Still, orphan drugs, which cost up to $600,000 a year, may end up driving a broader discussion of how much is too much. This is an issue “that society is going to have to deal with,” says Mike Scott, who serves on the board of directors of the International Myeloma Foundation. “And that has commercial implications, research implications and societal implications.”
As Coté sees it, the focus should be on getting orphan drugs made, whatever the cost, to repay a long overdue debt. Rare diseases have led to a host of medical knowledge, including the urea cycle, metabolism and blood clotting. “We owe people with rare diseases some therapies,” he argues, “because most of our understanding in medical science is based on rare diseases.”
This article was originally published with the title Adoption Agents.