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Characterizing the US FDA's approach to promoting transformative innovation

Nature Reviews Drug Discovery volume 14, pages 740–741 (2015)Cite this article

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In 2014, US lawmakers launched the 21st Century Cures initiative to support and accelerate the development of innovative new therapies (see Further information). In order to inform these ongoing legislative proposals, we investigated how the US Food and Drug Administration (FDA) is currently facilitating the approval of innovative therapeutics.

We identified all novel therapeutics approved by the FDA between 2005 and 2012 and used an established framework developed by the FDA to classify them by the degree of innovation: first-in-class, advance-in-class and addition-to-class (Health Aff. 32, 1433–1439; 2013). Although innovation can be measured in different ways, drugs with novel mechanisms of action (first-in-class) are largely considered to be the most innovative. Drugs that provide important clinical benefits despite not being mechanistically novel (advance-in-class) may be equally important innovations in terms of their clinical promise. We compared the use of priority review and accelerated approval regulatory pathways, regulatory review times, and characteristics of pivotal trials, including number, design, primary end point, duration, and size, for novel therapeutics stratified by degree of innovation (for details of the dataset and analysis methods, see Supplementary information S1 (box)).

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Figure 1: Characteristics of novel therapeutics approved by the FDA between 2005 and 2012, stratified by degree of innovation.

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Authors and Affiliations

  1. Center for Outcomes Research and Evaluation, Yale–New Haven Hospital, New Haven, Connecticut, USA

    Nicholas S. Downing, Harlan M. Krumholz & Joseph S. Ross

  2. Division of Health Care Policy and Research, Mayo Clinic, Rochester, Minnesota

    Nilay D. Shah

  3. Yale University School of Medicine, P.O. Box 208093, New Haven, 06520, Connecticut, USA

    Harlan M. Krumholz & Joseph S. Ross

Authors
  1. Nicholas S. Downing
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  2. Harlan M. Krumholz
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  3. Joseph S. Ross
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  4. Nilay D. Shah
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Corresponding author

Correspondence to Joseph S. Ross.

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Competing interests

H. Krumholz and J. Ross are funded by research agreements through Yale University from Medtronic and Johnson & Johnson (Janssen) to develop methods of clinical trial data sharing, by the US Centers for Medicare & Medicaid Services to develop and maintain performance measures, and by the US Food and Drug Administration to develop methods for post-market surveillance of medical devices. H. Krumholz chairs a cardiac scientific advisory board for UnitedHealth. The other authors do not have competing interests to report.

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This research was not supported by any external grants or funds.

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Supplementary information

Supplementary information S1 (box)

Data and analysis (PDF 449 kb)

Supplementary information S2 (figure)

Novel therapeutics approved by the FDA between 2005 and 2012 and their degree of innovation, stratified by drug type and therapeutic area. (PDF 113 kb)

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Downing, N., Krumholz, H., Ross, J. et al. Characterizing the US FDA's approach to promoting transformative innovation. Nat Rev Drug Discov 14, 740–741 (2015). https://doi.org/10.1038/nrd4734

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