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Beyond the Eleventh Hour: FDA Prepares to Finalize Intended Use Amendments Despite Midnight Rule Relief Act

On Monday, FDA issued a final rule to amend FDA’s established definitions of “intended use” for drugs and devices, the primary consideration in determining whether a product is regulated for a particular use and what regulations apply. The final rule also excludes products derived from tobacco from regulation as “tobacco products” if such products are intended for use as a drug, device, or combination product.  (We'll be discussing the tobacco products portion of the rule in a separate post.)  While this rule could have a profound effect on marketing schemes for tobacco products, drugs, and medical devices, Congress’s passage of the Midnight Rules Relief Act could eliminate the rule before it ever takes effect.

The House of Representatives recently passed the Midnight Rules Relief Act of 2017 largely along party lines. If passed by the Senate and signed into law by President Trump, the bill would amend the Congressional Review Act (CRA) to allow Congress to group multiple regulations together and disapprove, thus overturn, them en bloc. While the CRA only requires a simple majority to pass, current law requires Congress to review regulatory resolutions of disapproval one at a time. The regulations in danger of being overturned under the proposed bill would be ones submitted for congressional review within the last 60 legislative days, which in an election year covers regulations as far back as May 2016.

The House’s quick action to position itself for a potential regulatory overhaul comes on the heels of a report by the House Freedom Caucus calling for over 200 regulations to be repealed by the Congressional Review Act (CRA). House Republicans are eager to roll back regulations en masse and that enthusiasm should be taken seriously. However, the Senate 60-vote threshold remains a hurdle to passage and Senate Majority Leader Mitch McConnell has stayed quiet on this particular legislation.

House Republicans have decried other tobacco regulations in the past year, so we cannot rule out this rule getting extra scrutiny having been finalized in the last two weeks of the Obama Administration. Additionally, incoming Secretary of the Department for Health and Human Services Tom Price (R-GA) has opposed regulating tobacco in the past, and may support walking back this regulation so that it can be written under the new leadership of a new administration.

Congress has a lot on its plate in 2017 and it’s no certainty that it will move swiftly on disapproving large groups of regulations. That said, the House has sent early signals that it is determined to expedite the roll back process, which should begin soon after President Trump takes office.

Changes to Drug & Device Intended Use Definitions

When the proposed rule was published on September 25, 2015, it appeared that FDA was attempting to slip in a major regulatory change to the “intended use” definition applicable to drugs and devices without explanation. FDA proposed deleting the following language from 21 C.F.R. §§ 201.128, 801.4: “But if a manufacturer knows, or has knowledge of facts that would give him notice, that a drug introduced into interstate commerce by him is to be used for conditions, purposes, or uses other than the ones for which he offers it, he is required to provide adequate labeling for such a drug which accords with such other uses to which the article is to be put.”  The only explanation offered was that the “clarifying change” would “conform them regulations to how the Agency currently applies the regulations.”

The proposed amendment resulted in a number of comments asserting that, by deleting the language, FDA was relinquishing its ability to consider knowledge of off-label uses as part of an intended use determination. So, in response, FDA opted to amend the final sentence of both regulations rather than simply delete it.  The amended regulations clarify that FDA will determine intended use based on the “totality of evidence,” but not on knowledge that the product is being prescribed or used for an unapproved use (see Section IV.B-C of the preamble to the final rule).

Commenters also took the rule-making as an opportunity to attack FDA on its general policies related to the determination of intended use and off-label use of regulated products. FDA vigorously defended its established positions and provided substantive interpretations of recent court cases (such as United States v. Caronia, 703 F.3d 149 (2d Cir. 2012), and Amarin Pharma, Inc. v. FDA, 119 F. Supp. 3d 196 (S.D.N.Y. 2015)).  At the same time, FDA deflected criticism of its stance on off-label communications by citing its separate efforts to explore the issue (see our prior post on FDA’s off-label communication workshop).

Amendments Are a Win for Industry

Ultimately, the final rule snuffs out any enthusiasm that FDA had given up a significant factor affecting intended use decisions. But industry can still count the amendments as a win because they remove a major regulatory enforcement weapon that FDA has wielded against the drug and device industries since 1976.  The language of the “knowledge” provision of the intended use regulations gives the Agency freedom to punish any manufacturer for even knowing about off-label uses.  In contrast to situations in which a manufacturer affirmatively promotes a regulated product for an off-label use, FDA has only rarely invoked the “knowledge” provision as the basis of an enforcement action.

The revisions to 21 C.F.R. §§ 201.128, 801.4 provide much needed clarification of FDA’s intended use doctrine and will give drug and device manufacturers some protection against discretionary enforcement actions based on off-label uses.  Under the Midnight Rules Relief Act, Congress may eliminate this protection before it becomes effective, so industry stakeholders should consider working with their congressional representatives to preserve this final rule.

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Benjamin advises pharmaceutical, medical device and biotech companies on the FDA regulatory process to identify the correct regulatory pathway, assisting with FDA communications and strategy.