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How and Why Is Broader Exchange of Information Needed to Support Public Health and Delivery Reform?

October 13, 2016

On November 9-10, 2016, the Food and Drug Administration will host a stakeholder meeting to obtain input on how biopharmaceutical companies and others are allowed to exchange information, such as health care economic data and other types of evidence, with health care decision-makers. This will be an incredibly important discussion, because, as the National Pharmaceutical Council (NPC) has outlined:

“In an era increasingly focused on value, the exchange of information about treatment benefits, risks, and costs is necessary for payer organizations and others charged with determining whether to cover a medication. This information is not typically included in Food and Drug Administration (FDA)-approved labeling, and communication of this information was later permitted under Section 114 of the Food and Drug Administration Modernization Act of 1997. Yet this law is vaguely written and has been implemented without further FDA guidance, resulting in more limited use of these provisions and lack of clarity for typical payer questions.  

“It’s important to recognize that it’s been nearly 20 years since the law was written. In that time, there have been tremendous advances in medical treatments and technologies and how we conduct and communicate research. There also are many more types of health care decision-makers determining care for populations of patients. Yet the law hasn’t kept pace with the times, and the ambiguities remain.”

Given this uncertainty, payers often are seeking information about how a treatment might work in patient populations with varying levels of disease severity, at different dosage levels, or in “real-world” settings with different outcomes, all of which is often not included in approved product labeling.

To illustrate the uncertainty in how this kind of information could be communicated, Tufts Medical Center’s Dr. Peter Neumann led a landmark study, funded by NPC, which outlined 10 hypothetical case examples. He considered whether certain types of information could be shared, such as a costing exercise with physicians working in an accountable care organization setting who are responsible for total cost of care, an economic analysis of a formulary restriction policy on overall health care costs, adherence claims comparing one treatment to another, and an economic model to estimate long-term benefits based on surrogate endpoints of a treatment (e.g., survival projection for patients with heart disease based upon cholesterol levels), among other scenarios. This study also helped to spur the dialogue at a meeting of multiple health care stakeholders convened by the Academy of Managed Care Pharmacy (AMCP) earlier this year. There, the group developed several recommendations for FDA consideration:

  • Broaden the information that can be shared. Health care economic information (HCEI) includes much more than costs, and should encompass such things as health care utilization (e.g., hospitalizations, emergency department visits), patient benefits, adherence, endpoint extrapolations, quality of life, and adverse events.
  • Clarify certain definitions. "Competent and reliable scientific evidence" -- which will form the basis of HCEI -- should be defined as "truthful and non-misleading tests, analyses, research, studies, models, or other evidence."
  • Communicate information earlier. HCEI related to a drug in the pipeline should be communicated to appropriate stakeholders 12 to 18 months prior to approval.
  • Expand the types of decision-makers who can receive this information. "Other similar entities" that could receive HCEI besides pharmacy and therapeutics committees and formulary committees include health plans and integrated delivery systems that make health care decisions for patient populations, and organizations that evaluate HCEI or develop value frameworks and compendia.
  • Consider ways to make the exchange of information easier. AMCP's Format for Formulary Submissions and the eDossier system is one format well suited to seek HCEI from manufacturers, but other options also exist.

NPC welcomes FDA’s comprehensive review of its regulations and policies governing communication of unapproved uses of approved/cleared medical products. FDA has indicated its interest in hearing from a broad range of stakeholders on this topic, and numerous stakeholders are expected to attend the November meeting and submit comments on ways to improve existing laws. Comments for e-submission prior to the meeting are due on October 26 for stakeholder wishing to comment. Electronic and written comments can be submitted through January 9, 2017.

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