Current Edition

The Value of Patient Advocacy Groups in Rare Disease Development: A Path to Strong Patient Engagement

Clinical trials in therapeutic areas targeting rare diseases are riddled with operational difficulties, with patient recruitment being arguably the most challenging aspect. By definition, a rare disease occurs in fewer than 200,000 people in the US (and less than 250,000 in the EU). Cinzia Dorigo, Syneos Health, discusses why patient advocacy groups (PAGs) are therefore vital partners for sponsors in engaging with hard-to-find patients and their caregivers.