Off-label use is an important and often misunderstood area of FDA regulation. Healthcare professionals may prescribe or use an FDA-approved or cleared medical product for an indication, patient population, dosage, route, or other condition that is not included in its approved labeling. However, the ability of a healthcare professional to use a product off-label does not automatically give a manufacturer the right to promote that use. For pharmaceutical, biotechnology, medical device, and clinical research organizations, understanding the distinction between lawful scientific communication and impermissible promotional activity is essential to maintaining FDA compliance. The FDA’s regulatory framework is built around the principle that manufacturers generally cannot promote an approved or cleared product for an unapproved or uncleared use as though that use were authorized by the Agency. FDA explains that its premarket review is intended to evaluate the safety and effectiveness of a product for particular intended uses. Promoting an unapproved use can undermine these safeguards because the company may be communicating claims that have not undergone the appropriate FDA review.

At the same time, the regulatory landscape is more nuanced than a simple prohibition on discussing scientific information. FDA has established specific frameworks for certain communications concerning off-label information, including communications that are truthful, balanced, non-misleading, and appropriately presented. In January 2025, FDA finalized guidance addressing certain firm-initiated communications to healthcare providers involving scientific information on unapproved uses of approved or cleared medical products. The guidance describes an enforcement policy for qualifying communications and therefore represents an important development for companies developing compliant medical communications strategies. One critical distinction is between off-label use and off-label promotion. A physician’s independent decision to prescribe an approved drug for an unapproved indication is different from a manufacturer’s promotional claim encouraging that use. Companies should therefore establish clear controls governing sales representatives, medical affairs personnel, websites, social media, conference materials, scientific presentations, customer communications, and other channels that could influence product use. Statements made by a manufacturer can potentially contribute to evidence of a new intended use, depending on the facts and circumstances.

FDA’s 2018 guidance on communications consistent with FDA-required labeling also provides an important compliance framework. FDA explains that certain information may not appear in approved labeling but can nevertheless be consistent with the approved or cleared uses of a product. The Agency evaluates such communications using a fact-specific approach and states that it does not intend to rely on a communication that is consistent with FDA-required labeling, by itself, to establish a new intended use. However, other applicable requirements governing advertising, labeling, and promotional communications still apply. For companies communicating scientific information concerning an unapproved use of an approved drug or medical device, documentation and content controls are particularly important. Organizations should be able to demonstrate the scientific basis for statements, accurately characterize limitations, avoid selective presentation of evidence, and distinguish scientific discussion from promotional messaging. Medical and regulatory review should occur before distribution where appropriate, with documented approval pathways for promotional and scientific materials. The compliance risk extends beyond traditional sales representatives. Digital marketing has created additional challenges because websites, webinars, social media posts, search advertisements, email campaigns, downloadable materials, and online videos can all communicate product claims. Companies should therefore evaluate the intended audience, context, wording, supporting evidence, risk information, and overall presentation of every communication. FDA’s Office of Prescription Drug Promotion emphasizes that prescription drug promotion must be truthful, balanced, and not misleading, regardless of the communication platform.

For medical device manufacturers, the same underlying concern applies, although the specific statutory and regulatory framework differs from prescription drug promotion. Claims about a device’s intended use, indications, patient population, performance, or clinical application should be carefully assessed against the device’s FDA authorization and labeling. A manufacturer should not assume that the existence of published scientific literature or clinical experience independently establishes regulatory permission to market a new intended use. The 2025 FDA guidance is particularly relevant for organizations seeking to communicate legitimate scientific information to healthcare providers without turning that communication into impermissible promotion. Companies should assess whether a proposed communication falls within an applicable FDA enforcement policy and ensure that the communication satisfies all stated conditions. The guidance does not create a general authorization to promote every off-label use; rather, it describes a specific policy framework for qualifying communications.

Strong FDA compliance therefore requires a risk-based communication program. Organizations should maintain clear distinctions between medical affairs and commercial functions where appropriate, train employees on off-label promotion, implement medical-legal-regulatory review procedures, maintain evidence files for substantive claims, and periodically audit external communications. These controls can reduce the risk of regulatory action, misleading promotion allegations, reputational damage, and potential patient-safety concerns. For pharmaceutical, biotech, medical device, and clinical research organizations, the practical objective should not simply be to determine whether an off-label discussion is technically possible. The more important question is whether the communication is scientifically accurate, appropriately contextualized, supported by credible evidence, directed to an appropriate audience, and consistent with the specific FDA requirements and enforcement policies that apply. Careful interpretation of FDA off-label regulations, promotional labeling requirements, and scientific communication policies is essential before distributing materials.