Rare diseases can’t wait. Rather than sit on the sidelines, many patients are taking the initiative to do what they can to accelerate the diagnostic process, improve understanding of their disease and spark research into potential therapies.
The 2023 NEXT Report from nonprofit advocacy group, Global Genes, shares stories of these efforts. It serves as recap of recent accomplishments and a reminder of the significant impact individual patients and caregivers can make. There’s Ryan Colburn who proved that his rare disease is much less rare than the science community once believed. The report also features Terry and Georgia Pirovolakis who raised $2.7 million to develop a gene therapy for their son and others with his rare disease.
“The growing sophistication among rare disease patient advocates about the drug development process, along with emerging technologies from genetic medicine to artificial intelligence, is giving unparalleled power to patient advocates to change the destiny for themselves and others,” Global Genes CEO Charlene Son Rigby wrote in the introduction to the 2023 NEXT Report.
The report includes 10 lessons from next-generation advocates. Here are three of them:
1. Build a community, because there’s strength in numbers.
2. Becoming the expert on your disease can have a significant impact
3. Engage with a scientist to reimagine what’s possible for treatment