When Does FDAMA Section 114 Apply? Ten Case Studies.

BACKGROUND: Section 114 of the Food and Drug Administration Modernization Act of 1997 regulates the promotion of health economic information by pharmaceutical companies to US health plans. Greater clarity is important given demands by payers and other stakeholders for evidence of value. OBJECTIVES: To develop hypothetical case studies of health economic promotions to examine legal and policy implications. METHODS: We constructed for pedagogical purposes 10 categories of potential health economic promotions. We generated hypothetical case studies for each category, including questions about whether each might be allowable under Section 114. The case studies were developed around the following categories: 1) costing out on-label clinical end points; 2) promotion of a costing exercise to physicians working in an accountable care organization setting; 3) burden-of-illness claims; 4) economic analysis of a formulary restriction policy; 5) extrapolations to doses, populations, or settings not covered in trials; 6) adherence claims; 7) "utilization of care" as a secondary end point in randomized clinical trials; 8) costing out a competitor drug's adverse event; 9) economic analysis of comparative effectiveness claims using an indirect treatment comparison; and 10) extrapolating from surrogate to long-term outcomes in an economic model. DISCUSSION: Most cases seem to fall into a gray zone given haziness around what constitutes "competent and reliable evidence" and "directly relate[d]" to an approved indication. In practice, it is difficult to know what the section allows given the imprecision of the statute and lack of guidance about its scope. CONCLUSION: Ideally, future guidance will provide clarity and flexibility.