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For Sickle Cell, a Complex Disease, New Drugs Could Bring Complex Costs

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to take voxelotor in perpetuity. (Global Blood is also reporting this weekend mid-stage data for voxelotor in pediatric patients aged 6 to 17.)

Access to new medicines is a major concern, says patient advocate Bailey, who has been a paid consultant to drug developers, including Bluebird. She sees too many people turning to crowdfunding to cover medical bills. “GoFundMe shouldn’t be the option for getting life-saving medicines,” Bailey says. “As long as our health system is designed as it is, about profit and not human lives, I don’t know how we’ll change it.”

A Global Blood spokeswoman says it will announce the cost of voxelotor when it launches, which is standard industry practice. The company is committed to a “value based approach” based in part on the drug’s clinical results, she says, but offers no more specifics. Some drug makers have translated that vague phrase into rebates for their patients who suffer setbacks: cancer that doesn’t respond to treatment, say, or a stroke or heart attack that occurs despite a cholesterol-lowering therapy.

The outlook for genetic fixes or cures for sickle cell is complicated, too. The FDA is bullish, but it remains to be seen if companies set prices at hundreds of thousands of dollars, perhaps even $1 million for a treatment, with government insurers as the main payers in the U.S. Companies will say prices reflect the elimination of years of chronic treatment, hospital costs, and emergency room visits. (California alone reported that in-patient and emergency room visits by sickle cell patients in 2014 totaled at least $738 million, not including those covered by HMOs.)

But there are no long-term data; no one knows if effects will wear off, or when. And even though it stems from a known mutation, sickle cell is a multifaceted disease with diverse environmental factors seemingly at play. The “value” for one patient might be different than for another. Some people want their pain crises to go away; others can deal with the pain but can’t take the staggering exhaustion. “It’s the fatigue that knocks me on my behind,” says Bailey, a former researcher who advocates for more patient input into drug development and policy.

“The biology is complicated,” says Vichinsky of Children’s Hospital Oakland, “so what kind of clinical impact do you need for a drug to be used by everybody? If you can decrease the pain events and other complications by 50 percent, the drug will be used.”

Photo courtesy of Cassandra Trimnell.

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