5 Off-label Communication Hearing Themes

Attorneys Joanne Hawana and Benjamin Zegarelli (Mintz Levin) say that five recurring themes arose from a recent two-day FDA hearing on the appropriate regulatory approach to manufacturers’ communications about off-label uses for their marketed products. Writing in an online blog post, they summarize the themes as: 

•          Use different rules for different situations. Companies and trade associations said that the agency can’t use a “one size fits all” policy because not all communications are created equal and because not all products have the same risks associated with off-label uses.
•          Share off-label information with health plans and payors. Manufacturers, health plans, and payors urged FDA to create a safe harbor to permit product developers to share information with payors about their products before approval, while a new or supplemental application is under agency review. They said that patients can be harmed when insurers don’t have enough clinical data to make a coverage decision about the medically accepted off-label use for a product, because often the manufacturer is the best source of such clinical information.
•          Lack of drug development for vulnerable populations encourages off-label uses and increased communication would protect those patients. Thus, patient advocates said, pediatric cancer patients routinely receive off-label treatments with drugs, biologics, and medical devices because no cancer treatment is specifically approved by FDA for pediatric use. And doctors said that many drugs are useful in treating ophthalmological diseases and conditions, even though the agency has not approved such indications, and restricting the flow of scientific information to treating doctors would limit their ability to provide effective treatments.
•          Adverse events associated with off-label uses may be tempered with more off-label communications. Patients and some patient advocates strongly opposed any loosening of off-label communication rules, in part due to the increased safety risks with off-label uses. But other patient advocates said that off-label communication should be permitted to expand legitimate treatment options but with certain patient protections such as informed consent from the patient and the existence of sufficient literature supporting the off-label treatment’s safety and effectiveness.
•          Off-label communications are valuable in precision medicine. Growth of precision medicine could be directly affected by manufacturers’ ability to share information with other healthcare stakeholders.

The attorneys say it is not clear how FDA will proceed. It could do something incrementally, allowing some less controversial communications and then moving on to more complicated issues, or it could issue a draft guidance on how it will integrate First Amendment principles into its policies on manufacturer communications.