orphan drugs

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.

GOP Senators Ask GAO To Investigate High Prices For Orphan Drugs

9:39am March 07, 2017

Amid an uproar over soaring drug prices, three GOP senators are seeking an investigation into whether the Orphan Drug Act is being manipulated to jack up the cost of medications for rare diseases.

The list price for a year's treatment with Emflaza is $89,000. The drug is available outside the U.S. for about $1,000.

Duchenne Drug Delayed After Outrage Over Price

3:25pm February 14, 2017

by Sarah Jane Tribble

Many Duchenne muscular dystrophy patients in the U.S. have imported a medicine called deflazacort for about $1,200 a year. A brand-name version just approved for sale in America costs $89,000.

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.

Sen. Grassley Launches Inquiry Into Orphan Drug Law's Effect On Prices

1:20pm February 10, 2017

by Sarah Jane Tribble

Citing a Kaiser Health News investigation, Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, will examine potential abuses of incentives to spur development of drugs for rare diseases.

Seven of the top 10 drugs by U.S. sales in 2015 were orphan drugs.

Drugs For Rare Diseases Have Become Uncommonly Rich Monopolies

5:23pm January 17, 2017

Drugmakers have brought almost 450 orphan drugs to market and collected rich incentives by doing so. But nearly a third of the medicines aren't new or were repurposed many times for financial gain.

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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.

High Prices For Orphan Drugs Strain Families And Insurers

3:30pm January 17, 2017

Three decades ago, Congress set up a system to encourage drug companies to develop treatments for rare diseases. The law has worked, but at a high cost.

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.

Are Golden Tickets That Speed Drugs Through FDA Worthwhile?

2:16pm September 29, 2016

by Sarah Jane Tribble

A voucher awarded to a company that finds a treatment for a rare childhood disease can be sold to the highest bidder and then used to fast-track the review of another drug. Are the prizes worth it?

How big a deal is the spending on drugs for rare diseases?

How Much Do Drugs For Rare Diseases Add To Health Care Spending?

4:16pm September 07, 2016

by Sarah Jane Tribble

Researchers analyzing recent drug spending in the U.S. say that high-priced drugs for rare diseases aren't having a widespread or significant effect on overall health care spending.

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.

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

7:19pm February 04, 2016

by Alison Kodjak

After controversial pharmaceutical executive Martin Shkreli left the hearing on Capitol Hill, the representatives' attention turned to why prices for some drugs are rising so steeply.

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A critic says filing for FDA approval to exclusively sell a drug that's been available for years is "akin to trying to patent a ladder." It also may push the price of the drug beyond the reach of many patients.

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

10:26am December 23, 2015

by Alison Kodjak

Jacobus Pharmaceutical freely gives its experimental drug to patients with a rare disease. Now a rival wants FDA approval to sell its own version — and expects to charge at least $37,500 per year.

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