Gaucher Registry for Development, Innovation & Analysis of Neuronopathic disease
GARDIAN was borne out of the recognition that a global disease registry was needed to support a better understanding of the natural history of the disease, correlate global phenotypes and genotypes, validate new endpoints/outcomes and to generate a data source that can be used for both Regulatory and Health Technology Assessments evaluation of emerging drugs for neuronopathic Gaucher disease.
GARDIAN will be patient led and owned and enable the IGA as a global community to drive research and ensure that with an increase in the development of potential therapies for nGD that we work together with researchers and pharma to have one disease registry.
Our Vision for GARDIAN
We will strive to develop a registry that will capture clinical data from physicians/HCPs treating nGD patients and patient (caregiver)-reported data.
Our vision is that patients will be able to enrol in the registry as site patients (by their clinicians) and non-site patients (through our patient engagement portal) wherever you live in the world.
As we roll out GARDIAN, we will launch phase 1, this will be to collect patient and caregiver added data ONLY. We are working towards capturing clinical data, however this will take time and further investment, we are working hard towards this.
Using the information, we collected in our feasibility study interviews with patients and caregivers and clinical experience with patients we have designed a nGD-specific patient-reported outcome (PRO) and caregiver-reported outcome (ObsRO). Through GARDIAN we want patients and caregivers to add their own data through a questionnaire that asks quality of life questions to capture the wider disease impact. We will also use a number of other questionnaires that will capture information about quality of life.
GARDIAN will be translated into 7 languages to start with, these will be UK English, US English, Arabic, Japanese, Chinese, German and French.
Before we start collecting patient data, we need to undertake some testing of the PROs and ObsRo to make sure that the questions are easily understood by patients and caregivers. We will do this with nGD volunteers in several countries. Once this has been completed and the questions finalised, we will launch GARDIAN to the nGD global community and start collecting your data.
When the registry portal opens a link will be added here so that you can find out more about how to join GARDIAN and register your interest. In the meantime, please email Tanya Collin-Histed.