今日美国载文：小药丸, 大帐单, 孤药使用者的困惑 Rare disorders put spotlight on crippling drug costs
For Christina and Randy Culver, the bad news kept coming: First, doctors told the Colorado Springs couple that the clusters of "full-body hiccups" afflicting their 10-month-old son were seizures.
Specifically, Tyler had infantile spasms, a rare, potentially devastating brain disorder that can cause mental retardation.
Within hours of diagnosis, Tyler was hospitalized in Denver and prescribed a drug that doctors said had the best chance of stopping the seizures. But, just as it started working and Tyler was ready to go home, the family got one more shocker: The price of the drug, called Acthar, or ACTH (adrenocorticotropic hormone), had just increased from $1,600 to $23,000 a vial — and the family's insurer would not pay for the two vials they needed.
The Culvers did eventually leave with the medicine, after five days of negotiation between the insurer, a patient assistance group and the drug's manufacturer, Questcor Pharmaceuticals. But, weeks later, Christina Culver says, "I still don't know who's paying."
Thankfully, infantile spasms are rare. I know about the disorder only because my 9-year-old son had it as a baby. But supersized medication bills — and debates over who should pay them and whether they are worth the cost — are becoming less rare.
FIND MORE STORIES IN: Tyler
That's partly because of a success story. For nearly 25 years, drugmakers have had financial incentives to develop drugs for rare disorders, those that afflict fewer than 200,000 patients, under the federal Orphan Drug Act.
"We've come a long way, baby," says Maria Hardin, vice president of patient services at the non-profit National Organization for Rare Disorders (NORD). "Prior to the Orphan Drug Act, there were fewer than 10 drugs approved for rare conditions. Now we have more than 300."
But drugs for rare conditions aren't the only pricey ones: Some costly new drugs are specialized medicines for more common disorders, including cancer. And the future is "personalized medicine" — targeted drugs for people with different genetic profiles. High prices for such drugs are justified, companies say, by high development and production costs and the need for profits, even if sales are small.
A handful of rare disorders now come with six-figure yearly drug bills — for life. In the case of infantile spasms and ACTH, initial treatment lasts only a few weeks (though children often need more ACTH or other drugs later). Total cost for one treatment round: an average of $100,000 a child, Questcor says.
"We tried to make it a sustainable product at a lower price, and it didn't work," says company vice president Steve Cartt. Questcor bought Acthar, which has been used for decades, from another company in 2001 and solved problems that caused past shortages. It has been seeking Food and Drug Administration approval to label the drug for use in infantile spasms (it's labeled for use in multiple sclerosis but is not a first-line MS drug). The main goal now is to keep it on the market and to work with NORD's payment assistance program to make sure babies with seizures can get it, Cartt says.
Rare disorders put spotlight on crippling drug costs
But doctors say the high price inevitably will reduce use and mean that some babies will get less effective or delayed treatment. "I don't think there's any question that some kids won't get appropriate care," says Steve Roach, chief of child neurology at Ohio State University in Columbus. He's worried that some hospitals will not pay for in-house doses not covered by insurers. Some, he says, will "just have to say no."
Of course, no one wants to say no to drug development or to keeping drugs for the rarest disorders on the market. But the price question is not going to go away. "I don't know what the solution is," says Diane Dorman, vice president of public policy at NORD. "But this needs to be a national conversation."
For Christina and Randy Culver, the bad news kept coming: First, doctors told the Colorado Springs couple that the clusters of "full-body hiccups" afflicting their 10-month-old son were seizures.
Specifically, Tyler had infantile spasms, a rare, potentially devastating brain disorder that can cause mental retardation.
Within hours of diagnosis, Tyler was hospitalized in Denver and prescribed a drug that doctors said had the best chance of stopping the seizures. But, just as it started working and Tyler was ready to go home, the family got one more shocker: The price of the drug, called Acthar, or ACTH (adrenocorticotropic hormone), had just increased from $1,600 to $23,000 a vial — and the family's insurer would not pay for the two vials they needed.
The Culvers did eventually leave with the medicine, after five days of negotiation between the insurer, a patient assistance group and the drug's manufacturer, Questcor Pharmaceuticals. But, weeks later, Christina Culver says, "I still don't know who's paying."
Thankfully, infantile spasms are rare. I know about the disorder only because my 9-year-old son had it as a baby. But supersized medication bills — and debates over who should pay them and whether they are worth the cost — are becoming less rare.
FIND MORE STORIES IN: Tyler
That's partly because of a success story. For nearly 25 years, drugmakers have had financial incentives to develop drugs for rare disorders, those that afflict fewer than 200,000 patients, under the federal Orphan Drug Act.
"We've come a long way, baby," says Maria Hardin, vice president of patient services at the non-profit National Organization for Rare Disorders (NORD). "Prior to the Orphan Drug Act, there were fewer than 10 drugs approved for rare conditions. Now we have more than 300."
But drugs for rare conditions aren't the only pricey ones: Some costly new drugs are specialized medicines for more common disorders, including cancer. And the future is "personalized medicine" — targeted drugs for people with different genetic profiles. High prices for such drugs are justified, companies say, by high development and production costs and the need for profits, even if sales are small.
A handful of rare disorders now come with six-figure yearly drug bills — for life. In the case of infantile spasms and ACTH, initial treatment lasts only a few weeks (though children often need more ACTH or other drugs later). Total cost for one treatment round: an average of $100,000 a child, Questcor says.
"We tried to make it a sustainable product at a lower price, and it didn't work," says company vice president Steve Cartt. Questcor bought Acthar, which has been used for decades, from another company in 2001 and solved problems that caused past shortages. It has been seeking Food and Drug Administration approval to label the drug for use in infantile spasms (it's labeled for use in multiple sclerosis but is not a first-line MS drug). The main goal now is to keep it on the market and to work with NORD's payment assistance program to make sure babies with seizures can get it, Cartt says.
Rare disorders put spotlight on crippling drug costs
But doctors say the high price inevitably will reduce use and mean that some babies will get less effective or delayed treatment. "I don't think there's any question that some kids won't get appropriate care," says Steve Roach, chief of child neurology at Ohio State University in Columbus. He's worried that some hospitals will not pay for in-house doses not covered by insurers. Some, he says, will "just have to say no."
Of course, no one wants to say no to drug development or to keeping drugs for the rarest disorders on the market. But the price question is not going to go away. "I don't know what the solution is," says Diane Dorman, vice president of public policy at NORD. "But this needs to be a national conversation."
