From the perspective of a clinical trial, a rare disease suggests the
available patient and site population is quite small and distributed
diffusely. Likewise, any challenge related to study logistics,
competitive landscape or patient recruitment is magnified as
compared to trials in other indications. Kamila Subaieva and Earl
Seltzer, at INC Research/inVentiv Health, discuss how major
regulatory agencies encourage the development of treatments for
patients with rare diseases.