BMC Medical Ethics 20 (1):1-10 (2019)

Marcin Waligora
Jagiellonian University
Novel precision oncology trial designs, such as basket and umbrella trials, are designed to test new anticancer agents in more effective and affordable ways. However, they present some ethical concerns referred to scientific validity, risk-benefit balance and informed consent. Our aim is to discuss these issues in basket and umbrella trials, giving examples of two ongoing cancer trials: NCI-MATCH and Lung-MAP study. We discuss three ethical requirements for clinical trials which may be challenged in basket and umbrella trial designs. Firstly, we consider scientific validity. Thanks to the new trial designs, patients with rare malignancies have the opportunity to be enrolled and benefit from the trial, but due to insufficient accrual, the trial may generate clinically insignificant findings. Inadequate sample size in study arms and the use of surrogate endpoints may result in a drug approval without confirmed efficacy. Moreover, complexity, limited quality and availability of tumor samples may not only introduce bias and result in unreliable and unrepresentative findings, but also can potentially harm patients and assign them to an inappropriate therapy arm. Secondly, we refer to benefits and risks. Novel clinical trials can gain important knowledge on the variety of tumors, which can be used in future trials to develop effective therapies. However, they offer limited direct benefits to patients. All potential participants must wait about 2 weeks for the results of the genetic screening, which may be stressful and produce anxiety. The enrollment of patients whose tumors harbor multiple mutations in treatments matching a single mutation may be controversial. As to informed consent – the third requirement we discuss, the excessive use of phrases like “personalized medicine”, “tailored therapy” or “precision oncology” might be misleading and cause personal convictions that the study protocol is designed to fulfill the individual health-related needs of participants. We suggest that further approaches should be implemented to enhance scientific validity, reduce misunderstandings and risks, thus maximizing the benefits to society and to trial participants.
Keywords No keywords specified (fix it)
Categories (categorize this paper)
DOI 10.1186/s12910-019-0395-5
Edit this record
Mark as duplicate
Export citation
Find it on Scholar
Request removal from index
Revision history

Download options

PhilArchive copy

Upload a copy of this paper     Check publisher's policy     Papers currently archived: 60,901
External links

Setup an account with your affiliations in order to access resources via your University's proxy server
Configure custom proxy (use this if your affiliation does not provide a proxy)
Through your library

References found in this work BETA

Defining and Describing Benefit Appropriately in Clinical Trials.Nancy M. P. King - 2000 - Journal of Law, Medicine and Ethics 28 (4):332-343.

Add more references

Citations of this work BETA

No citations found.

Add more citations

Similar books and articles

Underestimation of Harms in Phase I Trials.Karolina Strzebonska & Marcin Waligora - 2019 - Journal of Law, Medicine and Ethics 47 (2):334-335.
Phase 1 Oncology Trials and Informed Consent.Franklin G. Miller & Steven Joffe - 2013 - Journal of Medical Ethics 39 (12):761-764.
Ethics at Phase 0: Clarifying the Issues.Jonathan Kimmelman - 2007 - Journal of Law, Medicine and Ethics 35 (4):727-733.


Added to PP index

Total views
12 ( #771,996 of 2,439,086 )

Recent downloads (6 months)
1 ( #434,440 of 2,439,086 )

How can I increase my downloads?


My notes