As we recently reported, in the wake of court rulings and settlements addressing the First Amendment in the context of providing truthful, non-misleading speech, including about unapproved (or, “off-label”) use of approved or cleared medical products, on September 1, 2016, the FDA issued a notice that it will hold a public hearing on November 9 and 10 concerning firms’ communications about such uses. FDA’s notice also follows the release of a set of joint principles endorsed by the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO), known as Principles on Responsible Sharing of Truthful and Non-Misleading Information About Medicines with Health Care Professionals and Payers (“Principles”). Although it is unclear whether or when FDA might provide guidance on its views regarding permissible truthful communications regarding off-label uses of approved or cleared medical products, statements and guidance from industry associations, such as the Principles proposed by and PhRMA and BIO, provide valuable insight to device manufacturers about the topics that are under review. The industry statements may also assist the FDA in formulating new guidelines that take into account First Amendment principles outlined in recent court decisions and settlements.
In their Principles, PhRMA and BIO articulate broadly-applicable concepts “intended to form the basis for defining new and clear regulatory standards governing responsible, truthful and non-misleading communications.” Key concepts on which the Principles rely include:
- Commitment to Science-based Communication: The principles seek to increase access to information based on scientific and statistically sound data.
- Commitment to Provide Appropriate Context about Data: Appropriate context should be provided to ensure health care professionals do not draw inaccurate conclusions about information concerning the safety and effectiveness of medicines.
- Commitment to Accurate Representation of Data: The sophistication of the intended audience of communications should be taken into account when communicating about data, which includes disclosing limitations on the data and the methodologies used.
The Principles articulate these concepts more specifically and then go further to discuss sample fact patterns as examples of responsible sharing of truthful and non-misleading information. The Principles also identify several specific categories of data that could be addressed in new FDA guidance, including:
- Data from randomized, controlled clinical trials, including those completed after the drug is approved and not included in the product label;
- Post hoc analysis, including sub-population data not analyzed as part of the initial label;
- Observation data and real-world evidence, including from claims data, electronic medical records, and patient registries; and
- Pharmaeconomic information obtained from clinical trials, observational studies, reviews of medical record databases and other predictive modeling techniques.
The upcoming FDA hearing is designed to “inform FDA’s policy development” and “provide a productive framework for discussing how to modernize the standards so that companies can engage in communications more productively with doctors, patients and the reimbursement community.” The Principles may foreshadow the types of information that FDA would address in updated guidance to the industry. For example, the Principles’ recognition that communications about off-label use should be tailored to the intended audience seems to be something that both industry and FDA can agree is worthwhile. In addition, FDA may seek to address some of the specific types of data that are outlined in the Principles, such as certain data from FDA-regulated randomized, controlled clinical trials that are not included within the product label.
Electronic or written comments on the topic of communications regarding unapproved uses may be submitted to FDA until January 9, 2017.