In rare disease research, every patient counts. Recruitment is challenging, retention even harder, and each data point is invaluable. Yet rigid trial protocols often create unnecessary barriers for patients, families, and sites alike. For rare conditions where participants are geographically...
Leslie Wetherell
Each year on September 17th, the World Health Organization recognizes World Patient Safety Day to raise awareness and promote action to reduce patient harm. This year’s theme, “Ensuring safe care for every newborn and child,” highlights the importance of protecting...
In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany...
Rare diseases are a distinctive and complex field within clinical development, characterized by significant hurdles from start to finish. Addressing these complexities requires innovative approaches to achieve a future of adaptability, quality, and efficiency. Here are 5 key steps to...