Last week, the U.S. Food and Drug Administration (“FDA”) released two guidance documents that relate to sharing of certain information about drugs and medical devices, including economic information about unapproved products and unapproved uses, as well as information not included in a product’s FDA-approved labeling. These guidance documents are part of broader blueprint published by the Department of Health and Human Services that aims to promote value-based models, with the aim of controlling rising drug spending and reducing the out-of-pocket cost burden for patients.
Guidance on payor communications
The first guidance details FDA’s recommendations for how manufacturers can share health care economic information (“HCEI”) under Section 114 of the Food and Drug Administration Modernization Act of 1997 (“FDAMA”). According to a statement by FDA Commissioner Scott Gottlieb, the goal of this guidance is to promote “innovative, value-based payment arrangements” by allowing manufacturers to “communicate information about how a drug might impact outcomes that are important to purchasers like a health plan or hospital, but is not an endpoint that is expressly described in the drug’s approved labeling.”
In particular, the guidance addresses how manufacturers can share HCEI with public and private sector payors, formulary committees, drug information centers, technology assessment committees, pharmacy benefit managers, third party administrators, and other entities that make drug or device selection or coverage/reimbursement decisions. Although the guidance maintains FDA’s position that communication of HCEI is considered “promotional labeling,” the guidance announces that FDA will not take enforcement actions against manufacturers for certain pre-approval communications that otherwise would violate FDA regulations.
Examples of the types of information manufacturers may now share under the guidance include:
- Analyses based on patient subgroups that differ from those described on the FDA-approved labeling
- Analyses based on dosing or use regimens that differ from a product’s labeling
- Analyses based on surrogate or intermediate endpoints
- Analyses derived from studies comparing the safety or effectiveness of a drug for its approved indication to another drug or intervention or to no treatment
The new guidance also requires manufacturers to include sufficient background and contextual information along with HCEI, as well as a clear statement regarding whether the product or use is approved or cleared by FDA.
The guidance explicitly does not apply to communications with healthcare providers (“HCPs”) making individual patient prescribing decisions or to patients, although the guidance could apply to HCPs who have multiple roles, such as an HCP who serves on a formulary committee.
Guidance on CFL promotional communications
The second piece of guidance explains how manufacturers can communicate information that is consistent with FDA-approved labeling (“CFL,” in FDA parlance) but that is not actually contained in the FDA-required labeling itself. The guidance announces the FDA will exercise enforcement discretion for communications that meet the requirements of the guidance, and FDA also will not consider such communications to establish a new intended use for a product. In particular, FDA will apply three factors in determining whether a communication is consistent with FDA-required labeling:
- How the information in the product communication compares to the information about conditions of use in the FDA-required labeling, in particular, whether the communication conflicts with the label’s information about indications, patient population, limitations and directions for handling/use, or dosing or use regimen/administration
- Whether the representations/suggestions about use of the product in the product communication increase the potential for harm to health relative to the information reflected in the FDA-required labeling
- Whether the directions for use in the FDA-required labeling enable the product to be safely and effectively used under the conditions represented/suggested in the product communication
FDA provided several examples of the type of information that might not appear on a product’s label or labeling but that could be considered CFL under the guidance, including:
- A comparison of the safety or efficacy of a medical product for its approved indication to another medical product approved for the same indication
- Contextual information about adverse reactions associated with a product, for example, if the drug’s label lists nausea as a potential adverse reaction and that the drug can be taken with or without food, a CFL communication might provide information about how taking the drug with food might reduce nausea
Examples of the types of information that would not be considered CFL include:
- Information about the use of a product to treat or diagnose a different disease or condition than the product is approved to treat or diagnose
- Information about the use of a product to treat a different stage, severity, or manifestation of a disease than the product is approved to treat
- Information about the use of a product in a different dosage form than what is set forth in the FDA-required labeling
* Special thanks to Vanisha Weatherspoon for her assistance with this post.