IMPROVE is a research project focused on understanding and addressing open-label bias of patient-reported outcomes (PROs) endpoints in cancer clinical trials.
Background
In many randomized controlled trials (RCTs) involving cancer treatments, it’s not always possible to blind patients to the treatment being given.
Regulatory bodies the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have raised concerns that the absence of blinding may compromise the trustworthiness of PROs, resulting for example in systematically favoring the experimental arm, independent of actual treatment efficacy or safety. However, empirical evidence on the extent and impact of open-label bias of PROs endpoint remains inconsistent.
Objectives
The IMPROvE project (The IMportance of Patient-Reported Outcomes in Cancer Clinical Trials: Evaluating the Effects of Therapy Masking) aims to fill this gap, by addressing the following study objectives:
- Interviews with stakeholders (e.g. academic organisations and patient advocacy groups) to identify concerns related to open-label bias and to identify possible risk factors for such bias.
- Investigation of the magnitude of a possible open-label bias of PRO endpoints in patients receiving anti-cancer treatment for haematological malignancies and of patient, treatment, and trial characteristics associated with such bias. (See PROSPERO: CRD420251061655)
- Investigation of the magnitude of a possible open-label bias of PRO endpoints in patients receiving anti-cancer treatment for solid tumours and of patient, treatment, and trial characteristics associated with such bias. (See PROSPERO: CRD420251061655)
- Development of a tool for the standardised assessment of the risk of open-label bias of PROs endpoints in RCTs evaluating anti-cancer treatments
Project Team
Fabio Efficace
Principal Investigator
Head, Health Outcomes Research Unit
GIMEMA
Amelie Anota
Co-Principal Investigator
Department of Clinical Research and Innovation
Centre Leon Bérard, Lyon, France
Francesco Sparano
Project Coordinator
Health Outcomes Research Unit
GIMEMA
Advisory Board
Neil Aaronson
Division of Psychosocial Research and Epidemiology
Netherlands Cancer Institute,
Amsterdam, the Netherlands
Gary Collins
Department of Applied Health Sciences
University Hospitals Birmingham
United Kingdom
Rajshekhar Chakraborty
Division of Hematology and Oncology,
Department of Medicine
Columbia University Irving Medical Center
New York, NY
David Cella
Department of Medical Social Sciences
Northwestern University Feinberg School of Medicine
Chicago, IL, USA
Massimo Di Maio
Department of Oncology
University of Turin, AOU Città della Salute e della Scienza di Torino
Torino, Italy
Publications
Abstracts
Stakeholder perspectives on possible open-label bias of patient-reported outcome results from cancer randomized controlled trials: the IMPROvE Project
Fabio Efficace, Neil Aaronson, David Cella, Gary S. Collins, Francesco Sparano, Massimo Di Maio, Johannes M. Giesinger, Rajshekhar Chakraborty, Amelie Anota
32nd Annual Conference of the International Society for Quality of Life Research (ISOQOL), Milwaukee, Wisconsin, USA. 2025
Quality and timing of patient-reported outcome reports in randomized controlled trials (RCTs) of patients with lymphoid malignancies: Analysis of 83 RCTs published between 2015 and 2025
Fabio Efficace, David Cella, Neil Aaronson, Gary S. Collins, Francesco Sparano, Rajshekhar Chakraborty, Darshi Shah, Veer Shah, Chiara Pallisco, Giorgia Novero, Valentina Ponti, Paola Sabbadini, Anna Amela Valsecchi, Massimo Di Maio, Amelie Anota
Blood (2025) 146 (Supplement 1): 4408.