Giving patients a voice in the development of new treatments
Different treatments will have different effects on patients. These can be positive, such as improvement of their health condition or its symptoms, or can be negative, such as side effects. Bringing the patient voice into the development of new treatments allows reporting of how the effects of a treatment impact patients’ lives and what matters most to them. This can be measured using tools called patient reported outcomes.
What are patient reported outcomes?
Patient reported outcomes are specially designed questionnaires completed by patients. They are designed to gather information on how a patient’s health conditions and treatments affect their lives.
Hearing loss in Alport syndrome
Alport syndrome is the second most common cause of inherited chronic kidney disease. As well as affecting the kidneys, it can also cause hearing loss and vision problems. Hearing loss affects up to 80% of people with Alport syndrome. This usually begins in childhood or adolescence and is usually more severe in males.
Meet Professor Devin Peipert
Devin is a Professor of Health Outcomes Measurement at the University of Birmingham. His research is on the use of patient reported outcomes in drug development and treatment decision making.
This Alport Research Hub grant of £250,000, funded by Kidney Research UK and the Stoneygate Trust, will mean Devin and his team can develop patient reported outcomes to gather information on how hearing loss affects the lives of people living with Alport syndrome. These could then be used in the development of new treatments.
“When developing new treatments, several measures will be used to look at how well the treatment works. For example, if this were a new treatment for kidney disease, we may look at the amount of protein in the urine. But what’s also important is understanding whether the treatment improves patients’ lives. We can only understand this by asking patients themselves. This information needs to be valued with the same importance as the other measures to inform the approvals process and ensure treatments that reach patients have the greatest impact on their lives.” Professor Devin Peipert
Understanding how hearing loss affects people living with Alport syndrome
Devin and his team will interview children, teenagers and adults with hearing loss caused by Alport syndrome. They will also interview the parents and caregivers of children. Through these interviews, Devin and his team will look at how hearing loss impacts patients’ lives, including communication, emotions, social life and wellbeing.
This will be used to inform the development of a set of patient reported outcomes specifically for people with Alport syndrome who have hearing loss. These will be tested with patients to make sure they are clear and understandable and reflect and measure their real-life experience well.
Using patient reported outcomes in new treatment development
Patient reported outcomes specific to people with hearing loss caused by Alport syndrome could be used to when developing new treatments to ensure treatments that have the biggest impact on people’s lives are supported to reach patients.
David Crosby, chief research officer at Kidney Research UK commented: “We are driving change across the rare and paediatric kidney disease research pipeline to accelerate the discovery and translation of new treatments. Embedding the patient voice is crucial to ensure new treatments have the greatest impact on the lives of patients. This research is an important step to achieving this aim and we look forward to hearing the results.”
Kidney Community
A new dedicated online forum for kidney patients and carers, helping people connect with others who might be going through similar experiences.
Co-developed with patients and carers, Kidney Community is a welcoming, safe space where you can share experiences, ask questions and find information and support.