FDA advisory panel votes against approval of DMD drugFor years the U.S. Food and Drug Administration (FDA) has withstood the push to relax its prohibition on the off-label promotion of products not approved by the agency. However, it has also acknowledged that the country’s healthcare system is impacted by information that comes from manufacturers about their products—specifically, information that is not misleading or untruthful.

Recently, the FDA held a two-day public hearing for the purpose of information-gathering, an exploratory phase that will continue through early January.

The hearing provided a forum for stakeholders to share their thoughts on off-label regulations. Healthcare professionals, patients, caregivers, product manufacturers, advocacy groups, researchers, insurers and other payors expressed viewpoints for an FDA panel…and, taken as a whole, those viewpoints brought up issues in some key areas.

Precision medicine can benefit from off-label communications

The FDA panel heard from developers of screening devices and genetic tests as they discussed the role of new evidence and uses for their screening measures in medical approaches tailored toward a patient’s specific profile. They made the argument that this published information should be made available to healthcare practitioners in the course of treating patients so that clinicians have the broadest data to consider when making decisions.

Increased communication can protect patients within vulnerable populations

Physicians made the case that there are medications that effectively treat ophthalmologic conditions but are not specifically approved by the FDA for that particular use. The doctors argued that shutting down their access to research data could ultimately limit treatment options for certain patients.

A patient advocacy group illustrated its point through the example of pediatric cancer patients receiving off-label treatments with medical devices, drugs, and biologics since there are no FDA-approved treatments for pediatric use.

Although speakers at the hearing stressed the need for reliable manufacturer data for practitioners working with vulnerable patient populations, they also acknowledged the need for a method of attesting to the reliability of manufacturers’ statements.

Rules should differ according to circumstance

Stakeholders requested that the FDA outline the purview of each type of off-label communication and to elucidate those “safe harbors” with clarity and thoroughness. Trade groups and companies expressed the opinion that the agency should not approach off-label communication as if it were one-size-fits-all since all products do not incur the same level of risk from off-label use.

Payors and healthcare plans should have access to off-label information

The FDA was encouraged to develop a “safe harbor” that would allow product manufacturers to share information with payors during the point in time a product is in review, but prior to the products receiving official agency approval. Payors and manufacturers were represented at the hearing; the overarching message was that healthcare plans have highly effective means for data collection and protection already in place, and therefore would logically serve as sound recipients of off-label guidance.

Off-label communication regarding adverse events might serve to prevent future adverse events

One of the many questions asked and explored over the course of the two-day hearing: Is it possible that there would be fewer—or perhaps less severe—adverse events involved with off-label product use if practitioners had access to more information in these cases?

At the hearing’s commencement, FDA Commissioner Robert Califf acknowledged the fact that off-label uses usually involve more adverse events than approved uses for the same drug. Patients and advocacy groups voiced opposition to relaxing regulations for off-label communication, citing specific cases of injury or death resulting from unapproved product usage.

Other patient advocacy groups, however, made the case that off-label communication could broaden useful treatment choices, but urged the use of safety measures like informative literature and patient informed consent.

Stakeholders can send comments to the agency through early January

During this information-gathering phase, the FDA is open to written remarks on the topic of off-label regulatory approaches from stakeholders until January 9th.


For more information/questions regarding any legal matters, please email info@nelsonhardiman.com or call 310.203.2800.