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Low-value approvals and high prices might incentivize ineffective drug development

  • An Addendum to this article was published on 12 November 2018

Drug regulators’ acceptance of any statistically significant improvement shown in a single randomized trial and lofty drug prices has created a situation where it is now, hypothetically, profitable for a company to run a clinical trials portfolio of chemically inert compounds. While the current cancer drug pipeline is certainly superior to inert drugs, we must rethink market incentives to encourage transformational drug development.

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The authors declare no competing interests.

Correspondence to Sham Mailankody.

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Fig. 1: Break- even point at which a null trials agenda would turn a profit based on one or two nominally significant randomized trials.