Rare or “Orphan” disease refers to a disease or condition affecting less than 200,000 persons in the United States, but in reality most rare diseases are far less prevalent. Approximately one in ten Americans are affected by a rare disorder and nearly 7000 rare diseases are reported.
Rare diseases are challenging, often poorly understood, difficult to diagnose, recruit low number of patients to participate and often affect pediatric population. Identifying these patients for clinical studies is difficult and requires experienced groups in managing challenging indications.
Recruiting in some of the countries we cover for rare diseases may be more challenging due to fewer centers of excellence, often less experienced investigators and limited patients’ registries. Additionally, these patients may often to be sent out of the country for complex treatments which requires strong strategies to manage patients and their families throughout the study. At ARIANNE, we are accustomed to complexity and challenging indications.
ARIANNE covers countries e.g., MENA and Africa, known for their higher consanguinity and the potential for recruitment and cost effective solutions.
We provide support for the development of products for orphan indications very early in the process, from regulatory strategy development to preparing US and EU orphan drug designation to study conduct.
For each of the projects:
- We conduct comprehensive and robust feasibilities
- Identify and train investigators when required
- Provide continual support for our investigators involved in rare diseases studies
- Constant communication and availability with the sites, investigators but also patients’ families when required.