John Kamp: Industry committing to accuracy in sharing drug information
You may have missed it in the hottest days in late July, but you shouldn’t have.
The two leading biopharmaceutical organizations made a bold public statement on FDA off-label policy that proposes new regulatory standard that provides a great starting point for the inevitable policy debates on how the FDA defines “false and misleading” in marketing. The existing FDA standard, essentially declaring anything beyond the label per se false and misleading, is dead, or nearly so everywhere but in pockets of the FDA itself. That standard has been discarded by a series of federal court decisions, including from the United States Supreme Court, leading major industry watchers and the even Congressional leaders to urge the FDA to move forward to redefine the standard in a way that meets the mandates of the First Amendment and good patient care.
While everyone knows that major changes are inevitably coming in the FDA’s marketing standard, the questions are when and what will they look like. PhRMA and BIO jumped in the vacuum late last month to propose several new guidelines in a document called “Principles on Responsible Sharing of Truthful and Non-Misleading Information About Medicines with Health Care Professionals and Payers.”
Anyone expecting industry endorsement of a wildly permissive regime by these industry giants will be incredibly disappointed. Through and through, the Principles call for “responsible, science-based parameters for accurate and trusted information sharing.” (See sidebar for the list of the nine principles.)
Similarly, anyone expecting quick, sweeping and decisive action by the FDA is likely to be disappointed.
As consensus FDA expert and former Associate Commissioner Wayne Pines told me recently, “The timing of FDA action in terms of issuing new guidances or formally changing enforcement standards is likely to depend on FDA leadership priorities, the election outcome, and any legislative action by this year’s and next year’s Congress.”
Regardless, as Pines notes, “the Principles provide a productive framework for discussing how to modernize the standards so that the companies can engage in communications more productively with doctors, patients and the reimbursement community.”
Three key commitments form the basis for the new PhRMA/BIO Principles:
1. Commitment to Science-based Communication;
2. Commitment to Provide Appropriate Context about Data; and
3. Commitment to Accurate Representation of Data
The statement makes clear that the two associations believe that to exercise sound medical judgment in treating patients, doctors must understand the full range of treatment options, both off-label and on-label. Indeed, the data recommended go well beyond existing FDA policy for such communication including pharmacoeconomics, usage based on real world evidence and post hoc analysis.
Appendix A lists the types of information about medicines that could well become part of the new standard of communication policy:
• Data from randomized, controlled clinical trials, including those done after the drug is approved and not included in the product label.
• Post hoc analysis, including sub-population data not analyzed as part of initial labeling.
• Observation data and real-world evidence, including from claims data, electronic medical records, comparative effectiveness research and patient registries.
• Pharmacoeconomic information obtained from clinical trials, observational studies, reviews of medical record databases and other predictive modeling techniques.
What happens next at the FDA is anyone’s guess. Indeed, although the FDA has promised guidance on some of these issues for at least two years, the well-publicized internal struggles have delayed action. Still, it is reasonable to guess that the FDA will start with some of the comparatively easy issues, especially more permissive rules relating to communication with payers. Payer communications have been on the forefront of FDA’s plate for some time, dating back to the 1997 passage of FDAMA 114, and few would deny that insurance companies and formulary committees are unsophisticated in their comprehension of clinical data.
So, stay tuned. The FDA communication standards are already in flux and sometime in the foreseeable future will alter some of the more important communication exchanges of drug sponsors with health care professionals, patients and payers. While little is certain, one thing is clear: More data and information will be available to doctors and that will be good for patients.
PhRMA/BIO Information-Sharing Principles
1. Commitment to accurate, science-based communication.
2. FDA-approved labeling is primary source in sharing information with health care professionals.
3. Companies should provide scientific substantiation if shared information is not contained in FDA-approved labeling.
4. Science-based information from sources other than FDA-approved labeling helps health care professionals and payers make informed decisions for patients.
5. Communication should be tailored to the sophistication of the intended audience.
6. Science-based information about alternative use of medicines can improve health care decision-making.
7. Communicating with payers about new medicines and new uses of approved medicines facilitates quicker patient access upon approval.
8. Real-world evidence based on patient experience and pharmacoeconomic information can improve understanding of health outcomes and costs.
9. Commitment to sharing information published in scientific or medical journals.
