Rare Disease: Taking a Different Approach

In Orphan Drug Congress by Paul Gilbertson

A Challenging Market Dynamic

An estimated 400 million people worldwide are currently living with rare diseases. There are approximately 7,000 different rare diseases and disorders, with more being discovered every day.

According to the National Institutes of Health Office of Rare Disease Research, approximately 6% of the inquiries made to the Genetic and Rare Diseases Information Center (GARD) are in reference to an undiagnosed disease.

At best, the knowledge base about any individual rare disease is small, fragmented, and often the preserve of a relatively few thought leaders and experts in the field.

Moreover, information that does exist may be spread across a number of language bases and contain markedly different medical, social, and cultural assumptions and judgments.

This extends not only to the nature of the disease, but also to any diagnosis paths, all of which add to the challenges facing any company that is looking to develop a suitable drug or treatment.

Consequently, a far greater emphasis is placed on the role of the individual patient; the patient with a rare disease not only serves as a potential expert in their condition, but also has ongoing involvement and influence in the development of any treatment.

Find out more by downloading the full white paper

Diagnosis and patient-finding remain the biggest challenge, even after rare disease drug is approved, but why? Download the white paper below to find out more from Cello Health. Alternatively come and meet them at the annual World Orphan Drug Congress.