Exploring variation in adherence to clinical practice guidelines in rare diseases using Autosomal Dominant Polycystic Kidney Disease as a model to improve the quality and safety of healthcare
Dr Matthew Gittus
Area of studyImplementation
Host universityDivision of Population Health
University of Sheffield
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited disease within renal medicine with an estimated 70, 000 people being affected in the UK. There is only one disease modifying treatment for ADPKD, tolvaptan, which was approved by NICE in 2015. NICE published guidance on patient eligibility which was contextualised for clinicians by the UK Kidney Association. However, NHS digital data and anecdotal evidence suggests variation in prescribing practices of tolvaptan amongst UK renal centres. This requires further investigation as all patients in the UK should have equal access to tolvaptan given the potential to delay the onset of end stage renal disease.
A qualitative residual analysis is used as the framework for combining mixed methods throughout the project. The first phase with quantitative methods will use regression to identify factors affecting variation between a large set of cases provided by the National Registry of Rare Kidney Diseases (RaDaR) dataset. The second phase will involve qualitative research in the form of a multiple case study. The multiple case study will be based on cases identified in the first phase analysis. This will be supplemented by a quantitative survey of all renal departments in the UK to identify additional factors. The third phase will involve returning to initial data for any further analysis prompted by the qualitative research. This should enable the identification of factors that ought to be targeted with interventions to reduce the variation in prescribing practices of tolvaptan in UK renal centres.