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Real-world evidence and regulatory drug approval

The FDA has demonstrated a willingness to expedite access to new cancer medicines by using real-world evidence to support regulatory drug approval. In this article, we explore three recent examples of such approvals and the lessons that can be learned from this collective experience.

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  1. Topp, M. S. et al. Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Oncol. 16, 57–66 (2015).

    Article  CAS  Google Scholar 

  2. Przepiorka, D. et al. FDA approval: blinatumomab. Clin. Cancer Res. 21, 4035–4039 (2015).

    Article  CAS  Google Scholar 

  3. Gokbuget, N. et al. International reference analysis of outcomes in adults with B-precursor Ph-negative relapsed/refractory acute lymphoblastic leukemia. Haematologica 101, 1524–1533 (2016).

    Article  Google Scholar 

  4. Kantarjian, H. et al. Blinatumomab versus chemotherapy for advanced acute lymphoblastic leukemia. N. Engl. J. Med. 376, 836–847 (2017).

    Article  CAS  Google Scholar 

  5. Pease, A. M. et al. Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. BMJ 357, j1680 (2017).

    Article  Google Scholar 

  6. Center for Drug evaluation and Research. Approval Package for: Application number: 761049Orig1s000 (2017).

  7. Wedam, S. et al. FDA approval summary: palbociclib for male patients with metastatic breast cancer. Clin. Cancer Res. (2019).

    Article  PubMed  Google Scholar 

  8. Bartlett, C. H. et al. Real-world evidence of male breast cancer (BC) patients treated with palbociclib (PAL) in combination with endocrine therapy (ET) [abstract]. J. Clin. Oncol. 37 (Suppl. 15), 1055 (2019).

    Article  Google Scholar 

  9. Weiss, J. R., Moysich, K. B. & Swede, H. Epidemiology of male breast cancer. Cancer Epidemiol. Biomarkers Prev. 14, 20–26 (2005).

    Article  Google Scholar 

  10. Fassnacht, M. et al. Combination chemotherapy in advanced adrenocortical carcinoma. N. Engl. J. Med. 366, 2189–2197 (2012).

    Article  CAS  Google Scholar 

  11. Gounder, M. M. et al. Phase III, randomized, double blind, placebo-controlled trial of sorafenib in desmoid tumors (Alliance A091105) [abstract]. J. Clin. Oncol. 36 (Suppl. 15), 11500 (2018).

    Article  Google Scholar 

  12. Escobar, C., Munker, R., Thomas, J., Li, B. D. & Burton, G. V. Update on desmoid tumors. Ann. Oncol. 23, 562–569 (2011).

    Article  Google Scholar 

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The work of C.M.B. is supported as the Canada Research Chair in population Cancer Care.

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Correspondence to Christopher M. Booth.

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The authors declare no competing interests.

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Raphael, M.J., Gyawali, B. & Booth, C.M. Real-world evidence and regulatory drug approval. Nat Rev Clin Oncol 17, 271–272 (2020).

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