Putting patients at the center of this year’s Rare Disease Day

This week's Expert View is from Alicia Staley, chief patient officer at Medidata. She explores how innovations such as decentralized trials, remote monitoring, and synthetic control arms can improve patient access and trial efficiency, ultimately driving progress in rare disease drug development.

Rare Disease Day, held annually on 28th February, raises awareness of people living with rare diseases across the world, their families, and their carers. The key aims of the campaign are to achieve equitable access to diagnosis, treatment, and social opportunity for those affected by rare diseases.

There are over 300 million people with more than 6,000 identified rare diseases worldwide. To be classified as rare, a disease must affect fewer than one in 2,000 people. Given the relatively small numbers of people living with rare diseases, there are some universal challenges that members of this global community face.