FDA Guidance on HCEI and Communications to Payors of Unapproved Products and Uses

Holland & Knight LLP

Holland & Knight LLP

On June 12, 2018, the U.S. Food and Drug Administration (FDA) released draft guidance entitled "Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities - Questions and Answers" (Guidance). The Guidance pertains to communications by medical product manufacturers, packers, distributors, and their representatives (Manufacturers) to payors1 (i) of approved drugs and approved/cleared devices (Approved Products) health care economic information (HCEI); and (ii) of unapproved drugs/devices (Unapproved Products) and unapproved uses of approved/cleared medical products (Unapproved Uses) information.

The stated goal of the Guidance is to ensure that the information on which Payors base their "coverage and reimbursement decisions . . . [is] truthful and non-misleading and that appropriate background and contextual information be provided to enable Payors to make informed decision[s]," as such decisions will have a far reaching impact on patients.

It is important to note that this Guidance only covers communication by Manufacturers to certain Payors. This Guidance does not address communications from Manufacturers to non-Payors, including physicians and patients, which as FDA notes, has been addressed in previous guidance.2 This Guidance is also limited to the communication of HCEI of Approved Products, defined to be any analysis that identifies, measures, or describes the economic consequences of the use of a particular drug, and communication of information of Unapproved Products and Uses.

Communication of HCEI Information Regarding Approved Products and Approved Uses

The FDA sets forth a two-pronged approach to communication of Approved Product HCEI. The threshold question is whether or not the HCEI relates to an FDA approved use.

The Guidance explains that HCEI relates to an approved use when the information "relate[s] to the disease or condition, the manifestation of the disease or condition, or symptoms associated with the disease or condition in the patient population for which the drug is indicated in the FDA-approved labeling."

If the HCEI relates to an FDA approved use, then the FDA will assess whether such HCEI includes material differences from the FDA approved labeling. If the HCEI does not include material differences, then so long as the Manufacturer provides certain background and contextual information, the FDA will not consider such communication false or misleading.

If, when compared to the FDA-approved labeling, the HCEI includes material differences, then such Manufacturers will need to include "a conspicuous and prominent statement describing any material differences between the health care economic information and the labeling approved for the drug." As one would expect, material differences include factors that affect patient safety, such as new or increased risks, different dosing/use regimens, different endpoints, more-limited/targeted patient populations.

Communication of Unapproved Product or Unapproved Use Information

The more interesting (and arguably more important) guidance relates to how FDA will treat communications with respect to Unapproved Products and Unapproved Uses. The Guidance outlines two general recommendations that Manufacturers should follow regarding communications relating to Unapproved Products and Unapproved Uses.

First, the Guidance explains that when information about unapproved products or unapproved uses is "unbiased, factual, accurate and non-misleading," FDA will not object, provided the Manufacturer follows the agency’s recommendations to include the following information in any communications.

  • "A clear statement that the product or use is not approved/cleared/licensed, and that the safety or effectiveness of the product or use has not been established;"
  • "Information related to the stage of product development (e.g., whether a marketing application for the product or new use has been submitted to FDA or when such a submission is planned);" and
  • "For communications that include factual presentations of results from studies . . . [Manufacturers] describe material aspects of study design and methodology . . . and results. [Manufacturers] should ensure that results are not selectively presented (e.g., both positive and negative findings should be presented)."

In addition, for Unapproved Uses, Manufacturers should further provide:

  • "A prominent statement disclosing the indication(s) for which FDA has approved, cleared, or licensed the product and a copy of the most current FDA-required labeling."

By including the above information in the Guidance, FDA has, in effect, provided Manufacturers with a "safe harbor" as to information about unapproved products and unapproved uses when communicating with Payors. In reconciling its current communications guidance with positions the agency has taken in previous guidance3 and litigation4, FDA explained in the Guidance that while it remains concerned that off-label communications may "potentially undermine substantial governmental interests related to health and safety . . . [including] motivating the development of robust scientific data on safety and efficacy . . . in order to prevent harm, protect against fraud, misrepresentation, and bias . . ." it recognizes that (1) accurate, non-misleading and balanced information regarding unapproved products and uses is needed for Payors to plan, budget and make informed coverage and reimbursement decisions; and (2) Payors represent a "sophisticated audience with established procedures to carefully consider the full range of relevant evidence about new uses of medical products" so that the risk that Payors will be misled "is relatively low." Consistent with its prior guidance and positions taken in litigation, FDA specifically noted that "communications to other audiences" (i.e., physicians and patient-consumers) "about unapproved products or unapproved uses . . . could raise additional or different considerations that are beyond the scope of this guidance" (footnote omitted).

The Guidance represents a significant and positive shift in FDA’s thinking with respect to a Manufacturer’s ability to communicate information regarding unapproved products and unapproved uses to a discrete audience — Payors. It is unlikely, however, in the absence of litigation, that FDA will go any further.


1 The FDA defines payors as to include third-party payors, health plan sponsors, pharmacy benefits managers, and state Medicaid programs, formulary committees, or other similar entities with knowledge and expertise in the area of health care economic analysis (Payors).

2 U.S. Food & Drug Admin., Draft Guidance for Industry on Responding to Unsolicited Requests for Off-Label Information About Prescription Drugs and Medical Devices (2011); U.S. Food & Drug Admin., Draft Guidance for Industry Distributing Scientific and Medical Publications on Unapproved New Uses – Recommended Practices (2014).

3 See FDA Guidance, supra note 2.

4 Amarin Pharma, Inc. v. FDA, 119 F. Supp. 3d 196 (S.D.N.Y. 2015); see also United States, v. Caronia, 703 F.3d 149 (2d Cir. 2012).

DISCLAIMER: Because of the generality of this update, the information provided herein may not be applicable in all situations and should not be acted upon without specific legal advice based on particular situations.

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