Improving the Exchange of Health Care Economic Data
One area of interest to the National Pharmaceutical Council (NPC) and our member biopharmaceutical companies—and an area we’ve focused on for several years—has been the exchange of evidence to inform decision-makers. 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.
One area of interest to the National Pharmaceutical Council (NPC) and our member biopharmaceutical companies—and an area we’ve focused on for several years—has been the exchange of evidence to inform decision-makers. 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.
That’s why we were pleased that the Academy of Managed Care Pharmacy (AMCP) brought together a group of stakeholders with a keen interest in addressing these issues. In early March, NPC was a panelist and participated in a discussion led by AMCP, and the proceeds of that conversation were published in the July edition of the Journal of Managed Care & Specialty Pharmacy.
The conversation focused on several areas of Section 114 that remain vague. Specifically, stakeholders recommended the following changes for FDA or policymakers to consider:
- 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.
AMCP has plans to continue this discussion, starting with a webinar on July 20 and a second meeting of stakeholders in September. We encourage you to read the synopsis in JMCP, as well as NPC’s resources on this topic.