Rare Diseases & Orphan Drugs

The Role of Patient Advocacy Groups in Rare Disease Research

Patient advocacy groups play a critical role in rare disease research by advocating for patients and their families, raising awareness, and providing support. These groups facilitate communication between patients, researchers, and healthcare professionals, and help to prioritize research efforts based on the needs of the patient community. They also provide funding for research, host patient registries and biobanks, and advocate for policy changes to improve access to care and treatments. By amplifying the patient voice and driving research efforts, patient advocacy groups are essential in advancing our understanding of rare diseases and developing effective treatments for these complex conditions.