The DevelopAKUre initiative leads on patient recruitment and retention for three clinical trials, 'SONIA 1' and 'SONIA 2' (phase II and III), as well as 'SOFIA' (cross-sectional observation study) to investigate a drug (nitisinone) that could be a potential treatment for the disease.
Since the society established in 2003, a database of 1000 patients has been built up, and now the society is focusing on retention of patients by organising patient workshops, events to link patients together and webinars.
The ultimate goal is to critically assess the drug and what effect it has on AKU patients- whether is is safe and has overarching positive benefits.
In addition patients were involved with the AKU Society for a period before the clinical trial began which allowed the society to educate them on the clinical trial process and also patients could provide input into the structure, design and implementation of the clinical trial.
Problems addressed by Initiative
Currently, most patients do not have access to nitisinone - previous research on the drug was stopped and so for a long period of time there has been poor, limited access.
Nitisinone is an enzyme inhibitor, which acts to interrupt the action of another enzyme in the tyrosine breakdown pathway. By stopping the breakdown at a safe point, AKU patients should not produce HGA, and can potentially avoid the symptoms of the disease.
In addition, the clinical trials and patient engagement provide a deeper understanding of the rare disease and help to form a patient community, which previously did not exist.
Involving patients in the lifecycle of medicine development