Rare diseases are uncommon by nature: no physician can be expected to know implicitly what is wrong with every patient that walks through his or her door, or who appears in a telehealth chat window. The biopharmaceutical industry, due to structural issues both corporate and legal, tend to focus on geographic markets; regulatory requirements reinforce this approach, since most countries require clinical trials conducted on local populations, as a condition of approval.
Rare diseases are any disease, disorder, illness or condition affecting fewer than 200,000 people in the US, as defined by the Orphan Drug Act of 1983. In the European Union, a disease is considered rare if it affects less than five individuals per 10,000. Most rare diseases have a genetic basis, affecting as many as 400 million people worldwide -- including 30 million in Europe and 25 million in the US – according to a World Health Organization estimate. Only 74 rare congenital or genetic diseases have an approved therapy, accounting for less than 3% of all such diseases, according to Datamonitor Healthcare.
Diseases, however, do not respect borders, a fact which COVID-19 has painfully demonstrated over the last 18 months. Similarly, rare...
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