XII Jornada Anual Programa Hospital Clínic de Enfermedades Minoritarias

How to Integrate the Patient Voice in Rare Disease Clinical Trials
Patient engagement is key to successful commercialization of new drugs and medical devices across all disease areas, but it is particularly important in rare diseases where patients are often the most expert on their disease. | More

The Role of Patients in the Development of Rare Disease Solutions
Over 300 million people worldwide are impacted by a rare disease, whether they are patients, caregivers, or families. We spoke to Giulia Pierini, Associate Director, Patient Engagement at Alira Health, about why patient engagement is crucial in the development of products to treat rare diseases and how you can incorporate patient engagement practices into a patient-centric business strategy. | More

Regulatory Strategy for Registering Rare Disease Products in the EU: What You Need to Know
Discover the current Orphan Drug Program, its limitations, and the proposed amendments are essential for building a robust drug development and regulatory plan that secures a successful rare disease product registration in the EU market. | More

Impact of the Orphan Drug Act on Rare Disease Patients and the Pharmaceutical Industry
The Orphan Drug Act (ODA) changed the way pharmaceutical and biotech companies thought about rare diseases, and most importantly, generated the development of life-saving and life-altering treatments for millions of patients around the world. | More