Experience: Rare & Orphan Diseases

Rare & Orphan Diseases Require Special Treatment

Experience: Rare & Orphan Diseases

A rare disease is one that affects a very small percentage of the population in any given country. An orphan disease is one that is “forgotten” by treatment – a disease for which there is no definitive, convincing treatment. In recent years, there has been a rapidly growing demand for trials in rare and orphan indications because of legislation in both the US and the EU that has established incentives to support research in these areas, making it more financially viable.

We recommend proactive recruitment and consideration of unique characteristics.

Both rare and orphan indications have some unique characteristics. Recruitment is always challenging and a high number of study sites are required. Case report forms are more complex and there may be some scientific limitations. Statistical significance may be limited as well so analysis may hinge on individual patients and historical comparison. Working with regulatory authorities throughout the process is critical.

For all these reasons and more, mental agility is required by investigators and trial clinicians. We have a strong history of working with these unique situations and the flexibility to build a study program that works.


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