Including the excluded: real-world precision medicine for chronic kidney disease patients

The exclusion of patients with chronic kidney disease (CKD) from clinical trials presents a common problem for clinicians managing patients in practice.  People with CKD are often ineligible for taking part in clinical trials on the basis of safety concerns. In fact, 85% of recent trials of therapies for the 5 most common cancer malignancies excluded patients with CKD.

But the problem is, in the real world, CKD - as defined by an abnormality in kidney function or structure, and measured through creatinine levels - is prevalent in approximately 13.4% of the global population.  In fact, in patients with cancer, reported prevalence levels range from 12% to 53% at cancer diagnosis.

So how do we as industry professionals address this gap in evidence-based medicine?  Especially when clinicians are turning to us for the answers.

This is exactly where real-world precision medicine can help.

The application of precision medicine for CKD patients

Precision medicine is an emerging healthcare approach that tailors disease prevention and treatment by accounting for variability in genes, environment, and lifestyle of an individual.  Large, longitudinal datasets enable robust observations and modelling to help with clinical decision-making.

According to The Precision Medicine Scotland Innovation Centre, the use of these large datasets and the right analysis of them can “enable better diagnostics and earlier intervention, optimal treatment selection and more effective medicine development”.

This real-world data can, therefore, be useful for considering treatment in patients with various aspects of kidney disease - the types of patients who have been excluded from clinical trials.  Examples from the University’s School of Medicine include the identification of adverse events from chronic pain killers in patients with CKD, the development of kidney injury risk prediction tools and assessing the impact of vaccination on patients receiving dialysis.  

Enhancing Randomised Controlled Trials with real world data

Randomised Controlled Trials are essential to evaluating a treatment’s efficacy and safety.  But when cohorts of patients, such as those with CKD, are deemed ineligible to participate, this leaves an absence of information. These unknowns make clinical decision-making in the real world difficult and there is a risk that people are missing out on treatment or receiving suboptimal treatment.

Using insights from real-world data

In addition to running a randomised control trial, insights can be collected from national healthcare data in a way that is very efficient and doesn’t take millions of pounds and several years to carry out.

Dr Samira Bell is a Consultant Nephrologist at the University of Dundee and she explains the role of precision medicine in practice:

"Using real-world data and linking datasets represents a significant way forward in addressing gaps from traditional RCTs.  In nephrology, our patients are often excluded from these large randomised control trials so you cannot translate the outputs of those trials to our patients. That is why using this data - data that is already available - and linking it together is an efficient way of answering questions but also, more importantly, changing practice and improving patient care.”

Clinicians at The University of Dundee’s School of Medicine, such as Dr Bell, are experienced in using healthcare data to answer questions that RCTs have not been able to address. This is due to the quantity and quality of health data that sits within the University’s Data Safe Haven.

Data safe haven provides reliable, secure data sets

For such a clinically oriented question, it is essential that the methodology and datasets are robust, reliable, and secure. For over a decade, Dundee’s Health Informatics Centre (HIC) has been operating as a Trusted Research Environment (or ‘Data Safe Haven’) on behalf of NHS Tayside, NHS Fife, and the Scottish Government. HIC was the first centre in Scotland to allow the secure use of eHealth data within a controlled Safe Haven environment. 

Many of the HIC’s core datasets provide longitudinal healthcare data extending back 30 years, for approximately 20% of Scotland’s population.  With the University of Dundee’s vast experience with managing and analysing data comes a unique level of health informatic expertise in close proximity to clinicians that treat and manage patients every day.  

About Dr Samira Bell

Dr Samira Bell is a Clinical Senior Lecturer and Consultant Nephrologist at the University of Dundee. Her research interests include the use of routinely collected healthcare data for renal epidemiology with a particular interest in acute kidney injury. She is also co-chair of the UK Renal Health Data Research Network (UKRHDRN) which enables joint working with industry.