longer categorically protected. Safeguards of the ability of manufacturers to share information about off-label uses in appropriate circumstances have been well established in law and in FDA policy for decades and have been repeatedly affirmed by the agency in litigation briefs and other statements. The final preamble to the rule identifies five ‘safe harbor’ guidance documents describing the circumstances under which manufacturers are permitted to release information that is not directly derived from product labeling, including information on product labeling. “off-label” uses:

  • Communications in accordance with FDA-approved labeling
  • Communications with payers
  • Scientific and educational activities supported by industry
  • Responses to spontaneous requests (draft)
  • Dissemination of scientific and medical publications (scientific or medical journal articles, scientific or medical reference texts and clinical practice guides) (draft)5

Although the preamble asserts that nothing in the Final Rule reflects a change in these policies,6 it also indicates that FDA can point to secure communications as evidence of a new intended use. The preamble states that by virtue of FDAapproach to intended use, these types of business communications “usually would not, by them selves», Establish a company’s intention that a legally marketed medical product be used for unapproved purposes.7 But these communications would be relevant to the manufacturer that a medical product is used off-label.

The situation is even more complex with regard to responses to spontaneous requests and the dissemination of scientific and medical publications. Guidance documents relating to these communication categories are in draft form and do not reflect the FDA Politics. Indeed, FDA recognizes the different status of communications undertaken on the basis of a draft safe harbor directive, noting that a directive only reflects the agency’s current policy once it has been finalized.8

The final rule does not explicitly exclude reliance on knowledge as evidence of a new intended use. FDA began procedures to change the regulations in 2015, in response to an industry petition asking FDA to delete the terms ostensibly defining the intended use by reference to the knowledge by a manufacturer that its drug or medical device was used off the label.9 Under the Final Rule, a company would not be considered to have the intention of an unapproved new use “solely on the basis of that company’s knowledge that such a drug was prescribed or used by health care providers. health care for such use ”. According to FDA, the word “only” is used to mean that FDA does not intend to take into account a company’s knowledge of off-label use, by himself, as sufficient to establish the intended use.ten This means that a company’s knowledge of the off-label use can be used to establish a new intended use even if FDA concluded that knowledge alone is insufficient. Thus, the “relevant sources” of evidence “May include”Knowledge of a company that health care supplier has used or prescribed the company’s medical product for an unapproved use.11 Therefore, while mere knowledge of the off-label use, and no more, is not sufficient to establish a new intended use, according to the final rule, such knowledge is relevant to intended use determinations. .

The final rule reduces the level of clarity in the regulatory and enforcement regime. The preamble asserts that the final rule is intended to provide “clarity and direction” regarding the types of evidence relevant to determining the intended uses of a product.12 In effect, the Final Rule purports to transform the regulatory definition of intended use from an objective standard based primarily on claims into a much broader and potentially without standard concept based on an unexplained notion of “relevance”. According to the codified text, “intended use” means “objective intention”, which “can be demonstrated” by (1) “expressions”, such as “labeling claims, advertising or oral statements or written “, (2) the product” design or composition “, (3)” the circumstances surrounding … the distribution “, or (4) the fact that a manufacturer knew that his product was” offered or used for any purpose for which it is neither labeled nor advertised. ” FDA states that “determining the intended use of a product is a precise factual investigation” in which “FDA can consider all relevant sources of evidence», Which are not limited to the sources identified in the consolidated text.13 Beyond “relevance”, the preamble does not illuminate a guiding principle applicable to intended use determinations. The preamble specifies rather than fulfilling its mission of protecting public health, FDA assess the “individual and unique circumstances of each case” to determine the intended use of a product.14 The preamble further notes that in some cases “a single piece of evidence may be determinative”, while in others “more than one evidence combined may establish the intended use of a product”.15

The final rule purports to change the fundamental regulatory definitions in important ways that are not adequately explained or even recognized by the corresponding preamble. Manufacturers of drugs and medical devices should not rely on standard regulatory watch procedures to assess the implications of the final rule on operational impacts. Instead, a cross-functional leadership team representing the core risk management functions of the organization, as well as the appropriate contribution from medical and business functions, should consider the degree to which FDAapproach to intended use potentially affects non-promotional communications of the type which historically have been considered categorically admissible when conducted in accordance with the recommendations set out in FDA guidance documents and other policy statements. Companies that have relied on the status of refuge for specific activities to inform about risks analyzes should review these assessments for the final rule.


1 86 Fed. Reg. 41 383 (August 2, 2021), codified in 21 CFR §§ 201.128 and 801.4.

2 21 USC § 352 (f) (1). See also username. §§ 355 (a), 351 (f) (1) (B), 352 (o).

3 86 Fed. Reg. at 41 386, 41 388. The preamble accompanying the final rule specifically notes that the knowledge component of 21 CFR §§ 201.128 and 801.4 has been deleted and that these regulations now include a provision according to which “a company would not be considered as intending for a new, unapproved use … based solely on this company’s knowledge of off-label use.

4 86 Fed. Reg. at 41,383, 41,384; see also username. to 41 390 (“[T]The intended use regulatory changes are intended to describe the types of evidence relevant to determining the intended use of a product based on current FDA practice ”). The preamble indicates that the only change made by the rule is the deletion of the wording defining the intended use only with reference to the manufacturer’s knowledge of the use outside Marketing Authorization. Username. at 41,391 (citing 82 Fed. Reg. 2,193, 2,207 (2017) (final rule withdrawn)). In addition, argues the FDA, the final rule would impose no costs, username. to 41 384, but would allow “reducing … uncertainty” among regulated entities, username. at 41,400.

5 86 Fed. Reg. at 41,396 (citing 85 Fed. Reg. 59 718, 59 725)).

6 86 Fed. Reg. at 41,393.

7 Username(emphasis added); see also username. at 41,396 (the final rule “does not reflect a change in FDA policies and practices regarding types of corporate communications that would not on their own normally establish a new intended use”).

8 The FDA did not directly ask whether activities undertaken pursuant to a proposed guideline will enjoy the same (albeit limited) protection as those undertaken pursuant to a final guideline (see 85 Fed. Reg. At 59,723 n.7 (even describing the advice project as “recognized”).

9 80 Fed. Reg. 57,756 (September 25, 2015); Citizen petition from the working group on medical information, file n ° FDA-2013-P-1079 (September 3, 2013).

ten 86 Fed. Reg. at 41,397 (emphasis in original).

11 Username. (emphasis added); see also username. (“Although the use by the health care provider is not under the control of the company, what may be relevant to the intended use is the knowledge by the company that the item is being used by the health care provider ”).

12 Username. at 41,384.

13 Username. at 41,397 (emphasis added); see also 82 Fed. Reg. at 2,207 (listing additional “relevant” sources of evidence, such as “consumer intent” and “evidence of claims that have never been disclosed to the public”).

14 86 Fed. Reg. at 41,387 n.3; see also 85 Fed. Reg. at 59,724 (idem).

15 86 Fed. Reg. at 41,387 n.3; see also username. at 41,397 (“Each scenario described in the preamble is fact-specific and, in other circumstances or contexts, similar elements may be assessed differently”); 85 Fed. Reg. at 59,724 (“Each scenario is fact-specific and, in other circumstances or contexts, similar items may be assessed differently”).


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