Pharmaceutical Executive January 19, 2022
Significant challenges still lie ahead, but several specialists and a plethora of new technologies are well positioned to gather and analyze the evidence needed for diagnosing and treating rare diseases, writes Catherine Tak Piech.
The global rare disease community is steadily increasing awareness of the plight of those <1 in 2000 people who suffer with rare conditions, which often makes them the orphans of the health care system. Their most recent efforts have been focusing on the repercussions of a delayed or missed diagnosis, and the heavy social and financial burdens rare disease patients and their families share.
On Rare Disease Day, February 28th, campaigners nationwide mobilize events to literally shine a light on the challenges faced by this community....