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...
Expertise: Rare Disease 80 results
Rare disease trials face some of the most complex challenges in drug development. Patient populations are small and geographically dispersed. Protocols are often demanding, with intensive procedures and long travel requirements. Families are already carrying the burden of managing a...
In recent years, the pharmaceutical landscape for rare diseases has witnessed a remarkable evolution. Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than 300 million people worldwidei and account for a...
Rare Disease
Paulla Dennis
In rare diseases, patient opinion and insight often go unheard. However, for sponsors of rare disease clinical trials, study success often rests on incorporating the patient perspective as early as possible in development. Many patients with rare diseases have undertaken...
Rare Disease