Birmingham-led international guidance will help ensure research and treatments better reflect patients' priorities.
Birmingham researchers have led two impactful publications designed to improve how patients' experiences are measured and used in healthcare research, clinical trials and drug development.
Together, the publications, led by researchers from the University of Birmingham's Centre for Patient-Reported Outcomes Research (CPROR) and supported by the National Institute for Health and Care Research (NIHR) Biomedical Research Centre (BRC): Birmingham and the LifeArc Centre for Acceleration of Rare Disease Clinical Trials (ARDT), provide new guidance for collecting patient-reported outcomes in real-world studies and practical recommendations for incorporating clinical outcome assessments into rare disease drug development.
A new ISPOR – the leading professional society for health economics and outcomes research globally – Good Practices Report has been published in Value in Health, providing guidance on how patient-reported outcomes (PROs) should be collected and used in real-world studies.
The report involved an international task force of experts from academia, industry, regulatory agencies and patient organisations. It was co-chaired by Professor Melanie Calvert, Director of CPROR and Professor of Outcomes Methodology at the University of Birmingham, alongside Dr Angela Rylands, Global Head of Patient Centred Outcomes at pharmaceutical group Kyowa Kirin, and involved several members of the Birmingham CPROR team.
Dr Konrad Maruszczyk, Research Fellow at CPROR, said: "The utilisation of patient-reported outcomes in real-world studies can significantly enhance our understanding of disease burden and patients' experiences of living with their condition.
"Patient-reported outcomes also provide evidence of treatment effectiveness and tolerability in real-world settings. By offering direct patient insights, these data can inform regulatory and health technology assessment decisions, ultimately helping improve patient access to treatments."
The guidance sets out nine key recommendations covering areas such as patient partnership, study design, data quality, technology use and engagement with regulators and health technology assessment bodies. It is intended to support researchers generating real-world evidence from observational studies, registries and pragmatic trials.
Researchers from Birmingham have also led a new study, published in eClinicalMedicine, that provides recommendations for the use of clinical outcome assessments in rare disease drug development. The work was undertaken through the LifeArc Centre for Acceleration of Rare Disease Trials, a collaboration between the University of Birmingham, Newcastle University and Queen's University Belfast funded by LifeArc.
Rare diseases collectively affect an estimated 250–450 million people worldwide, yet measuring treatment benefit can be particularly challenging because patient populations are often small and conditions highly variable.
Drawing on a literature review and multi-stakeholder consultation involving patients, caregivers, researchers, industry representatives, regulators and health technology assessment experts, the team developed a series of recommendations to help researchers select, develop and implement meaningful outcome measures in rare disease trials.
The recommendations cover areas including stakeholder engagement, selecting fit-for-purpose outcome measures, the use of digital technologies, integration of patient experience data and early engagement with regulators and decision-makers.
Professor Olalekan Lee Aiyegbusi, study lead, Professor of Patient-Centred Research at the University of Birmingham and co-lead of the NIHR Biomedical Research Centre: Birmingham’s Patient-reported outcomes research theme, said: "Rare diseases affect millions of people worldwide, yet developing and evaluating new treatments remains challenging because patient populations are often small and conditions can vary considerably from person to person.
"Clinical outcome assessments help capture how patients feel and function, providing evidence that is crucial for patient-focused drug development. These recommendations offer a practical framework to support researchers, industry, regulators and patient communities in generating meaningful evidence and ensuring that the outcomes measured in rare disease trials reflect what is most important to patients and families."
Professor Tim Barrett, Leonard Parsons Professor of Paediatrics at the University of Birmingham and Centre Lead for LifeArc ARDT added: “Prioritising patient-relevant outcomes is key to ensure that trials of new treatments in rare diseases address health issues of importance to people living with rare conditions. This work is a critical step forward towards including the patient voice in rare disease treatment trial designs.”
Professor Melanie Calvert, FMedSci, Professor of Outcomes Methodology and NIHR Senior Investigator at the University of Birmingham, and co-lead of the NIHR Biomedical Research Centre: Birmingham’s Patient-Reported Outcomes research theme, concluded: "These two publications reflect the growing importance of ensuring that the patient voice is embedded throughout the research pathway. By improving how we measure and use patient-reported outcomes, we can help deliver research that is more relevant, more inclusive and more impactful for patients."
Professor of Outcomes Methodology
Staff Profile for Professor Melanie Calvert who is Professor of Outcomes Methodology, School of Health Sciences at the University of Birmingham
Professor of Patient-Centred Research
Profile page for Dr Olalekan Lee Aiyegbusi, Associate Professor in the Department of Applied Health Sciences.
Leonard Parsons Professor of Paediatrics and Child Health
Staff Profile for Professor Timothy Barrett, Director of Centre for Rare Disease Studies, Department of Cancer and Genomic Sciences, College of Medicine and Health, University of Birmingham
