Patient and Caregiver Experiences of CAR T Cell Therapy for Blood Cancer in the UK: A Qualitative Study.

Background: Advanced cell therapies, including chimeric antigen receptor T cell (CAR T cell) therapies, offer novel opportunities for the treatment of advanced blood cancers such as lymphoma and leukaemia. However, as these therapies are relatively new, there is limited information on the experiences of patients and informal caregivers of the treatment which may influence the uptake of these therapies. The aim of this qualitative study was to explore their experiences to facilitate the identification of specific issues that should be addressed to positively impact patient outcomes and experiences of care.

Implementation of patient and public involvement and engagement (PPIE) for the therapies for long COVID in non-hospitalised individuals (TLC) project.

Background: Patients, their family members and caregivers have firsthand experiences of living with or supporting someone living with a disease or medical condition. This knowledge by experience cannot be replaced by the knowledge acquired by clinicians, researchers, or other professionals through study and/or work. The Therapies for Long COVID in non-hospitalised individuals (TLC) research project was funded in the UK by the National Institute for Health and Care Research (NIHR) and UK Research and Innovation to investigate the impact of long COVID on affected individuals.

Recommendations to promote equity, diversity and inclusion in decentralized clinical trials.

Decentralized clinical trials involving the use of digital tools to facilitate remote research are gaining momentum. Rapid advancements in digital technologies have supported the adoption of these trials. These innovations facilitate virtual interactions between clinical trial teams and participants by making it easier to collect, transfer and store electronic data.

Development of a quality of life measure for left ventricular assist device recipients using a mixed methods approach.

Background: Left ventricular assist device (LVAD) recipients report symptom improvement but find adjusting to life with the LVAD challenging. These challenges are unique, and existing patient-reported outcome measures (PROMs) do not reflect their experiences. This study aimed to develop a culturally relevant quality of life PROM for use with LVAD recipients in future research, design evolutions and clinical practice.

Recommendations to address respondent burden associated with patient-reported outcome assessment.

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making.

[Guidelines for clinical trial protocols for interventions involving artificial intelligence: the SPIRIT-AI extensionDiretrizes para protocolos de ensaios clínicos com intervenções que utilizam inteligência artificial: a extensão SPIRIT-AI].

Objective.: To determine if there was an association between intrapartum stillbirths and both traveled distance for delivery and delivery care accessibility, assessing periods before and during the COVID-19 pandemic.

Methods.

[Reporting guidelines for clinical trial reports for interventions involving artificial intelligence: the CONSORT-AI extensionDiretrizes para relatórios de ensaios clínicos com intervenções que utilizam inteligência artificial: a extensão CONSORT-AI].

The CONSORT 2010 statement provides minimum guidelines for reporting randomized trials. Its widespread use has been instrumental in ensuring transparency in the evaluation of new interventions. More recently, there has been a growing recognition that interventions involving artificial intelligence (AI) need to undergo rigorous, prospective evaluation to demonstrate impact on health outcomes.

[Guidelines for clinical trial protocols for interventions involving artificial intelligence: the SPIRIT-AI extensionDiretrizes para protocolos de ensaios clínicos com intervenções que utilizam inteligência artificial: a extensão SPIRIT-AI].

The SPIRIT 2013 statement aims to improve the completeness of clinical trial protocol reporting by providing evidence-based recommendations for the minimum set of items to be addressed. This guidance has been instrumental in promoting transparent evaluation of new interventions. More recently, there has been a growing recognition that interventions involving artificial intelligence (AI) need to undergo rigorous, prospective evaluation to demonstrate their impact on health outcomes.

Healthcare professionals' views on the most important outcomes for non-infectious uveitis of the posterior segment: A qualitative study.

Background: Uveitis comprises a range of conditions that result in intraocular inflammation. Most sight-threatening uveitis falls into the broad category known as Non-infectious Posterior Segment-Involving Uveitis (PSIU). To evaluate treatments, trialists and clinicians must select outcome measures.

The effect of disease modifying therapies on fatigue in multiple sclerosis.

Introduction: Fatigue is one of the most common and debilitating symptoms in people with multiple sclerosis (PwMS). Disease-modifying therapies (DMTs) are currently the gold standard in the treatment of MS and their effectiveness has been assessed through randomized clinical trials (RCTs). However, there is limited evidence on the impact of DMTs on fatigue in (PwMS).

Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation.

Objectives: Real-world evidence (RWE) generation can be enhanced by including patient-reported outcomes (PROs). Methods for collecting and using PRO data in the real-world setting are currently underdeveloped and there is no international guidance specific to its use in this context. This study explored stakeholders' perspectives and needs for using PROs in RWE generation.

Considerations for patient and public involvement and engagement in health research.

Patient and public involvement and engagement (PPIE) can provide valuable insights into the experiences of those living with and affected by a disease or health condition. Inclusive collaboration between patients, the public and researchers can lead to productive relationships, ensuring that health research addresses patient needs. Guidelines are available to support effective PPIE; however, evaluation of the impact of PPIE strategies in health research is limited.

Identifying patient-valued outcomes for use in early phase trials of ocular surface disease interventions.

Background: Patient-reported outcomes (PROs) can be used to evaluate the impact of dry eye symptoms (DES) on daily life. Early-phase clinical trials provide an opportunity to evaluate PRO strategies. Existing measures identified through systematic review omitted important concepts that mattered to patients.

"I just wanted to speak to someone- and there was no one…": using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients.

Background: Advanced therapy medicinal products such as Chimeric antigen receptor T-cell therapy offer ground-breaking opportunities for the treatment of various cancers, inherited diseases, and chronic conditions. With development of these novel therapies continuing to increase it's important to learn from the experiences of patients who were among the first recipients of ATMPs. In this way we can improve the clinical and psychosocial support offered to early patient recipients in the future to support the successful completion of treatments and trials.

Non-Pharmacological Therapies for Post-Viral Syndromes, Including Long COVID: A Systematic Review.

Background: Post-viral syndromes (PVS), including Long COVID, are symptoms sustained from weeks to years following an acute viral infection. Non-pharmacological treatments for these symptoms are poorly understood. This review summarises the evidence for the effectiveness of non-pharmacological treatments for PVS.

Embedding patient-reported outcomes at the heart of artificial intelligence health-care technologies.

Integration of patient-reported outcome measures (PROMs) in artificial intelligence (AI) studies is a critical part of the humanisation of AI for health. It allows AI technologies to incorporate patients' own views of their symptoms and predict outcomes, reflecting a more holistic picture of health and wellbeing and ultimately helping patients and clinicians to make the best health-care decisions together. By positioning patient-reported outcomes (PROs) as a model input or output we propose a framework to embed PROMs within the function and evaluation of AI health care.

The role of patient-reported outcome measures in trials of artificial intelligence health technologies: a systematic evaluation of ClinicalTrials.gov records (1997-2022).

The extent to which patient-reported outcome measures (PROMs) are used in clinical trials for artificial intelligence (AI) technologies is unknown. In this systematic evaluation, we aim to establish how PROMs are being used to assess AI health technologies. We searched ClinicalTrials.

Reducing the pressures of outpatient care: the potential role of patient-reported outcomes.

The global demand for hospital treatment exceeds capacity.The COVID-19 pandemic has exacerbated this issue, leading to increased backlogs and longer wait times for patients. The amount of outpatient attendances undertaken in many settings is still below pre-pandemic levels and this, combined with delayed referrals, means that patients are facing delays in treatment and poorer health outcomes.

Electronic patient-reported outcomes in chronic kidney disease.

Considerable research and investment have focused on the use of electronic patient-reported outcomes (ePROs) in nephrology. However, systematic collection of ePROs to inform the care of patients with chronic kidney disease remains sporadic. A change in culture is needed to encourage their wider adoption in clinical practice.

Implementation of patient-reported outcome measures in real-world evidence studies: Analysis of ClinicalTrials.gov records (1999-2021).

Background: Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by collecting and using patient-reported outcomes (PROs), which can provide valuable information on the effectiveness, safety, and tolerability of health interventions from the patient perspective. This analysis aims to examine and summarise the utilisation of patient-reported outcomes measures (PROMs) in real-world studies.

Listening to the Patient Voice Adds Value to Cancer Clinical Trials.

Randomized clinical trials are critical for evaluating the safety and efficacy of interventions in oncology and informing regulatory decisions, practice guidelines, and health policy. Patient-reported outcomes (PROs) are increasingly used in randomized trials to reflect the impact of receiving cancer therapies from the patient perspective and can inform evaluations of interventions by providing evidence that cannot be obtained or deduced from clinicians' reports or from other biomedical measures. This commentary focuses on how PROs add value to clinical trials by representing the patient voice.

Systematic review of guidance for the collection and use of patient-reported outcomes in real-world evidence generation to support regulation, reimbursement and health policy.

Background: Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by the collection and use of patient-reported outcomes (PROs), which can provide valuable information on the effectiveness, safety, and tolerability of health interventions from the patient perspective. This systematic review aims to examine and summarise the available PRO-specific recommendations and guidance for RWE generation.