https://www.bloomberg.com/news/features/2017-05-24/when-the-patient-is-a-gold-mine-the-trouble-with-rare-disease-drugs
Kerry Owens, a doctor in Oklahoma City who specializes in kidney disorders, was stunned by the call to her mobile phone in September 2015. She and a team of specialists were treating a new mother from nearby Enid, whose health had begun to deteriorate after giving birth. The doctors ran countless tests but couldn’t pinpoint what was wrong. Briefly they worried it might be an extremely rare and potentially fatal blood disorder called atypical hemolytic-uremic syndrome, or aHUS, which afflicts about 1 in 500,000 people each year. They put the patient on a drug called Soliris, which had recently been approved to treat the condition. But her health continued to decline, so they stopped the Soliris infusions.

Now a sales rep was on the other end of Owens’s phone from Alexion Pharmaceuticals Inc., the New Haven-based maker of Soliris—one of the world’s most expensive drugs, typically priced from $500,000 to $700,000 a year.

The rep was calling to argue with the treatment plan. She pressured Owens to continue Soliris treatments, ticking off detailed information about the mother’s organs that the doctor hadn’t shared with the drugmaker. “How did you know that?” Owens remembers thinking. She was monitoring the patient’s condition with seven hematologists and wasn’t swayed by the Alexion rep. “I was really taken aback by how bold and brash she was,” Owens says. “I’ve never had an experience like that—before or since.”