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Statistical refocusing in the design of Phase II trials offers promise of increased R&D productivity

Nature Reviews Drug Discovery volume 13, pages 638–640 (2014)Cite this article

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The process of drug research and development (R&D) is expensive and time-consuming, and productivity in terms of the number of new drugs resulting from a given level of investment in R&D has been declining for decades, as highlighted in a recent article (Diagnosing the decline in pharmaceutical R&D efficiency. Nature Rev. Drug Discov. 11, 191–200 (2012))1. Despite many calls for innovation to the drug development process, few substantively different proposals have been articulated or used. Here, we propose a simple and implementable means to increase productivity by focusing on decisions that are under the industry's control — the choices of statistical parameters in Phase II trial design.

Rigorous modelling and quantification of strategic and business decisions required during clinical development can supplement intuition and judgement, reveal levels of uncertainty in outcomes, and foster insights into interrelationships and implications. By extending work done by Paul and colleagues2, which examined the relative importance of cost, cycle time and success rates across eight phases in preclinical and clinical development on overall R&D productivity, we created a model called the 'Strategic R&D portfolio model' (Fig. 1b). Using this model, we can provide detailed analyses based on industry-wide data to address company-specific questions regarding how to most effectively improve productivity.

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Figure 1: The strategic R&D portfolio model.
Figure 2: Optimal false positive and false negative rates in Phase II trials to achieve the highest productivity across an R&D portfolio.

References

  1. Scannell, J. W. et al. Diagnosing the decline in pharmaceutical R&D efficiency. Nature Rev. Drug Discov. 11, 191–200 (2012).

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  2. Paul, S. M. et al. How to improve R&D productivity: the pharmaceutical industry's grand challenge. Nature Rev. Drug Discov. 9, 203–214 (2010).

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Authors and Affiliations

  1. Biogen Idec, 14 Cambridge Center, Cambridge, 02142, Massachusetts, USA

    Stacy R. Lindborg

  2. Eli Lilly and Company, Lilly Corporate Center, Indianapolis, 46285, Indiana, USA

    Charles C. Persinger, Andreas Sashegyi, Craig Mallinckrodt & Stephen J. Ruberg

Authors
  1. Stacy R. Lindborg
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  2. Charles C. Persinger
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  3. Andreas Sashegyi
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  4. Craig Mallinckrodt
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  5. Stephen J. Ruberg
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Corresponding author

Correspondence to Stacy R. Lindborg.

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Competing interests

S.R.L, C.C.P., A.S., C.M. and S.J.R. own stock of the companies that they are employed by.

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Lindborg, S., Persinger, C., Sashegyi, A. et al. Statistical refocusing in the design of Phase II trials offers promise of increased R&D productivity. Nat Rev Drug Discov 13, 638–640 (2014). https://doi.org/10.1038/nrd3681-c1

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