Community Advisory Board

The Worldwide Cystinosis Advisory Board has been formed by patient groups for cystinosis in Europe, USA, South Africa and Australia based on the EuroCAB principles as developed by Eurordis. The CAB is a working group of Cystinosis Network Europe.

The CAB has a number of goals.

  1. We engage with pharmaceutical companies for clinical trials, drug development, educational materials and any other appropriate topics.
  2. We want to engage with early-stage research as part of PPI – Public and Patient Involvement in research.
  3. 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 EMA and FDA processes.

The CAB can offer expertise on:

  1. patient outreach
  2. education on research
  3. clinical studies and their design
  4. criteria for participation
  5. informed consent forms and processes
  6. compassionate use programmes
  7. better retention of participants
  8. reporting on results
  9. autonomy and independence

While individual patient groups in individual countries will continue to engage with researchers, doctors and sometimes pharmaceutical companies, the CAB umbrella will have a role in formal engagements particularly around the areas mentioned above. These are issues which affect all those living with cystinosis, not just patients in one country. The cystinosis community wishes to work together for the benefit of all those living with cystinosis, as we feel with a strong community and a strong voice, we best serve them. We believe it should be – ‘nothing about us without us.’

The key principles of the CAB are:

  1. Neutral setting
  2. Confidentiality
  3. Transparency
  4. Sharing
  5. Openness
  6. Optimisation of research and development
  7. A joint action plan as an outcome of the meeting

The Worldwide Cystinosis Community Advisory Board is chaired by Anne Marie O’Dowd and co-ordinated by Denise Dunne.

For more information on the CAB, please contact annemarie.odowd@cystinosis.ie or denise.dunne@cystinosis-europe.eu.