When Biomarkers Are Not Enough: FDA Evaluation of Effectiveness of Neuropsychiatric Devices for Disorders of Consciousness


Studies demonstrating the efficacy of interventions for neuropsychiatric disorders often rely on the assessment of a trained observer or patient reports, evidence that can be more subjective than biomarkers. The problem with relying upon subjective evidence of efficacy is that objective evidence is advantaged in the medical product approval process, notwithstanding the increase in submission of patient-reported outcomes in applications to the U.S. Food and Drug Administration (FDA). The FDA’s preference for biomarkers may thus adversely impact the approval process for interventions targeting neuropsychiatric conditions, for which there are no biomarkers or only emerging biomarkers (with limited sensitivity or specificity) associated with the disorders.

In this Article, we first review how the FDA evaluates different types of evidence of an intervention’s efficacy, focusing on Class III medical devices. We use the case of disorders of consciousness to analyze relevant regulations and guidance for the development of novel neurotechnologies when subjective data is used in an approval process. We then offer suggestions for reform, arguing for a more expansive approach to what constitutes evidence of efficacy en route to more objective and vetted biomarkers. Specifically, we argue that patient-provided information is valuable and should be included in applications to the FDA, yielding a more integrated and holistic approach, or what some scholars refer to as a “pluralistic way of knowing.” We also argue for greater transparency from the FDA about how patient-provided information is evaluated, valued, and weight as part of the application for approval of new drugs and devices. While our Article focuses on disorders of consciousness and neurotechnology, our arguments have broader implications for the evaluation of emerging drugs and devices designed to ameliorate other neuropsychiatric conditions where the subjective experience of patients has particular relevance.


Stanford University Stanford, California
  • 21 Stan. Tech. L. Rev. 276 (2018)
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