orphan drugs orphan drugs

Kerri De Nies plays with her son, Gregory Mac Phee at their home in San Diego. Gregory tested positive for adrenoleukodystrophy, a rare brain disorder that affects 1 in about 18,000 babies. Roughly 30 percent of boys with the genetic mutation go on to develop the most serious form of the disease. Anna Gorman/KHN hide caption

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Anna Gorman/KHN

Parents Lobby States To Expand Newborn Screening Test For Rare Brain Disorder

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Lulabelle Berry surveys the 600 acres she and her husband, Jimmy, own in the Ozarks near Mountain View, Ark. Berry walks laps on her porch to aid her recovery from a stroke. She says she's grateful for the drug that saved her life. "It's been a good life," she says. Sarah Jane Tribble for Kaiser Health News hide caption

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Sarah Jane Tribble for Kaiser Health News

Rural Hospitals Struggle To Stock Expensive Drugs That Could Save Lives

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FDA Commissioner Scott Gottlieb says the agency will close a loophole that lets drug manufacturers skip pediatric testing in some drugs intended for children. Zach Gibson/Getty Images hide caption

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Sen. Tom Cotton, R-Ark., is one of three GOP senators seeking an investigation into six-figure annual costs for drugs intended to treat rare diseases. Alex Brandon/AP hide caption

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Alex Brandon/AP

Senate Judiciary Committee Chairman Chuck Grassley, R-Iowa, is looking into how the Orphan Drug Act may be affecting a wide range of drug prices. Andrew Harnik/AP hide caption

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Andrew Harnik/AP

Luke Whitbeck, 2, sits on his mother's lap while the nurse prepares his treatment at a pediatric infusion center in Hawthorne, N.Y., in October 2016. Heidi de Marco/Kaiser Health News hide caption

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Heidi de Marco/Kaiser Health News

High Prices For Orphan Drugs Strain Families And Insurers

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NPR, Kaiser Health News/Evaluate Pharma analysis for Kaiser Health News on Sept. 21, 2016

Drugs For Rare Diseases Have Become Uncommonly Rich Monopolies

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Sarepta Therapeutics was awarded a voucher for a fast-track drug review by the Food and Drug Administration when the company's medicine for Duchenne muscular dystropy was approved Sept. 19. Now Sarepta is looking to sell the voucher to the highest bidder. Mick Wiggins/Ikon Images/Getty Images hide caption

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Mick Wiggins/Ikon Images/Getty Images

Nancy Retzlaff, chief commercial officer for Turing Pharmaceuticals, was asked how much the drug Daraprim costs at the House Oversight and Government Reform Committee on Capitol Hill on Thursday. Brendan Smialowski/AFP/Getty Images hide caption

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Brendan Smialowski/AFP/Getty Images

House Hearing Probes The Mystery Of High Drug Prices That 'Nobody Pays'

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FDA Approval Could Turn A Free Drug For A Rare Disease Pricey

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Flemming Ornskov, CEO of Shire, says the company's offer for ViroPharma is part of a broader push into orphan drugs. Bloomberg via Getty Images hide caption

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Bloomberg via Getty Images