Advertisement

A systematic review of basket and umbrella trials in oncology: the importance of tissue of origin and molecular target

Published:December 06, 2022DOI:https://doi.org/10.1016/j.ejca.2022.10.027

      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 access
      One-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 Cancer
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Renfro L.A.
        • Sargent D.J.
        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
        • Park J.J.H.
        • Siden E.
        • Zoratti M.J.
        • Dron L.
        • Harari O.
        • Singer J.
        • et al.
        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
        • Chakiba C.
        • Grellety T.
        • Bellera C.
        • Italiano A.
        Encouraging trends in modern phase 1 oncology trials.
        N Engl J Med. 2018; 378: 2242-2243https://doi.org/10.1056/NEJMc1803837
        • Chen E.Y.
        • Raghunathan V.
        • Prasad V.
        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
        • Newton W.
        Basket and umbrella trials: untapped opportunities in rare disease.
        Clinical Trials Arena [Internet]. 21 Dec 2021; ([cited 19 Aug 2022]. Available:)
        • Park J.J.H.
        • Hsu G.
        • Siden E.G.
        • Thorlund K.
        • Mills E.J.
        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
      1. 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.

      2. 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.