Building the patient community

F Raffai and O Timmis; Gene Therapy (3 May 2017) doi:10.1038/gt.2017.

There are many challenges in conducting rare disease research. The conditions are often poorly understood, small patient populations are dispersed around the world, and there are limited funding opportunities. Patient groups can serve as a key partner in overcoming these challenges, as they understand the impact of rare conditions on patients’ lives. This gives patient groups valuable scientific insights into the disease. This can be used to create research strategies, address research bottlenecks and directly fund research that appropriately addresses patient needs. Patient groups can also play a critical role in recruiting and retaining patients for clinical trials, which reduces time and resource waste. By partnering with patient groups, research teams can improve efficiency of research and best meet the needs of patients. Researchers can also play an important role in building and supporting patient groups to unlock these benefits.