Overcoming obstacles in rare disease clinical trials

Abstract

Overcoming obstacles in rare disease clinical trials With an urgent need to develop new treatments for people with rare diseases, the University of Birmingham’s Professor Timothy Barrett explains how a new consortium will provide a step change in the delivery of rare disease clinical trials. Amina was diagnosed with diabetes when she was four years old. Her parents were told she had type 1 diabetes, the commonest form in childhood, and learnt to inject her with insulin several times a day. They had many sleepless nights checking her blood glucose in case it went too low. Unfortunately, aged ten years, she began to lose her vision, and family photos showed her on holiday at the seaside using a white stick. She was diagnosed with Wolfram syndrome, a rare genetic disease. This is a progressive degenerative disorder without treatment, and people die prematurely from complications.