Clinical trials are extensive patient-based tests to see if promising treatments are effective and safe. Traditionally they have been in 3 phases starting with low doses, small numbers and patients with severe vision loss and a placebo group that receives a sham treatment. These are necessary but tedious, expensive and strictly controlled by drug registration bodies, the FDA in the USA and the EMA in Europe.

Retina South Africa has participated in a global phase 3 clinical trial for Stargardt Disease and continues to seek more participation. We are only hampered by the number of patients on our database and funding.

Clinical trials are a necessary way of safeguarding patients and ensuring new technologies are effective. Unfortunately, the high standards, and costs, have led to many promising treatments being abandoned by drug companies. As most IRD’s are considered Orphan or Rare diseases, ways to fast-track both testing and registration are being instituted.

The recent death of patients using mRNA editing for Sickle Cell disease shows that safety should still be the most important issue in clinical trials and long term follow up of patients.