The Food And Drug Administration Has The Legal Basis To Restrict Promotion Of Flawed Comparative Effectiveness Research

Health Affairs (Impact Factor: 4.97). 10/2012; 31(10):2200-5. DOI: 10.1377/hlthaff.2012.0787
Source: PubMed


Under Food and Drug Administration (FDA) policy, communications by prescription drug manufacturers must be backed by "substantial evidence" from "adequate and well-controlled investigations." But numerous exceptions permit manufacturer promotion based on data other than randomized trials. The observational research presented in the Hemikrane hypothetical case in this month's Health Affairs is methodologically flawed and also does not meet any of these exceptions. Therefore, plausible scientific and policy rationales support rules restricting the company's communication of its findings. The FDA's current reluctance to authorize promotional claims based on observational research is understandable. Further work is required to define the characteristics of high-quality observational research. However, as this field matures, higher-quality observational studies could meet the legal standard of an "adequate and well-controlled investigation." At that point, the FDA will need to issue formal guidance to minimize confusion on what kinds of observational research can meet its evidentiary standards.

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    ABSTRACT: Hypothetical comparative effectiveness studies of two migraine drugs offer an opportunity to analyze the legal issues that manufacturers face in conveying to practicing physicians information from such research about prescription drugs. This information, derived from observational studies, supposedly cannot be communicated to clinicians because it does not constitute "substantial evidence" as required by law. In fact, however, the emerging effectiveness data can be included in manufacturers' communications to physicians about drugs' clinical utility through various existing channels defined by other law. This article argues that Congress should further clarify the circumstances under which such communications can occur to comply with First Amendment requirements that speech constraints be narrowly drawn, known in advance, and precise. Otherwise, the Food and Drug Administration risks losing First Amendment arguments in cases involving the agency's regulation of the communications-or "speech"-of drug manufacturers.
    Health Affairs 10/2012; 31(10):2220-4. DOI:10.1377/hlthaff.2012.0813 · 4.97 Impact Factor
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    ABSTRACT: This article explores issues of concern to payers evaluating the hypothetical comparative effectiveness case study of two fictitious migraine treatments in this month's Health Affairs. The case study presents the seemingly paradoxical situation in which randomized controlled trials produce one result, and real-world observational comparative effectiveness research produces another. For the payer making coverage decisions, this scenario raises three major themes related to interpretation and communication. First, there is a need for a well-considered set of criteria that weigh evidence across comparative effectiveness studies to determine whether enough evidence exists to communicate or enact new health care policies. Second, emphasis should be placed on studies that are published or presented in peer-reviewed settings. Third, access to raw comparative effectiveness research data would enable payers to more deeply explore research interests relevant to their particular constituencies. Payers' involvement in comparative effectiveness research should be encouraged, not discouraged, to advance our understanding of what works best and for whom.
    Health Affairs 10/2012; 31(10):2225-30. DOI:10.1377/hlthaff.2012.0730 · 4.97 Impact Factor
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    ABSTRACT: As norms of comparative effectiveness research are sought within the biomedical and health care communities, and the science of conducting and interpreting this research develops, the Food and Drug Administration (FDA) must balance diverse interests. The agency's overarching interest is the development of high-quality comparative effectiveness information that contributes to improved patient care. To further this interest, the FDA can provide expertise in trial design and postmarketing surveillance. The FDA can also ensure that manufacturers of medical products use comparative effectiveness information in product promotion in a manner consistent with regulatory requirements. In this article we observe that these requirements would preclude the manufacturer's use in a promotional context of comparative effectiveness findings derived from an observational study. The FDA recognizes, however, that there are ongoing efforts to address the methodological problems inherent in observational approaches and to foster consensus on enhanced methods. The FDA must work to navigate challenges that relate to both the science of comparative effectiveness research and the agency's statutory responsibilities to the public health.
    Health Affairs 10/2012; 31(10):2188-92. DOI:10.1377/hlthaff.2012.0958 · 4.97 Impact Factor
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