Industry for years has sought clarity on off-label communications. Numerous meetings on the topic have taken place between the U.S. Food and Drug Administration (FDA) and industry, citizen petitions have been filed, legislation has been introduced in Congress and a series of legal cases have been brought, culminating in the consternation around this topic. Just this week, the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) teamed up on the release of new guidelines for how biopharmaceutical companies communicate information about their products. Overall, the stated intention of the guidelines and their nine Principles is to “form the basis for defining new and clear regulatory standards governing responsible, truthful and non-misleading communications to inform healthcare professionals about the safe and effective use of medicines.”
The Principles included in the guidelines appear to be a response to FDA’s perceived inability to deliver timely guidance in this area. In fact, in the ninth Principle, FDA is scolded for “certain of [its] recommended practices [that] would restrict truthful and non-misleading communication with health care professionals and ultimately risk delaying the provision of timely, educational, and accurate information to healthcare professionals about certain unapproved uses, many of which are medically accepted and indeed even the standard of care for certain diseases.” The guidelines go on to suggest a roadmap for how FDA should modify its Good Reprint Practices Guidance.
PhRMA and BIO urge increased access to product information, arguing that the biopharmaceutical companies “are uniquely positioned to help health care professionals achieve the best outcomes for patients, because companies can provide timely, accurate, and comprehensive information about both approved and unapproved uses of [their] medications.” In fact, PhRMA and BIO assert that providing the information, as they propose, would equip healthcare professionals to make better treatment decisions and insurance coverage, which ultimately result in better patient care.
Our ten key takeaways are that the Principles:
1. Apply primarily to data and information outside of FDA- approved labeling, both information not contained in the approved label and alternative (unapproved) uses.
2. Are grounded in the belief that these communications can “benefit patient care and enhance the efficiency of our health care system.”
3. Reinforce that the product’s FDA-approved labeling is the primary source of information for communications with healthcare professionals about approved medications.
4. Apply only to communications with healthcare professionals and payers (not consumers).
5. Cover only communications subject to FDA’s oversight, not exempt scientific communications.
6. Make clear that presentation of scientifically-sound information in a truthful and non-misleading manner is what matters, not the identity of the party communicating the information.
7. Include the following Key Concepts:
- Commitment to Science-based Communication- Communications should be based on “analyses using scientifically- and statistically-sound methodologies.” The guidelines recognize, in Appendix A, several analyses that can improve patient care and improve the efficiency of the health care system, including: randomized, controlled clinical trials; post-hoc analyses, including sub-population data; observational data and real-world evidence; and pharmacoeconomic information.
- Commitment to Provide Appropriate Context about Data- Appropriate context about the data should be included in communications to enable the healthcare professional or payer to “to prevent healthcare professionals and payers from reaching inaccurate conclusions or forming misimpressions about the efficacy or safety of a medicine.”
- Commitment to Accurate Representation of Data- When considering what limitations to include in communications, the focus should be “on ensuring that data are represented accurately, which includes disclosing limitations of the data and the scientific and analytical methodologies used.”
8. Direct that companies should include disclosures in oral, as well as written, communications with healthcare professionals and payers.
9. Recognize the unique informational needs of health care organizations and how pharmacoeconomic data and real-world evidence can help payers and health systems better understand a medicine’s cost and expected benefits for patients.
10. Provide fifteen very detailed scenarios illustrating application of the Principles.
As those who work in this field know all too well, “the devil is in the detail” when it is time to apply principles like these. Each company’s situation will be unique and consultation with regulatory and legal advisors will be important should a company elect to share this kind of communication. The Principles note that several factors can affect the disclosures required to ensure that a communication is made in a truthful and non-misleading manner. If PhRMA and BIO are correct, companies who implement these guidelines effectively have an opportunity to positively affect patient care.
This article was co-authored by Lee Tumminello and Emily Puchalski