For drug companies, Dr. Avorn said, “it’s a magical formula: Lifesaving drug, child at risk of bleeding to death – it kind of casts anybody who looks at costs into the role of some evil Scrooge-like person.”
“The insurers don’t want to end up on the front page of the newspaper saying Little Timmy bled to death because his drug wasn’t covered,” he said.
Also, because prices are high across the hemophilia market, no drug company wants to be the one to blink first. “They don’t want to get a price war started and end up at a super low price point,” said Edmund Pezalla, a consultant to pharmaceutical companies and former executive at Aetna.
So, these drugmakers compete not on price but clinical benefits – such as how long the drugs’ effects last – and through intensive marketing. The pool of potential customers is so valuable that companies often vie directly for individual patients.
Manufacturers, as well as specialty pharmacies that sell the drugs, hire patients and parents as recruiters and advisers, hold dinners and holiday parties, offer scholarships to patients, and even run summer camps for children with the disease. The Morris family regularly receives such invitations.
Jonathan Ducore, MD, a pediatric hematologist-oncologist at the University of California, Davis, Hemophilia Treatment Center in Sacramento, said some of his patients are persuaded by drug company presentations to switch medications. ”But the real differences between the drugs are limited,” he said.
Dr. Ducore said he tells patients if he thinks they are being misled by drugmakers about what a product will do. “But even though the tactics may seem a little smarmy, if it’s the patient’s choice, you have to go with it,” said Dr. Ducore, who has been Landon’s doctor since the boy was born.
The first clotting factor products, which came onto the market in the mid-1960s, were derived from human blood plasma, with thousands of donations combined to create one batch. This proved disastrous in the 1980s, when donors unwittingly spread HIV into the blood supply. An estimated 4,000 people with hemophilia – about 40 percent of the patient population in the United States – died from AIDS as a result.