Recent FDA draft guidance for sponsors of oncology clinical trials encourages enrolment of patients with incurable cancer and no potential for prolonged and/or near-normal survival, regardless of whether they have received existing treatment options. This guidance constitutes a substantial departure from current standards, with potentially profound implications for trial participants as well as drug regulation and reimbursement.
This is a preview of subscription content
Subscribe to Journal
Get full journal access for 1 year
only $4.92 per issue
All prices are NET prices.
VAT will be added later in the checkout.
Tax calculation will be finalised during checkout.
Rent or Buy article
Get time limited or full article access on ReadCube.
All prices are NET prices.
US Food & Drug Administration. FDA In Brief: FDA Encourages Inclusion of Patients with Incurable Cancers in Oncology Clinical Trials Regardless of Prior Therapies. FDA https://www.fda.gov/news-events/press-announcements/fda-brief-fda-encourages-inclusion-patients-incurable-cancers-oncology-clinical-trials-regardless (2021).
Tao, D. L., Kartika, T., Tran, A. & Prasad, V. Phase I trials and therapeutic intent in the age of precision oncology: what is a patient’s chance of response? Eur. J. Cancer 139, 20–26 (2020).
Stensland, K. D. et al. Adult cancer clinical trials that fail to complete: an epidemic? J. Natl Cancer Inst. 106, dju229 (2014).
US Food & Drug Administration. Oncology Center of Excellence Guidance Documents. FDA https://www.fda.gov/about-fda/oncology-center-excellence/oncology-center-excellence-guidance-documents (2021).
Abola, M. V. & Prasad, V. The use of superlatives in cancer research. JAMA Oncol. 2, 139–141 (2016).
Swain, S. M. et al. Pertuzumab, trastuzumab and docetaxel in HER2-positive metastatic breast cancer. N. Engl. J. Med. 372, 724–734 (2015).
Kim, M. S. & Prasad, V. New drugs and options can enhance patient outcomes: but can they also erode them? Eur. J. Cancer 154, 1–3 (2021).
Chino, F. & Zafar, S. Y. Financial toxicity and equitable access to clinical trials. Am. Soc. Clin. Oncol. Educ. Book 39, 11–18 (2019).
Powell, K., Lythgoe, M. P. & Prasad, V. The Oncologic Drugs Advisory Committee votes of April 2021 — implications for the fate of Accelerated Approval. JAMA Oncol. https://doi.org/10.1001/JAMAONCOL.2021.3046 (2021).
The RECOVERY Collaborative Group. Dexamethasone in hospitalized patients with Covid-19. N. Engl. J. Med. 384, 693–704 (2021).
V.P. has received research funding from Arnold Ventures, royalties from the Johns Hopkins Press, Speaker’s fees from Evicore, and honoraria from Medscape and from various universities, medical centres, non-profit organizations and professional societies. V.P. has also acted as a consultant of UnitedHealthcare, and his Plenary Session podcast has Patreon backers. M.P.L. declares no competing interests.
About this article
Cite this article
Lythgoe, M.P., Prasad, V. The FDA’s latest move to expand eligibility for oncology trials — a double-edged sword?. Nat Rev Clin Oncol 18, 745–746 (2021). https://doi.org/10.1038/s41571-021-00559-0