@article{nokey,

title = {Understanding the patient journey: Barriers, facilitators, and expectations in joining a chemotherapy de-escalation trial among premenopausal patients with breast cancer},

url = {https://www.sciencedirect.com/science/article/pii/S0960977626000809},

doi = { 10.1016/j.breast.2026.104770},

issn = {0960-9776},

year  = {2026},

date = {2026-03-25},

urldate = {2026-03-25},

journal = {The Breast},

volume = {87},

pages = {11},

abstract = {Background

De-escalation trials aim to balance clinical outcomes and quality of life (QoL), especially for premenopausal women with early breast cancer (BC), who often experience a greater treatment burden affecting their physical and psychosocial well-being. We sought to understand their patient journey—barriers, facilitators, and expectations—when joining de-escalation trials to inform patient-centered design and implementation.



Methods

OPTIMA-YOUNG, an EU-co-funded international trial, investigates a genomic assay-guided approach to de-escalate chemotherapy in premenopausal patients with early-stage hormone receptor (HR) + BC. A Co-creation Board of patients and healthcare professionals (HCPs) ensured stakeholder involvement throughout the trial. Focus groups (FGs) explored decision-making, needs, and QoL priorities related to joining a de-escalation clinical trial. Discussions were recorded, transcribed, anonymized, and analyzed using MAXQDA software.



Results

The three FGs included 20 participants (11 patients, 9 HCPs) from 14 countries. Three themes emerged: 1) emotional and cognitive responses to BC diagnosis/treatment; 2) environmental, HCP, and social influences on choices; 3) coping with side effects and QoL challenges. Barriers to joining a de-escalation trial included limited emotional support at diagnosis, cross-country variations in shared decision-making, poor communication on long-term side effects, fear of recurrence, and HCPs’ tendency to overtreat younger patients. Developing training for patients and HCPs was seen essential for improving communication, shared decision-making skills, and enhancing symptom management and QoL.



Conclusions

This pre-implementation study identified factors at the patient, HCP, and system levels that, if addressed, could improve the trial experience. Insights helped refine the OPTIMA-YOUNG protocol and implementation plan and may inform future de-escalation trials.

},

note = {Source: This research is part of the Path-for-Young EU-funded project.},

keywords = {Clinical Development / Trials, Patient Involvement},

pubstate = {published},

tppubtype = {article}

}

@article{nokey,

title = {The READI European project: Enhancing inclusivity in  clinical research},

url = {https://onlinelibrary.wiley.com/doi/10.1111/eci.70146},

doi = {10.1111/eci.70146 },

year  = {2025},

date = {2025-10-28},

urldate = {2025-10-28},

journal = {European Journal of Clinical Investigation},

volume = {56},

note = {Source: It is produced within the READI project consortium.},

keywords = {Clinical Development / Trials, Inclusion and Representativeness in Clinical Research},

pubstate = {published},

tppubtype = {article}

}

@article{nokey,

title = {A toolkit for capturing a representative and equitable sample in health research},

url = {https://www.nature.com/articles/s41591-023-02665-1},

doi = {10.1038/s41591-023-02665-1},

year  = {2023},

date = {2023-12-08},

urldate = {2023-12-08},

journal = {Nature Medicine},

volume = {29},

issue = {12},

pages = {3259–3267},

note = {Source: Springer Nature},

keywords = {Clinical Development / Trials, Inclusion and Representativeness in Clinical Research},

pubstate = {published},

tppubtype = {article}

}

@article{nokey,

title = {Checklist to assess Trustworthiness  in RAndomised Controlled Trials (TRACT  checklist): concept proposal and pilot},

url = {https://link.springer.com/article/10.1186/s41073-023-00130-8},

doi = {10.1186/s41073-023-00130-8},

year  = {2023},

date = {2023-06-20},

urldate = {2023-06-20},

journal = {Research Integrity and Peer Review},

volume = {8},

abstract = {Objectives 

To propose a checklist that can be used to assess trustworthiness of randomized controlled trials (RCTs).



Design 

A screening tool was developed using the four-stage approach proposed by Moher et al. This included defining the scope, reviewing the evidence base, suggesting a list of items from piloting, and holding a consensus meeting. The initial checklist was set-up by a core group who had been involved in the assessment of problematic RCTs for several years. We piloted this in a consensus panel of several stakeholders, including health professionals, reviewers, journal editors, policymakers, researchers, and evidence-synthesis specialists. Each member was asked to score three articles with the checklist and the results were then discussed in consensus meetings.



Outcome 

The Trustworthiness in RAndomised Clinical Trials (TRACT) checklist includes 19 items organised into seven domains that are applicable to every RCT: 1) Governance, 2) Author Group, 3) Plausibility of Intervention Usage, 4) Timeframe, 5) Drop-out Rates, 6) Baseline Characteristics, and 7) Outcomes. Each item can be answered as either no concerns, some concerns/no information, or major concerns. If a study is assessed and found to have a majority of items rated at a major concern level, then editors, reviewers or evidence synthesizers should consider a more thorough investigation, including assessment of original individual participant data.



Conclusions 

The TRACT checklist is the first checklist developed specifically to detect trustworthiness issues in RCTs. It might help editors, publishers and researchers to screen for such issues in submitted or published RCTs in a transparent and replicable manner.},

note = {Source: Published in BioMed Central (BMC), Springer Nature},

keywords = {Clinical Development / Trials, Inclusion and Representativeness in Clinical Research},

pubstate = {published},

tppubtype = {article}

}

@article{nokey,

title = {Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs},

author = {Cook NS, Cave J, and Holtorf A-P.},

url = {https://www.frontiersin.org/journals/medicine/articles/10.3389/fmed.2019.00082/full},

year  = {2019},

date = {2019-04-24},

abstract = {Although patient preferences have been studied broadly for marketed products or around the time of submission to authorities and launch, patient preference studies have rarely been used during the early drug development phases. In this paper, we formulate three hypotheses supporting the use of patient preference studies in early product development: (1) integration of the patient perspective into the development process from phase 1 onwards will result in healthcare solutions with outcomes that best address patients' needs; (2) a structured process to build patient-based evidence involving partnerships between patients and other key stakeholders will improve alignment of development activities with the needs of patients; (3) quantitative patient preference research built on robust qualitative insights is necessary to strengthen development decisions in the interests of patients. To illustrate such a structured process, we describe qualitative insights research (social media analysis and online bulletin boards) and quantitative patient preference studies in dry eye disease and non-alcoholic steatohepatitis conducted during early product development by a pharmaceutical company to generate patient-based evidence. The outputs from such early patient preference studies are being used to inform patient reported outcome strategies, clinical development strategies, product design and delivery features, and form the basis for early dialog with regulators, health technology assessment (HTA) bodies and payers to ensure focus and alignment on patient-relevant endpoints. Furthermore, to discuss and theoretically substantiate our hypotheses, we review how different groups and organizations are working to embrace fully the patient perspective in product development and healthcare decision-making. The hypotheses are commensurate with the general trend toward patient-centered healthcare and the activities initiated by regulators, HTA agencies, and patient organizations. We advocate that all healthcare players should actively contribute to aligning on best practices concerning choice of methodologies and engage in multi-stakeholder dialog along the entire product development chain, to realize health technologies that best meet the needs of patients.},

note = {Source: Published in Frontiers},

keywords = {Clinical Development / Trials},

pubstate = {published},

tppubtype = {article}

}

@article{nokey,

title = {Impact of patient and public involvement on enrolment and retention in clinical trials: systematic review and meta-analysis},

author = {Joanna C Crocker, Ignacio Ricci-Cabello, Adwoa Parker, Jennifer A Hirst, Alan Chant, Sophie Petit-Zeman, David Evans, Sian Ree},

url = {https://www.bmj.com/content/363/bmj.k4738},

year  = {2018},

date = {2018-10-30},

abstract = {Objective: To investigate the impact of patient and public involvement (PPI) on rates of enrolment and retention in clinical trials and explore how this varies with the context and nature of PPI.



Design: Systematic review and meta-analysis.



Data sources: Ten electronic databases, including Medline, INVOLVE Evidence Library, and clinical trial registries.



Eligibility criteria: Experimental and observational studies quantitatively evaluating the impact of a PPI intervention, compared with no intervention or non-PPI intervention(s), on participant enrolment and/or retention rates in a clinical trial or trials. PPI interventions could include additional non-PPI components inseparable from the PPI (for example, other stakeholder involvement).



Data extraction and analysis: Two independent reviewers extracted data on enrolment and retention rates, as well as on the context and characteristics of PPI intervention, and assessed risk of bias. Random effects meta-analyses were used to determine the average effect of PPI interventions on enrolment and retention in clinical trials: main analysis including randomised studies only, secondary analysis adding non-randomised studies, and several exploratory subgroup and sensitivity analyses.



Results: 26 studies were included in the review; 19 were eligible for enrolment meta-analysis and five for retention meta-analysis. Various PPI interventions were identified with different degrees of involvement, different numbers and types of people involved, and input at different stages of the trial process. On average, PPI interventions modestly but significantly increased the odds of participant enrolment in the main analysis (odds ratio 1.16, 95% confidence interval and prediction interval 1.01 to 1.34). Non-PPI components of interventions may have contributed to this effect. In exploratory subgroup analyses, the involvement of people with lived experience of the condition under study was significantly associated with improved enrolment (odds ratio 3.14 v 1.07; P=0.02). The findings for retention were inconclusive owing to the paucity of eligible studies (odds ratio 1.16, 95% confidence interval 0.33 to 4.14), for main analysis).



Conclusions: These findings add weight to the case for PPI in clinical trials by indicating that it is likely to improve enrolment of participants, especially if it includes people with lived experience of the health condition under study. Further research is needed to assess which types of PPI work best in particular contexts, the cost effectiveness of PPI, the impact of PPI at earlier stages of trial design, and the impact of PPI interventions specifically targeting retention.},

note = {Source: Published in BMJ},

keywords = {Clinical Development / Trials},

pubstate = {published},

tppubtype = {article}

}