Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 May:167:152-160.
doi: 10.1016/j.ejca.2021.12.037. Epub 2022 Feb 27.

Characteristics of clinical trials for haematological malignancies from 2015 to 2020: A systematic review

William Wesson  1 , Vincent L Galate  1 , Douglas W Sborov  2 , Brian McClune  2 , Aaron M Goodman  3 , Bishal Gyawali  4 , Vinay Prasad  5 , Saqib Abbasi  1 , Ghulam Rehman Mohyuddin  6
Affiliations
Free article

Characteristics of clinical trials for haematological malignancies from 2015 to 2020: A systematic review

William Wesson et al. Eur J Cancer. 2022 May.
Free article

Abstract

Background: As the landscape of haematological malignancies dramatically changes due to diagnostic and therapeutic advances, it is important to evaluate trends in clinical trial designs. The objective of our study was to describe the design of clinical trials for five common haematological malignancies with respect to randomisation and end-points. We also aimed to assess trends over time and examine the relationships of funding source and country of origin to proportions of randomisation and utilisation of clinical end-points.

Methods: This systematic review identified haematological malignancy clinical trials starting in 2015-2020 registered at ClinicalTrials.gov as of 20th February 2021. Trial-related variables including randomisation status, type of primary end-point, and both projected and actual enrolment numbers were captured. Clinical end-points were defined as overall survival and quality of life, while surrogate end-points included all other end-points.

Results: Of 2609 relevant trials included in this analysis, only one-fifth were randomised (538, 21%), with a significant decrease in the proportion of randomised clinical trials from 26% of trials in 2015 to 19% in 2020 (p < 0.00001). Between the years 2015 and 2020, the proportion of randomised trials for all haematological malignancies using primary surrogate end-points remained relatively consistent, ranging from 84% in 2015 to 78% in 2020 (p = 0.352). Overall, only 15% of trials utilised primary end-points of overall survival or quality of life in a randomised design.

Conclusions: This systematic review of haematological malignancy trials found that the majority of trials are non-randomised and that there has been an increase in the ratio of non-randomised to randomised studies over time. The vast majority of randomised haematological malignancy trials use surrogate primary end-points.

Keywords: Clinical trials; Drug development; Hematologic neoplasms; Neoplasms; Outcomes; Overall survival; Quality of life; Randomisation.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest statement The authors declare the following financial interests/personal relationships which may be considered as potential competing interests, although the study did not recieve any funding from these sources: Vinay Prasad reports royalties from Johns Hopkins Press, Medscape, MedPage, consulting for UnitedHealthcare, and speaking fees for Evicore. Vinay Prasad has a plenary session podcast that has Patreon backers. Vinay Prasad is funded to study low value drugs through a grant from Arnold Ventures. The funder had no role in the design of this study. Aaron Goodman reports consulting for Seattle Genetics and EUSA Pharma. Douglas Sborov reports consulting for Janssen, SkylinDx, GlaxoSmithKline, Legend Biotech, Amgen and Celgene. None of the authors have any other conflicts of interest.

Comment in

Publication types