Treatment For Rare Diseases Can Be A Gold Mine For Big Pharma


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.

At this point 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.”

Alexion is a powerhouse in the market for orphan drugs, a fast-growing pocket of the pharmaceutical industry that focuses on rare diseases. In the U.S., an orphan drug is defined as one that treats a disease affecting fewer than 200,000 people in the country. Orphan drugs accounted for a disproportionate share, 41 percent, of all medications brought to market in 2014, according to a study by Johns Hopkins University. Globally, sales of orphan drugs are expected almost to double, to $209 billion, by 2022, based on numbers gathered by Evaluate Ltd., a life-sciences consulting company.

These drugs have helped millions of people. Patients with aHUS, for instance, faced years of kidney dialysis treatments and sometimes succumbed to deadly blood clots before Soliris came to market. “It’s the best drug I’ve seen for changing the outcome of a disease since I graduated in 1985,” says Gianluigi Ardissino, a doctor in Milan who’s treated more than 70 patients with Soliris.



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