Highlights
- •Of the 180 basket trials, 55% had published results with a median response rate of 14%.
- •Of the 73 umbrella trials, 38% had published results with a median response rate of 18%.
- •Response rates varied by tumour type and drug target.
Abstract
Introduction
We sought to characterise oncology basket and umbrella trials that have been implemented,
determine how many have been completed, and calculate the response rate, by tumour
type and drug target.
Methods
We conducted a retrospective, cross-sectional review of PubMed, Embase, and clinicaltrials.gov for all oncology basket and umbrella trials. We included all trials and publications
reporting on the results of these trials, and we calculated overall response rates,
stratified by tumour type and drug target.
Results
Most basket and umbrella trials are phase II and non-randomised in design. Of the
180 basket trials, 99 (55.0%) had published results and 81 (45.0%) did not. Of the
73 umbrella trials, 28 (38.4%) had published results and 45 (61.6%) did not. The median
response rate was 14.0 (IQR: 4.2, 31.2) for basket trials and 17.8 (IQR: 3.8, 40.4)
for umbrella trials. These responses varied, depending on tumour type and drug target.
Conclusions
Understanding what is known about these trials, especially given the limited but heterogenous
response reported in these trials, provides context about the strengths and limitations
of drugs, especially since several drugs have been approved in recent years for tumour-agnostic
indications, based on the results of these types of trials.
Keywords
To read this article in full you will need to make a payment
Purchase one-time access:
Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online accessOne-time access price info
- For academic or personal research use, select 'Academic and Personal'
- For corporate R&D use, select 'Corporate R&D Professionals'
Subscribe:
Subscribe to European Journal of CancerAlready a print subscriber? Claim online access
Already an online subscriber? Sign in
Register: Create an account
Institutional Access: Sign in to ScienceDirect
References
- Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples.Ann Oncol. 2017; 28: 34-43https://doi.org/10.1093/annonc/mdw413
- Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols.Trials. 2019; 20: 572https://doi.org/10.1186/s13063-019-3664-1
- Encouraging trends in modern phase 1 oncology trials.N Engl J Med. 2018; 378: 2242-2243https://doi.org/10.1056/NEJMc1803837
- An overview of cancer drugs approved by the US food and drug administration based on the surrogate end point of response rate.JAMA Intern Med. 2019; 179: 915-921https://doi.org/10.1001/jamainternmed.2019.0583
- Basket and umbrella trials: untapped opportunities in rare disease.Clinical Trials Arena [Internet]. 21 Dec 2021; ([cited 19 Aug 2022]. Available:)
- An overview of precision oncology basket and umbrella trials for clinicians.Ca - Cancer J Clin. 2020; 70: 125-137https://doi.org/10.3322/caac.21600
Freepik. Physiology vectors & illustrations for free download. [cited 28 Sep 2022]. Available: a href="https://www.freepik.com/vectors/physiology">Physiology vector created by brgfx- www.freepik.com.
Freepik. Medicine bottle vectors & illustrations for free download. [cited 28 Sep 2022]. Available: a href="https://www.freepik.com/vectors/medicine-bottle">Medicine bottle vector created by macrovector-www.freepik.com.
Article info
Publication history
Published online: December 06, 2022
Accepted:
October 26,
2022
Received:
October 25,
2022
Identification
Copyright
© 2022 Elsevier Ltd. All rights reserved.
