Kevin Sites in the Hot Zone - Chapter 15: Coming HomeIn this final chapter of "A World of Conflict," Kevin Sites returns home to the U.S., only to confirm what he suspected -- that in the year that he was gone little had changed.
Kevin Sites in the Hot Zone - Chapter 14: Israel-Hezbollah WarThe war between Israel and Hezbollah shook the landscape in the Middle East.
Kevin Sites in the Hot Zone - Chapter 13: Sri LankaKevin Sites covered Sri Lanka as violence erupted between the government and Tamil Tiger rebels, pushing a nation with so much to lose back to the brink of all-out war. In rebel-held territory Sites interviewed Tiger fighters about their tactics and reported on the many effects of war still seen in the region.
Kevin Sites in the Hot Zone - Chapter 12: Nepal and KashmirKevin Sites covered Nepal during a time of sweeping political change that followed mass nationwide protests, forcing the autocratic King to cede power.
Kevin Sites in the Hot Zone - Chapter 11: Child BrideIn Afghanistan, Kevin Sites met a 12-year-old girl named Gulsoma, whose incredible story of resilience resonated with millions of people worldwide. She was only six years old when she was sold to a neighbor family in Kandahar as a child bride.
Kevin Sites in the Hot Zone - Chapter 10: AfghanistanReporting from Afghanistan in spring 2006, more than four years after the U.S.-led coalition ousted the Taliban, Kevin Sites found that war is not over in the country.
Kevin Sites in the Hot Zone - Chapter Nine: ChechnyaIn Chechnya during the winter of 2005-2006, Kevin Sites reported on a region still reeling from lingering conflict between Russia and Islamic separatists. The conflict engulfed Chechnya in the 1990s, and even now, half of the population is yet to return. Those that have eke out a living amid the rubble.
Kevin Sites in the Hot Zone - Chapter Eight: Iran
Kevin Sites in the Hot Zone - Chapter Seven: IsraelIn Israel, Kevin Sites interviewed Kinneret Boosany, a victim of a suicide bombing at a Tel Aviv cafe in 2002.
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Trevor Foltz was six months old last fall, fresh off a visit to Disney World in Orlando, when the spasms first began.
Healthy until that point in his life, he began thrusting backward in his car seat, repeatedly and forcefully, as he rode with his parents north toward home in Rhode Island. "I thought it was temper tantrums," says his mother, Danielle. The next day, at home, Trevor was hit with a series of 40 convulsions and rushed to the hospital, where he was diagnosed with infantile spasms, a rare form of epilepsy. Treatment would cost $1,600 per vial of steroid drug H.P. Acthar Gel, and Trevor would need three of them.
As if the idea of a $4,800 tab wasn't bad enough, when the Foltzes submitted their claim, they found out the company that made the drug, Questcor Pharmaceuticals, had just recently jacked up the price—to $23,000 per vial, or $69,000 for a three-vial treatment—and the insurance company wasn't going to pay. And all the while, unbeknownst to anyone at that time, an alternative, for $15, existed.
On Thursday, the Joint Economic Committee will open hearings in Congress on dramatic price hikes for drugs used to treat children, with a focus on companies such as Questcor and Ovation Pharmaceuticals, which in 2006 bought rights to a drug that treats heart problems in premature infants, and increased the price 1,800 percent to $1,875 per three-vial treatment.
"We need answers to why a company would increase the price of a drug 18-fold when costs related to marketing, physician education, and research appear stable," says hearing chair Amy Klobuchar, a Democratic senator from Minnesota.
Politicians say they are not opposed to drug companies earning strong returns on the costs of researching innovative drugs, and understand the high prices of many medications. But they are investigating whether some companies are price-gouging, concerned more about executive stock options than about running innovative companies.
Some of those drugs, like Questcor's, are decades-old drugs that were bought on the cheap and redesignated under the federal government's Orphan Drug Act, which marks its 25th anniversary this year. Not infrequently, the drugs' new owners pass on big price hikes to consumers.
At Questcor, the increase is explained as the cost of doing business with an orphan drug.
"The company was heading toward bankruptcy," says Steve Cartt, executive vice president for business development at Questcor, which is based in Union City, California, an industrial enclave on San Francisco Bay.
"The whole rationale for the price increase was to ensure availability of the product," says Cartt. "We talked to physicians. They wanted the drug to be available. The choice was risk of availability or a price increase."
Originally approved for multiple sclerosis in 1952, Acthar Gel had been owned by pharma giant Aventis, which was losing money on it, when the 11-year-old Questcor acquired it in 2001. Questcor, too, failed to gain traction with M.S. patients, so it sought a new track.
Now the gears at Questcor began to turn more quickly. It won orphan designation for Acthar Gel in 2003, and proceeded to the next step: getting F.D.A. approval to market the drug explicitly for infantile spasms, which under the orphan act would also include a seven-year monopoly for Questcor. The company prepared for a marketing blitz and doubled its sales force early last year. But when the F.D.A. rejected Questcor's application in May 2007, the company quickly slashed its staff and jacked up the price.
Cartt says the price was set "within the range of other orphan drugs," noting that many others go from $50,000 to $500,000 a year or higher. For instance, BioMarin, an orphan-drug specialty company, charges $70,000 a year for Kuvan, a drug to treat phenylketonuria, a genetic enzyme disorder that can cause mental retardation and brain seizures. But unlike BioMarin, which spends 64 percent of sales on research and development, Questcor spends very little; in 2007, Questcor's research and development accounted for 9.5 percent of sales revenue.
What other considerations played into the price Cartt would not say. Sales for 2007, when the price hike took effect, were $49.7 million, and net income was $37.5 million—a net profit margin of 75 percent. It was significant not only for its size, but also because it was the first profit since the company was formed, as Cypros Pharmaceuticals, in 1990.
Investors were pleased, driving up Questcor's share price from 40 cents to over $6 after the August 2007 price hike. But executives at the company started selling their shares in December, seven months after the former C.E.O., James Fares, stepped down and around the time Questcor executive Don Bailey took his place. Since December, Cartt himself has sold shares based on grants and options totaling $1.68 million; many of those options were granted at 46 cents a share. He holds nearly a million more options on Questcor stock.
Doctors were unhappy with the price hikes.
"Most of us in the child-neurology community were outraged at the extent of the price hike, unusual even for orphan drugs," says Eric Kossoff, a pediatric neurologist and infantile spasms expert at Johns Hopkins Children's Center. "Most of us had no choice, unfortunately. At the time it was felt to be the best drug out there, and they're the only company that makes it. This is an incredibly serious form of epilepsy with devastating implications if not treated."
Curiously, though, he found that the price hike "was one of the best things that could have happened." Why? "Because we found something better and cheaper." Far cheaper, it turns out. "We spent a few days going through all the medical literature, looking for what works, what doesn't."
The team turned up a study from the United Kingdom that gave infants high doses of prednisolone, a well-known, generic steroid. Prednisolone had been dismissed as relatively ineffective for infantile spasms-based research that used low doses. The high doses made all the difference: The British study found efficacy rates reached 70 percent and more. Johns Hopkins began using high-dose prednisolone and found it worked in about 70 percent of cases, on par with the hospital's experience with Acthar Gel. And the price was $15 per injection—essentially free—compared with the three-injection $69,000 treatment from Questcor. "It was like in times of war. You get focused, and amazing things come out," Kossoff says. "We don't use [Acthar Gel] at Hopkins anymore for infantile spasms because the oral steroids [high-dose prednisolone] work just as well."
It's unclear, though, how many other doctors and hospitals in the U.S. will switch from the $69,000 drug to the $15 drug.
"I don't understand what's behind the price increase," says Finbar O'Callaghan, a pediatric neurologist at the Bristol Royal Hospital for Children and coauthor of the United Kingdom Infantile Spasms Study, or UKISS. The study showed that high-dose prednisolone and a synthetic form of ACTH, the active hormone in Acthar Gel, were equally effective. He cautioned that the purpose of the study was not to compare the two, but to compare steroid treatment with another drug called vigabatrin. "Having said that, you couldn't get a piece of paper between the results of the prednisolone and the results of the ACTH."
Costs aside, Hopkins is achieving the same results against Acthar Gel. "There is no reason to favor one over the other, unless there is a financial reason for doing so. That's been a big issue in the U.S.," says O'Callaghan. Comparing $15 against $69,000 "puts a different perspective on it," he says.
"Historically, and unfortunately," he adds, "doctors in general are very traditional and tend to use what's worked before."
Asked about the $15 price on prednisolone, Cartt said studies from the 1990s show low efficacy rates for the drug. When informed that those studies looked at low doses, not high doses, Cartt said no one knows the long-term implications of high-dose prednisolone, and said the company's higher profits will help it find out. "We can afford to study the long-term effects" of Acthar Gel and the alternatives, he said.
What the congressional hearing may find is that Questcor had a business problem: While its drug had a potential market of 300,000 multiple sclerosis patients, not enough of them were buying. But among a smaller market, just 2,000 babies per year, Acthar Gel was extremely effective in fighting infantile spasms. Questcor's astronomical rates may simply be a matter of hard business realities in a small potential market.
For the Foltzes, Questcor's high prices proved irrelevant, after much struggle. When at first his insurance company, WorldWide Insurance, rejected the claim, Trevor's doctor faxed in a letter stating that there was a good chance Trevor would end up mentally retarded for life without treatment; the insurer relented. But on Thursday, his mother, Danielle, will join those who testify against companies like Questcor. She says, "I feel they're going to soak every penny if they can get it."
