The CAB is a working group of Cystinosis Europe which is formed by patient groups for cystinosis in Europe, USA, Mexico, Russia South Africa and Australia. It is based on the EuroCAB principles as developed by Eurordis (https://www.eurordis.org/content/eurordis-community-advisory-board-cab-programme). A key plank of the CAB is to allow patient organisations to interact with researchers and healthcare companies in a formalised way to avoid any possibility of a research or healthcare company seeming to influence the activities of any individual patient organisation.
The CAB has a number of goals.
- We want to engage with pharmaceutical companies for clinical trials, drug development, educational materials and any other appropriate topics.
- We want to engage with early-stage research as part of PPI – Public and Patient Involvement in research.
- We want to engage with physicians/clinicians in any way that might be helpful. In particular, with physicians and clinicians, we would like to engage around research, drafting clinical guidelines, etc.
The CAB has a wide remit to engage on behalf of the cystinosis patient community. The members of the CAB are parents of children with cystinosis and adults who themselves have cystinosis. They are nominated to sit on CAB by their national cystinosis patient organisations. CAB members are educated and trained in clinical trials, drug development and research. The CAB can provide the patient voice and patient expertise.
In other disease areas, CABs have been able to provide recommendations that have ensured better recruitment and retention on clinical trials, recommendations on improvements in research study design, recommendations on improvements for educational materials, to name but a few. The CAB process has been shown to support faster progress through European Medicines Agency (EMA) and US Food and Drug Administration (FDA) processes.
The CAB can offer expertise on:
- patient outreach
- education on research
- clinical studies and their design
- criteria for participation
- informed consent forms and processes
- compassionate use programmes
- better retention of participants
The Worldwide Cystinosis Community Advisory Board is chaired by Anne Marie O’Dowd and co-ordinated by Denise Dunne.