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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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References

  1. 1.

    Tang, J., Shalabi, A. & Hubbard-Lucey, V. M. Comprehensive analysis of the clinical immuno-oncology landscape. Ann. Oncol. 29, 84–91 (2018).

  2. 2.

    Fojo, T., Mailankody, S. & Lo, A. Unintended consequences of expensive cancer therapeutics-the pursuit of marginal indications and a me-too mentality that stifles innovation and creativity: the John Conley Lecture. JAMA Otolaryngol. Head Neck Surg. 140, 1225–1236 (2014).

  3. 3.

    Gyawali, B. & Prasad, V. Drugs that lack single-agent activity: are they worth pursuing in combination? Nat. Rev. Clin. Oncol. 14, 193 (2017).

  4. 4.

    Carlisle, B. et al. Benefit, risk, and outcomes in drug development: a systematic review of sunitinib. J. Natl Cancer Inst. 108, djv292 (2016).

  5. 5.

    Zhu, A. X. et al. Effect of everolimus on survival in advanced hepatocellular carcinoma after failure of sorafenib: the EVOLVE-1 randomized clinical trial. JAMA 312, 57–67 (2014).

  6. 6.

    Chan, A. et al. Neratinib after trastuzumab-based adjuvant therapy in patients with HER2-positive breast cancer (ExteNET): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 17, 367–377 (2016).

  7. 7.

    Gyawali, B. & Ando, Y. Adjuvant sunitinib for high-risk-resected renal cell carcinoma: a meta-analysis of ASSURE and S-TRAC trials. Ann. Oncol. 28, 898–899 (2017).

  8. 8.

    Prasad, V. & Mailankody, S. Research and development spending to bring a single cancer drug to market and revenues after approval. JAMA Intern. Med. 177, 1569–1575 (2017).

  9. 9.

    Sertkaya, A. et al. Key cost drivers of pharmaceutical clinical trials in the United States. Clin. Trials 13, 117–126 (2016).

  10. 10.

    Basch, E. Toward a patient-centered value framework in oncology. JAMA 315, 2073–2074 (2016).

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The authors declare no competing interests.

Correspondence to Sham Mailankody.

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Further reading

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