Real world data play an increasingly important role in informing decisions that affect patient access to new medicines. The patient perspective, in terms of outcomes and experience of healthcare intervention is a crucial component in defining value in a real world setting. Currently, however, the collection of patient-reported outcomes and experience within electronic health records, registries, or population health surveys may be suboptimal. Patient-reported outcomes which capture the patient’s views on the impact of disease and treatment on their physical, functional and psychological well-being, including symptoms and adverse events, can provide valuable evidence that is important to patients, clinicians, commissioners and providers. These data may be used pre-approval to establish unmet clinical need, the burden of disease and side effects of therapy. Post-approval these data can help demonstrate long term safety, effectiveness and value and help promote medicine optimisation and adherence. Selection of appropriate patient-reported outcome measures, a clear rationale for assessment and robust data collection (including e-PROs) to minimise missing data will be crucial to ensure high quality evidence. It is important that patient-reported outcomes are optimally integrated into real world data collection to ensure that the patient voice is central to decision making.
Professor Melanie Calvert has extensive experience in the design and analysis of clinical trials, trials methodology research, economic evaluation and clinical epidemiology. Professor Calvert leads the Patient Reported Outcomes (PROs) Research Group at the University of Birmingham, UK and has made several key advances in the knowledge of PROs in recent years including the development of the CONSORT-PRO extension. Her research team are conducting cutting-edge research on the management of PRO-alerts, embedding e-PROs in routine clinical practice and the development of a SPIRIT-PRO extension. Her research into PROs has reached international audiences, impacting on policy and educational guidelines.
Prof Calvert is a member of the International Society for Quality of Life Research Board of Directors, the Birmingham Institute for Translational Medicine Project Commissioning Board, the MRC HTMR Outcomes Working Group and is an expert advisor to the NIHR West Midlands Research Design Service. She has over 80 publications, including work in major international journals such as the BMJ, NEJM, Circulation and JAMA.