Clinical trials aim to provide robust estimates of both the benefits and risks of healthcare interventions, by comparing them to standard care or a placebo.
Patient-reported outcomes (PROs) are increasingly assessed within clinical trials using questionnaires completed by patients. These questionnaires seek to capture the patient's opinion on the impact of their disease and treatment on their life. They are used to inform future patients about the potential symptoms or quality of life they may experience on a particular therapy, thereby helping individual patients and their clinicians choose the best treatment for them. This is particularly important in addressing patient questions such as ‘how am I likely to feel on this treatment?’
Patient-reported outcomes are often selected as primary, secondary or exploratory outcomes within clinical trials as they provide the patients' unique perspective on the physical, functional and psychological consequences of treatment and the degree and impact of disease symptoms.
If captured in a scientifically rigorous way, the patient-reported outcome results can provide valuable evidence to inform shared-decision making, labelling claims, clinical guidelines and health policy. However, evidence suggests that PRO content of clinical trial protocols is often suboptimal.
The Centre for Patient Reported Outcomes Research (CPROR) at the University of Birmingham aims to address this issue and optimise the use of PROs in clinical trials and routine care, to improve service delivery, enhance patient care and outcomes and ensure that the patient perspective is at the heart of health research and NHS decision-making.
Last month, members of the CPROR and international collaborators, funded by Macmillan Cancer Support, published new international guidelines providing recommendations for items that should be addressed and included in clinical trial protocols in which PROs are a primary or key secondary outcome.
Improved design of clinical trials including PROs will help ensure high-quality data to inform patient-centered care. We encourage use and uptake of the new guidance by researchers, funders, ethics committees, journal editors and patient partners involved in the co-design of research to endorse and improve the quality of PRO data from trials and maximise the benefits to patients.
The importance of this new Birmingham-led research is highlighted by Daniel O’Connor, Expert Medical Adviser at the Medical and Healthcare products Regulatory Authority (MRHA), who said he welcomed initiatives to facilitate the standardisation of PRO data in clinical trials, because it can contribute to a more robust patient-focused drug development strategy.
One patient reported that from their own personal experience of a serious illness and reporting their own outcomes, they were able to see how clinicians could use their PROs proactively, helping them to make the right decisions, which helped speed up treatment and recovery.
Patients really value this information and it is our duty to ensure high-quality data on symptoms and quality of life are available to them.
This work was funded by Macmillan Cancer Support (grant 5592105) and the University of Birmingham and was sponsored by the University of Birmingham. Drs Calvert, Kyte and Slade are funded by the NIHR Birmingham Biomedical Research Centre and teh NIHR Surgical Reconstruction and Microbiology Research Centre. Dr King is supported by the Australian government through Cancer Australia. Dr Blazeby is partially supported by the MRC ConDuCT-II Hub for Trials Methodology Research.