Hiltzik: A lifesaving drug, but it will cost $2.8 million
For the estimated 1,500 Americans suffering from a blood disorder known as beta thalassemia, the Food and Drug Administration’s approval Aug. 17 of the drug Zynteglo was almost unalloyed good news.
As a scientific achievement, Zynteglo is remarkable. The treatment has been shown to effectively cure the disorder in as many as 90% of test cases.
That’s a life-changing development for those with the inherited condition, many of whom are otherwise condemned to receiving blood transfusions as often as every two weeks. The median age of death of those with beta thalassemia, which is typically diagnosed within a few weeks of birth, is 37.
Zynteglo is remarkable for another reason, not so happily. At the price of $2.8 million set by its developer, Cambridge, Massachusetts-based Bluebird Bio, it’s the most expensive single-treatment drug ever approved in the U.S.
That makes it “one hell of a policy challenge," in the words of health insurance expert David Anderson of Duke University.
The reason, Anderson explains, is that even though Zynteglo might be cost-effective over the long term and from a social perspective even at $2.8 million, the one-year charge for it can bring ruin to an insurer’s profit-and-loss statement.
Zynteglo is one of a wave of genetics-based treatments destined to wash up on American health care shores in coming years. They’re aimed at varieties of cancer and other diseases for which no treatments have been previously found, or that require burdensome and expensive lifelong therapies.
Beta thalassemia falls into the latter category. The previous standard cure involved finding a matched donor of healthy red blood cells — typically a sibling. The donor’s healthy cells could be transplanted into patients to supplant the inherited cells, which can’t efficiently carry oxygen, leading to severe anemia.
Fewer than 1 in 4 patients have access to a matched donor, according to a report on Zynteglo issued in July by the Institute for Clinical and Economic Review, an independent nonprofit that evaluates data to help health care stakeholders decide on prices and access to treatments.
Others require transfusions every two to five weeks as well as regular chelation treatment, which removes iron from the blood.
Those with severe beta thalassemia are forced to become “tethered to the health care system," and even to specific geographical areas with medical centers that can provide the needed care, according to the report. “Patients plan their lives around transfusions, disrupting their ability to travel, take vacations, work, and study." Even so, their average life expectancy is far below that of healthy people.
Multiple issues swirl around the launch of Zynteglo. One is sticker shock.
Bluebird has rationalized the high price of Zynteglo by referring to the lifetime cost of transfusions and chelation therapy, which the company says can run as high as $6.4 million. “The average total health care cost per patient per year is 23 times higher than the general population," Bluebird spokeswoman Jess Rowlands told me by email.
“Prices just keep getting higher and higher," says Rita Redberg, a UC San Francisco cardiologist and an expert in assessing new medical technologies for effectiveness. “It used to be that $5,000 sounded high, and now we’re at $2.8 million. It’s wild."
Many high-priced drugs reach the market without sufficient evidence that they deliver the benefits their manufacturers claim, Redberg says. “In our system there’s generally very little relationship between costs and benefits to patients."
An important complication is what the late economic commentator Barbara Ehrenreich called America’s “increasingly impersonal, fragmented, irrationally arranged set of health services” — in 1970.
Since then, the challenges have only multiplied, despite occasional outbreaks of reform such as the Affordable Care Act and the recently enacted cap on insulin prices (the latter only for enrollees of government programs such as Medicare).
The Affordable Care Act has brought health coverage to previously uninsured or underinsured populations, but the gains in health care access are threatened with becoming undone by the explosion in prices for innovative therapies.
In our system, insurers saddled with the one-time cost of a treatment aimed at creating economic benefits in the future can’t be assured of reaping those benefits themselves, because of the possibility that the patient will be covered by a different insurer in the future.
Perversely, that’s less of an issue for drugs aimed at large populations of disease patients. Consider Gilead Sciences’ Harvoni and Sovaldi, hepatitis C cures, which once reigned as the emblematic high-priced drugs because they were priced at close to $100,000 per treatment.
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