While there is a growing recognition that the world of rare disease is different and comes with unique challenges, the body of knowledge around how to address those challenges is very much a work in progress. At rareLife solutions, we’re helping to build that knowledge base by sharing what we’ve learned from our work with patients, advocates, caregivers, healthcare professionals, treatment industry leaders, and beyond. We share our thinking with the community here and via email. To be added to our email list and get future resources directly in your inbox, please get in touch.
Patients, advocates, and caregivers are the market-shapers in the world of rare disease. A powerful rare disease community can improve drug development and commercialization, influence regulatory decisions, shape treatment paradigms, and educate others about the disease. What’s more, survey data show that a significant majority of patients would provide feedback to drug developers, and would choose treatments from companies that are engaged with patients over those that are not.
Our most recent Perspective explores these topics, along with the potential impact that patient, advocate, and caregiver feedback can have during Food and Drug Administration (FDA) regulatory processes; the value of an engaged community in clinical trial recruitment, and more.
He who masters the power formed by a group of people working together has within his grasp one of the greatest powers known to man.Idowu Koyenikan
— Author of “All You Need is a Ball: What Soccer Teaches Us About Success in Life and Business”