Theme 3: Access and patient-reported outcomes

Current Landscape

We are entering a new era of advanced cell therapies for treatment of patients with blood disorders building on a long history of success in blood stem cell transplantation.  However, emerging data suggest that not all communities benefit from these therapies to the same extent.   Indeed, lack of participation in clinical trials for some patient populations can lead to 'health data poverty' and inability to extrapolate research outcomes to all patient groups.  There is therefore an important unmet need to understand why this might be the case and to devise strategies to improve outcomes for all patients.

ThEMe AIMS

We aim to identify health inequalities for advanced cellular therapies through interrogation of large anonymised clinical data bases.  We also aim to develop and validate a digital solution to efficiently capture the efficacy, safety, and tolerability of advanced cell therapies via patient-reported outcomes and wearables.

OUR EXperTISE and TEAM 

Professor Alastair Denniston leads research into the use of health data research and artificial intelligence to improve patient care in the ‘real world’.  Professor Melanie Calvert working with Drs. Lee Aiyegbusi, Sarah Walker and Karen Shaw have experience in outcomes research, including the use of composite outcomes and best practice for patient reported outcome assessment in clinical trials and routine practice. Professor Craddock and Dr. David Burns are part of the clinical team delivering CAR-T therapies and provide insight into clinical trial design.

OUR RESEARCH APPROACH

We are interrogating large, anonymised clinical data bases to evaluate population access to and outcome following advanced cellular therapeutics.  We are collaborating with King's College Hospital, London, to assess disparities in access to Chimeric Antigen Receptor T-cell (CAR-T-cell) treatment of Diffuse Large B-Cell Lymphoma.

CAR-T therapy is a new approach to cancer treatment in which the body's own immune cells are modified to recognise and kill off cancer cells. CAR-T therapy is promising but the side effects of treatment can be serious. It is important to catch side effects early so people can receive medicines to help treat them. One way of monitoring side effects is to ask patients to report their symptoms. Digital tools to assess CAR-T patients’ symptoms are not yet widely available.  We are conducting a research study to develop and test a new digital tool specifically designed to collect patients’ symptom and quality of life data reports following CAR-T therapy.