$300,000 for a drug? Doctors ask if high prices for rare-disease drugs is right

The challenge of orphan drugs

Charging high prices for drugs to treat rare diseases—particularly when the lives of thousands are at stake—is an immoral practice that should be stopped, three physicians argue in JAMA.

Case study: Vertex Pharmaceuticals' ivacaftor

In a commentary published in JAMA on Tuesday, pediatrician Brian O'Sullivan and two colleagues at the University of Massachusetts Medical School question whether this trend of developing drugs for small, niche populations and marketing these 'orphan drugs' for whopping sums is sustainable.

The authors referenced Vertex Pharmaceuticals' cystic fibrosis (CF) drug, ivacaftor, which was approved in January 2012 to treat a small fraction of patients with the rare, and often fatal, inherited lung disease. Many doctors balked at the drug's price tag—about $294,000 annually—calling it "unconscionable" to charge so much for a treatment that had been financed in part through $75 million in charitable donations from patients, families, and doctors.

In an interview, O'Sullivan told the Boston Globe's Carolyn Johnson that subsequent attempts to lower the drug's cost appeared to have failed; the price has since increased by about $13,000. Though he acknowledged that pharmaceutical companies are not charitable organizations and drug research requires a significant investment, O'Sullivan questioned whether the way drug pricing is done is responsible.

Meanwhile, a Vertex spokesperson in a statement said that ivacaftor's cost "reflects how well this medicine works, the time and cost it took to develop and our commitment to reinvest to help many more people with [cystic fibrosis]—work that is highly expensive, risky, and takes the dedication of hundreds of people over decades." Moreover, he argued that the drug is covered by insurance, and most patients will only pay between $15 and $50 for the treatment. 

Pushback against high-priced treatments on the rise

The Globe's Johnson notes separate evidence suggesting a rising offensive against high-priced treatments for rare diseases. For example, Memorial Sloan-Kettering Cancer Center last year said the colon cancer drug Zaltrap, which costs $11,000 for a month's treatment, was too costly and would not be offered at the system. Accordingly, the drug's manufacturers halved the price.

Earlier this year, a group of 100 oncologists submitted an opinion piece to the journal Blood protesting the high cost of treatment for chronic myeloid leukemia, Johnson adds. The oncologists argued for "capitalism with a conscience"—pricing that is not simply based on what individuals can pay when life hangs in the balance.

"Examples include the price of bread during famines, polio vaccine, ivermectin for river blindness (provided for free by Merck and estimated to have saved the vision of 30 million individuals), and treatments of chronic medical conditions," the authors wrote in JAMA, adding that "[w]hen commodities are not essential to life or suffering, what the market will bear is appropriate…because it is not restrained by ethical considerations. Examples include the price of a Picasso painting, a luxury cruise, [or] a two-week vacation in New York…" (Johnson, Globe, 10/1).

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