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Focus on Regulation

FDA Official Offers Interpretation of FDAMA 114 Standard for Health Care Economic Information (HCEI)

Dr. Robert Temple, CDER’s Deputy Director of Clinical Science, recently presented slides at a meeting of the National Pharmaceutical Council on potential uses for comparative effectiveness research.  Significantly, his speech included an interpretation of section 114 of the Food and Drug Administration Modernization Act of 1997 (“FDAMA 114”) applying to HCEI.   We believe that Dr. Temple’s interpretation of FDAMA 114 is the first one by any FDA official or by the agency itself.   His interpretation, and some possible implications of it, are summarized below, and Hogan Lovells recently released a client update offering a more detailed analysis.  

In short, according to Dr. Temple, FDAMA 114: 

  • Does not allow clinical claims where “those claims do not satisfy FDA’s evidentiary standards…” for clinical claims.
  • When the HCEI relates to the economic consequences directly related to the labeled effect of the drug, “any comparative claims must be supported by substantial evidence that directly compares the treatments in question (i.e. head-to-head clinical trials that provide a valid comparison of two drugs).”
  • When the claims are about economic consequences that follow from the labeled effectiveness of the product, any comparative claims “can be supported by competent and reliable scientific evidence related to the consequences of an effect once the primary clinical outcomes have been established.”
  • In other words, establishing the economic impact of a therapy would be based on economic analysis; and establishing the clinical outcome assumptions would be based on traditional means of establishing effectiveness. 

Dr. Temple’s interpretation’s of FDAMA 114 introduces a new layer of complexity that pharmaceutical manufacturers must consider in their communications with formulary committees.  Already, HCEI communications occur within the shadow of guidance from both the OIG and PhRMA: 

  • The OIG Compliance Guidance for Pharmaceutical Manufacturers cautions manufacturers against impermissibly influencing committee deliberations, but observes “[s]o long as the determination of clinical efficacy and appropriateness of formulary drugs by the formulary committee precedes, and is paramount to, the consideration of costs, the development of a formulary is unlikely to raise significant issues under the anti-kickback statutes.”
  • The PhRMA Code provides guidance regarding interactions with health care professionals who serve on formulary committees. Under this guidance, a company should require such individuals to disclose their relationship with the company to the committee, and follow the committee’s procedures, possibly including recusing themselves from certain decisions.

Now, pharmaceutical companies must also weigh Dr. Temple’s speech as they approach formulary committee members with cost data. To meet Dr. Temple’s standard of scientific evidence sponsors may be required to conduct very large trials to permissibly communicate HCEI. Where they have not conducted such trials, useful information about the costs and effects of treatments might not reach the hands of formulary committee members, stakeholders who are in an ideal position to make decisions about the cost effectiveness of drugs.