Clinical Trials Units (CTUs) in the UK are required to integrate patient and public involvement (PPI) into their research and operational strategies to meet national standards and registration criteria. While PPI in individual clinical trials is increasingly documented, strategic-level involvement within CTUs remains underreported. The Medical Research Council Clinical Trials Unit (MRC CTU) at University College London (UCL) has embedded PPI across its organisational strategy, partnering with PPI representatives to co-develop and deliver annual PPI implementation plans aligned with a five-year communications strategy. Since 2016 the MRC CTU at UCL has collaborated with its PPI Group, made up of staff and PPI representatives, to design and execute annual implementation plans that operationalise strategic PPI objectives. The 2024/25 plan included 18 activities, ranging from training and guidance development to evaluation and dissemination. All activities involved PPI representatives, with three led by them. Progress was monitored through predefined indicators and discussed in quarterly meetings. By year-end, 12 activities were fully achieved, five were underway, and one was postponed. Notable successes included high satisfaction with training sessions and the release of a podcast featuring PPI members. Challenges included delays in finalising a new participant information sheet template as a result of extensive internal review. The introduction of role descriptions for PPI representatives improved transparency and engagement for all parties. The MRC CTU at UCL’s strategic, co-designed approach to PPI fosters genuine partnership and integration of public involvement across the Unit. Annual implementation plans, collaboratively developed and evaluated, ensure alignment with long-term objectives and responsiveness to challenges. This model demonstrates how sustained, cross-cutting PPI can be effectively embedded in research infrastructure, contingent on core funding support. The approach offers a replicable framework for other CTUs aiming to enhance the impact of their strategic PPI. Not applicable. This paper explores how patients are involved in decision-making at a strategic level in an academic clinical trials unit. It looks at how working together with patients helps the unit achieve its broader goals for patient and public involvement (PPI) beyond individual trials, and how working together supports the unit’s communications strategy. It also looks at how patients help track progress and judge results. The Medical Research Council Clinical Trials Unit at University College London is a large academic clinical trials unit delivering studies in cancer, infectious diseases and neurodegenerative diseases in and beyond the UK. For over twenty years it has involved patients in its research, starting with individual studies and gradually expanding to other areas like reviewing trial protocols. In 2016 the PPI group (made up of staff and PPI members) started to design and deliver annual plans to embed PPI in all the unit’s activities beyond the trials themselves. The 2024-25 unit PPI implementation plan had 18 activities, including training, writing guidance and sharing knowledge. PPI members were involved in all activities, and led three of them. The PPI group measured how well the plan was put into practice. This paper describes a model for long-lasting PPI that is built in effectively to the research organisation. It offers a framework for other CTUs aiming to enhance the value of their strategic PPI, and for PPI members seeking to widen their influence and involvement.

In a 2022 consultation, the UK public highlighted the need to disseminate trial results to participants. We assess whether the information provided to trial participants in publicly available participant information sheets (PISs) of trials conducted in the UK is helpful for future trials. This cross-sectional study is based on a search conducted on 18 August 2023 on ClinicalTrials.gov looking for UK completed or terminated phase 2-4 medicine trials. The posted PIS (or the protocol, if the PIS was unavailable) were reviewed checking the text used to inform participants on how results will be disseminated to participants. Of the 48 records retrieved, 32 were included: 23 and 9 had the PIS or the protocol posted, respectively. Seven (22%) did not mention dissemination of results to participants. Thirteen (41%) used the same short "common, standard text" of four sentences to inform participants. This text mentioned ClinicalTrials.gov as the source for further information and US Law as the reason for it. Twelve (38%) used different texts with different scopes and lengths. These results showed that publicly available PISs of medicinal product trials conducted in the UK are very limited and of scarce utility for investigators aiming to start a new trial.

Optimising the use of approved drugs requires evidence from post-approval trials that investigate variations of their use. Determining optimal drug use goes beyond the dominant, academic effort to conduct trials to identify effective lower doses of new drugs. Other important therapeutic approaches that use either less, similar, or more drug than the standard dose need testing in clinical trials, to get the most out of these drugs. Trial objectives on survival outcomes vary greatly; some aim for superiority, others for equivalent exposure or non-inferiority. This Personal View aims to inform academic trialists in how to conceive and prioritise questions aimed at determining the optimal use of drugs, taking into account the perspectives of patients, clinicians, and trial funders, to maximise the chances of successful delivery and impact for patients globally.

Key points Patient authorship is a new topic in medical publishing, with the number of patient‐authored publications growing rapidly. Publication stakeholders (e.g., editors, publishers, funders, researchers) should be aware of the legitimacy of patient authorship. Practical actions can be taken by different publication stakeholders to encourage and support patient authorship—ethically and effectively. Patient authorship can contribute to enhancing diversity, equity, and inclusion in medical publishing. Research on patient authorship could be strengthened with consensus on publication tagging practices (e.g., affiliation terms) and the use of artificial intelligence. A plain language summary of this article and the GRIPP2 form for reporting patient involvement are available as supplementary information.

Traditionally, patients with asymptomatic, advanced-stage follicular lymphoma were managed with a watchful-waiting approach until disease progression. The 'Watch and Wait' Phase-3 randomised international trial examined whether rituximab could delay the need for treatment and the effect on quality of life (QoL). In this article, we present the long-term results of the QoL aspect of the trial. Patients were randomised to watchful-waiting (Arm A), rituximab induction (Arm B) or rituximab induction followed by maintenance (Arm C). We present the QoL outcomes from 180 patients (Arm A), 188 patients (Arm C) and an exploratory analysis of 82 (Arm B) compared to 81 and 84 patients concurrently randomised to arms A and C. Arm C reported greater improvement in emotional well-being overtime (Month 37, p = 0.0078) and were significantly more likely to feel in control of their situation than watchful-waiting patients (Month 25, p = 0.0004; Month 37, p = 0.0476). Watchful-waiting patients were significantly more likely to avoid thinking about their illness, did not find learning about their illness helped them and were more likely to attach unpleasant connotations to clinic visits (Month 7, p = 0.0032; Month 13, p = 0.0015; Month 25, p = 0.0104). These results demonstrate improved QoL scores in the induction and maintenance rituximab arm, indicating that rituximab was not detrimental to QoL and resulted in an improved QoL in some domains.

Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported. The REMoDL-B phase III adaptive trial compared rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone (R-CHOP) versus R-CHOP + bortezomib (RB-CHOP) in patients with diffuse large B-cell lymphoma (DLBCL), stratified by molecular subtype. Primary analysis at a median follow-up of 30 months found no effect of bortezomib on progression-free survival (PFS) or overall survival (OS). Retrospective analysis using a gene expression–based classifier identified a molecular high-grade (MHG) group with worse outcomes. We present an updated analysis for patients successfully classified by the gene expression profile (GEP). Eligible patients were age older than 18 years with untreated DLBCL, fit enough for full-dose chemotherapy, and with adequate biopsies for GEP. Of 1,077 patients registered, 801 were identified with Activated B-Cell (ABC), Germinal Center B-cell, or MHG lymphoma. At a median follow-up of 64 months, there was no overall benefit of bortezomib on PFS or OS (5-year PFS hazard ratio [HR], 0.81; P = .085; OS HR, 0.86; P = .32). However, improved PFS and OS were seen in ABC lymphomas after RB-CHOP: 5-year OS 67% with R-CHOP versus 80% with RB-CHOP (HR, 0.58; 95% CI, 0.35 to 0.95; P = .032). Five-year PFS was higher in MHG lymphomas: 29% versus 55% (HR, 0.46; 95% CI, 0.26 to 0.84). Patients with ABC and MHG DLBCL may benefit from the addition of bortezomib to R-CHOP in initial therapy.

The role of patients in cancer research is undergoing a significant evolution as all stakeholders seek to enhance the level of direct patient involvement in the design and development of clinical trials. However, there are significant hurdles that patients, patient advocates, laboratory researchers, clinical investigators, and funding institutions must overcome to implement relevant patient involvement in all aspects of biomedical research. By using innovative grant funding models, philanthropic organizations can lead the field in overcoming these challenges. Rising Tide Foundation for Clinical Cancer Research (RTFCCR), a private philanthropy that funds academic research, has developed a novel approach for requiring and supporting partnerships among grantees and patients in designing and conducting research projects. This paper presents a reflective case study of efforts to advance the field of patient involvement in clinical research.The decision to focus on patient involvement stems from an expressed focus area established by the RTFCCR board of directors. In conducting this work, RTFCCR partnered with Patvocates, a patient advocacy and engagement network, to create a set of guiding documents and resources aimed at public and private health research funders within various national, international, and therapeutic settings. This effort included a landscape assessment, interviews with experts, and an iterative development process.To date, RTFCCR has completed and disseminated three guiding documents, one for funders, one for grant applicants, and one for patient advocates. These resources have already generated three major ongoing initiatives at RTFCCR: (1) inclusion of these recommendations in the foundation's funding guidelines; patient input to prioritization of research focus areas; and in topic selection for calls for proposals; (2) direct involvement of patient experts in the grant review process; and (3) a commitment to support high impact clinical research projects in Low- and Middle-Income Countries. Moreover, the foundation has launched a partnership with the International Cancer Research Partnership, the global alliance of cancer research organizations.By using its grantmaking function and developing standardized approaches for implementation of patient involvement, RTFCCR is seeking to advance patient-centric cancer clinical research. This approach will continue to develop as it is implemented and shared with partners throughout the world.The Rising Tide Foundation for Clinical Cancer Research (RTFCCR), a private philanthropy that funds academic research, has developed a novel approach for requiring and supporting partnerships among grantees (scientists) and patients in designing and conducting research projects.The decision to focus on patient involvement stems from an expressed focus area established by the RTFCCR board of directors. In conducting this work, RTFCCR partnered with Patvocates, a patient advocacy and engagement network. Patvocates conducted a landscape assessment, interviews with experts, and their collective experience as patient advocates. This work generated a set of guiding documents and resources. These resources are to help public and private health research funders to better understand current challenges and support scientists and patients through their funding mechanisms. Three guiding documents, one for funders, one for grant applicants, and one for patient advocates are now available for download at the RTFCCR website: https://www.risingtide-foundation.org/clinical-cancer-research/patient-engagement#start Delivering a paradigm change involves not only the introduction of additional requirements and rules, but also enhanced education of patients and investigators. By using its grantmaking function and developing standardized approaches for implementation of patient involvement, RTFCCR is seeking to advance patient-centric cancer clinical research.Development and implementation of consistent policies and procedures for the integration of the patients’ view in the design and review of research proposals is needed for funders as well as for research institutes, both public and private.

Background Early phase dose-finding (EPDF) trials are crucial for the development of a new intervention and influence whether it should be investigated in further trials. Guidance exists for clinical trial protocols and completed trial reports in the SPIRIT and CONSORT guidelines, respectively. However, both guidelines and their extensions do not adequately address the characteristics of EPDF trials. Building on the SPIRIT and CONSORT checklists, the DEFINE study aims to develop international consensus-driven guidelines for EPDF trial protocols (SPIRIT-DEFINE) and reports (CONSORT-DEFINE). Methods The initial generation of candidate items was informed by reviewing published EPDF trial reports. The early draft items were refined further through a review of the published and grey literature, analysis of real-world examples, citation and reference searches, and expert recommendations, followed by a two-round modified Delphi process. Patient and public involvement and engagement (PPIE) was pursued concurrently with the quantitative and thematic analysis of Delphi participants’ feedback. Results The Delphi survey included 79 new or modified SPIRIT-DEFINE ( n = 36) and CONSORT-DEFINE ( n = 43) extension candidate items. In Round One, 206 interdisciplinary stakeholders from 24 countries voted and 151 stakeholders voted in Round Two. Following Round One feedback, one item for CONSORT-DEFINE was added in Round Two. Of the 80 items, 60 met the threshold for inclusion (≥ 70% of respondents voted critical: 26 SPIRIT-DEFINE, 34 CONSORT-DEFINE), with the remaining 20 items to be further discussed at the consensus meeting. The parallel PPIE work resulted in the development of an EPDF lay summary toolkit consisting of a template with guidance notes and an exemplar. Conclusions By detailing the development journey of the DEFINE study and the decisions undertaken, we envision that this will enhance understanding and help researchers in the development of future guidelines. The SPIRIT-DEFINE and CONSORT-DEFINE guidelines will allow investigators to effectively address essential items that should be present in EPDF trial protocols and reports, thereby promoting transparency, comprehensiveness, and reproducibility. Trial registration SPIRIT-DEFINE and CONSORT-DEFINE are registered with the EQUATOR Network ( https://www.equator-network.org/ ).

The UK Secretary of State for Health and Social Care, Stephen Barclay, has set out plans for an alternative approach to cancer service provision based on a major conditions strategy.1 The six areas for inclusion in the strategy are cancer, cardiovascular disease, respiratory disease, dementia, mental health, and musculoskeletal disorders. All these areas are priorities for a national health strategy but is lumping them all together an effective route to success in treating them? As patients and patient advocates, we do not consider that this approach is wise, either for people with these diseases or for the National Health Service (NHS).

BACKGROUND:Patient and public involvement in health economic evaluation is still relatively rare, compared to other areas of health and social care research. Developing stronger patient and public involvement in health economic evaluation will be important in the future because such evaluations can impact on the treatments and interventions that patients can access in routine care.MAIN TEXT:The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) is a reporting guideline for authors publishing health economic evaluations. We established an international group of public contributors who were involved in the update of the CHEERS 2022 reporting guidance, ensuring two items (areas of reporting) specifically about public involvement were included. In this commentary we focus on the development of a guide to support public involvement in reporting, a key suggestion made by the CHEERS 2022 Public Reference Group, who advocated for greater public involvement in health economic evaluation. This need for this guide was identified during the development of CHEERS 2022 when it became apparent that the language of health economic evaluation is complex and not always accessible, creating challenges for meaningful public involvement in key deliberation and discussion. We took the first step to more meaningful dialogue by creating a guide that patient organisations could use to support their members to become more involved in discussions about health economic evaluations.CONCLUSIONS:CHEERS 2022 provides a new direction for health economic evaluation, encouraging researchers to undertake and report their public involvement to build the evidence base for practice and may provide some reassurance to the public that their voice has played a part in evidence development. The CHEERS 2022 guide for patient representatives and patient organisations aims to support that endeavour by enabling deliberative discussions among patient organisations and their members. We recognise it is only a first step and further discussion is needed about the best ways to involve public contributors in health economic evaluation.

Patient advocates are increasingly authoring peer-reviewed publications that could enhance patient care and understanding of the lived experience. Although patient authorship may be seen as an innovation in the peer-reviewed publication environment and some may not be aware of or accept patient authorship, we know patient-authored publications exist. However, identifying patient-authored publications is often challenging and time-consuming. In this commentary, we propose a definition for a patient author and patient-authored publications. We outline factors driving the increase in patient authorship, including patient interest, recognition of the value of including the patient voice and major funders recognising the importance of involving patient advocates in research. Evidence and experience-based guidance on patient authorship is emerging, and we highlight practical guidance for patient advocates on authoring peer-reviewed publications. To gain a better understanding of patient authorship, an efficient method is needed to identify patient-authored publications. A dataset on patient-authored publications could be used for a range of quantitative and qualitative research studies. The affiliation search function in PubMed can provide an easy, and reproducible way to identify a dataset of patient-authored publications in the international peer-reviewed literature, but only if patient authors include a standard metatag, (e.g. Patient Author) as one of their listed affiliations, combined with other affiliations as appropriate. From 2020 to 2021, there was a nine-fold increase in patient-authored publications in PubMed identified using the Patient Author tag. We recognize that terminology can be contentious and some authors may prefer alternative metatags. Further efforts are required to gain consensus on a suitable, standard metatag or set of metatags to use to show the true extent of patient authorship. Patient authorship is not only legitimate, but it also exemplifies the principles of diversity, equity and inclusion. Stakeholders in the publication community need to review their policies and procedures to identify and address barriers to patient authorship. Patient advocates, funders, researchers and publishers could all help to promote awareness and acceptance of patient authorship and the merits of using a standard metatag or set of metatags, so that patient-authored publications are no longer hidden in plain sight. Some patients are leading or helping with medical research to improve understanding of their condition and patient care. To share research findings, patients can author articles published in scientific journals. These articles are reviewed by experts and are known as peer-reviewed publications. Patient authors can provide unique and valuable insights from their experience of living with a condition. Demonstrating that patients can be authors would be easier if there was a quick way to find patient-authored publications. In this article, we describe who a patient author is and what patient-authored publications are. We identify factors that may encourage patients to author research publications. We highlight the practical guidance available to help patient authors and those working with them. To help future research about patient authorship, we need a way to find patient-authored publications. One way is for patients to include a standard search term, such as ‘Patient Author’ in the affiliation section of their publication. Like all authors, patient authors can list more than one affiliation, such as their workplace if they wish. We used the ‘Patient Author’ search term to look at publications in PubMed, a free resource to access scientific publications. We found the number of patient-authored publications using the ‘Patient Author’ tag increased nine times from 2020 and 2021. We encourage patients, funders, researchers and publishers to use a standard metatag or an agreed set of metatags. This could make it easier to find and raise awareness of patient-authored publications.

Introduction: The REMoDL-B trial compared R-CHOP versus R-CHOP + bortezomib (RB-CHOP) in patients with diffuse large B-cell lymphoma (DLBCL) stratified by cell of origin (COO) determined by gene expression profile (GEP). The primary analysis of the trial at a median follow-up of 30 months found no overall difference in progression free (PFS) or overall survival (OS) by treatment arm. Retrospective analysis using a gene expression based classifier identified a molecular high grade group (MHG) with distinct molecular features. We present an update of this analysis and the trial final results for patients with lymphomas of activated B-cell (ABC), germinal-centre B-cell (GCB) or MHG type. Methods: REMoDL-B was an open label randomised phase 3 adaptive trial with patients recruited from UK and Switzerland. Eligible patients had untreated, histologically confirmed DLBCL stage I-IV, were >18 year old, ECOG performance status≤2, and had cardiac, lung, renal, and liver function sufficient to tolerate full dose chemotherapy. Patients with sufficient material from initial biopsies for GEP were randomised between R-CHOP or RB-CHOP from cycle 2, stratified by international prognostic index (IPI) and COO. COO classification was performed in the trial with the DLBCL automatic classifier in time for randomisation and reassessed retrospectively adding in the MHG classifier. Time to event analyses were performed using the Kaplan-Meier method and cox regression modelling. REMoDL-B was coordinated by the Southampton Clinical Trials Unit and funded by a grant from Janssen-Cilag and endorsed by Cancer Research UK after independent peer review (C328/A12128). Results: 801 of the 1,077 patients registered were identified as ABC, GCB or MHG type in the final analysis. 128 were unclassified and in 148 the diagnostic biopsy sample was insufficient. Median age was 65 (range 20-86) and 67% were stage III/IV, 48% had abnormal LDH, 29% had bulk, 17% had high IPI and 30% high intermediate IPI, 31% ABC, 59% GCB and 10% MHG. At a median follow up of 64 months in those still alive, there was no overall benefit of bortezomib on PFS or OS (5-year PFS of 64% in R-CHOP and 70% in RB-CHOP HR, 0.81 p=0.085, 5-year OS of 76% vs 79% HR, 0.86; p=0.32). Improved PFS and OS with RB-CHOP was however seen in ABC patients; 5-year PFS of 54% with R-CHOP vs 69% with RB-CHOP (Figure 1); 5-year OS of 67% in the R-CHOP arm vs 80% in the RB-CHOP arm: OS HR 0.58; (95% CI, 0.35 to 0.95) p=0.032. PFS was significantly improved in MHG patients: 5-year PFS 29% with R-CHOP vs 55% with RB-CHOP: HR 0.46 (95% CI 0.26-0.84) p=0.011 (Figure 2). 5-year OS was 48% vs 60% HR, 0.62; 95% (CI, 0.32 to 1.20) p=0.156. Outcomes by LymphGen classification will be presented. Adverse events were similar to those reported previously, with the most common grade 3 or worse being haematological toxicity, reported in 38% R-CHOP patients and 42% RB-CHOP patients. Conclusions: Gene expression profiling can identify subtypes of DLBCL which may benefit from the addition of bortezomib to R-CHOP in initial therapy, specifically, ABC and MHG types. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

65-74 years, Sex category (female) CIED Cardiac implantable electronic device CML Chronic myeloid leukaemia CMR Cardiac magnetic resonance COMPASS-CAT Prospective COmparison of Methods for thromboembolic

Abstract Background Patient-reported adverse events may be a useful adjunct for assessing a drug’s tolerability in dose-finding oncology trials (DFOT). We conducted surveys of international stakeholders and the National Cancer Research Institute (NCRI) Consumer Forum to understand attitudes about patient-reported outcome (PRO) use in DFOT. Methods A 35-question survey of clinicians, trial managers, statisticians, funders, and regulators of DFOT was distributed via professional bodies examining experience using PROs, benefits/barriers, and their potential role in defining tolerable doses. An 8-question survey of the NCRI Consumer Forum explored similar themes. Results International survey: 112 responses from 15 September–30 November 2020; 103 trialists [48 clinicians (42.9%), 38 statisticians (34.0%), 17 trial managers (15.2%)], 7 regulators (6.3%), 2 funders (1.8%)]. Most trialists had no experience designing (73, 70.9%), conducting (52, 50.5%), or reporting (88, 85.4%) PROs in DFOT. Most agreed that PROs could identify new toxicities (75, 67.0%) and provide data on the frequency (86, 76.8%) and duration (81, 72.3%) of toxicities. The top 3 barriers were lack of guidance regarding PRO selection (73/103, 70.9%), missing PRO data (71/103, 68.9%), and overburdening staff (68/103, 66.0%). NCRI survey: 57 responses on 21 March 2021. A total of 28 (49.1%) were willing to spend <15 min/day completing PROs. Most (55, 96.5%) preferred to complete PROs online. 61 (54.5%) trialists and 57 (100%) consumers agreed that patient-reported adverse events should be used to inform dose-escalation decisions. Conclusion Stakeholders reported minimal experience using PROs in DFOT but broadly supported their use. Guidelines are needed to standardize PRO selection, analysis, and reporting in DFOT.

Patient-reported outcomes (PROs) are used in clinical trials to provide evidence of the benefits and risks of interventions from a patient perspective and to inform regulatory decisions and health policy. The collection of PROs in routine practice can facilitate monitoring of patient symptoms; identification of unmet needs; prioritisation and/or tailoring of treatment to the needs of individual patients and inform value-based healthcare initiatives. However, respondent burden needs to be carefully considered and addressed to avoid high rates of missing data and poor reporting of PRO results, which may lead to poor quality data for regulatory decision making and/or clinical care.