PROCEED: Patient Reported OutComEs in rarE Disease

Aim of Study

The overarching aim of this research is to develop a pathway for the optimal selection of PROs for inclusion within the Centre for Rare Disease clinical information platform.  Initial work in this area will focus on two diverse patient populations that receive clinical care within the new centre – (i) patients with Primary Sclerosing Cholangitis, and (ii) patients transitioning from child to adult renal transplantation services.


The Institute of Transitional Medicine (ITM) Birmingham have recently opened a new Centre for Rare Disease, which brings together multi-specialty and multi-disciplinary teams to deliver care of patients with rare diseases. The Centre will bring multiple benefits to patients, including a clinical information platform enabling collection of detailed information on patients, their disease, and their responses to treatment. Two patient groups receiving clinical care within the centre are: (i) patients with Primary Sclerosing Cholangitis, and (ii) patients transitioning from child to adult renal transplantation services.

Patient Reported Outcome

One way to access the burden and symptoms of PSC and to fully understand why  patients have poorer outcomes after renal transplant is through patient reported outcomes. PRO measures can capture a variety of concepts that are relevant to the patients, such as symptom frequency, severity or change; level of physiological functioning; health- related quality of life (HRQOL); treatment satisfaction; treatment side effects and treatment adherence.

There is a need to identify appropriate PRO measures for use in patients with rare disease and to determine how best to capture data that is meaningful to inform patient care and also acceptable and helpful to patients. The study will help inform selection of PRO for other diseases which can be used in the Institute of Transitional Medicine Centre for Rare Disease.

PROCEED phases of research

Phase 1- A systematic review of patient reported outcome measures used in patients with primary sclerosing cholangitis

Phase 2- Qualitative study to explore the experience of patients living with primary sclerosing cholangitis or renal transplant and their views on using PRO measures in the NHS to support their care.

Research team

Professor Melanie Calvert (University of Birmingham, Director PRO Research Group)
Dr Derek Kyte (University of Birmingham, Deputy Director PRO Research Group)
Dr James Ferguson (Consultant Hepatologist, University Hospitals Birmingham)
Dr Tanya Pankhurst (Consultant in Nephrology and Electronic Patient Record,University Hospitals Birmingham)
Dr Larissa Kerecuk (Consultant Paediatric Nephrologist, Birmingham Children's Hospital)

Contact us

Research Fellow

Dr Fatima Isa
Telephone: +44 (0) 121 414 2688