orphan drugs

Gilead Sciences CEO Daniel O'Day (center) took part in a meeting of the White House Coronavirus Task Force on March 2.

FDA Grants Experimental Coronavirus Drug Benefits For Rare Disease Treatments

7:26pm March 24, 2020

by Sydney Lupkin

Gilead Science's remdesivir, an antiviral medicine being tested for treatment of COVID-19, would get a seven-year monopoly if approved by the Food and Drug Administration.

The Food And Drug Administration reviews new drugs for approval much faster than it used to, but changes in the agency's standards have drawn questions.

Health & Safety

FDA Approves Drugs Faster Than Ever But Relies On Weaker Evidence, Researchers Find

11:45am January 14, 2020

by Sydney Lupkin

Changes in the way the Food and Drug Administration reviews new medicines means that there are more cures and treatments on the market. But there's also less proof the drugs are safe and effective.

The Food and Drug Administration has seen a sharp increase in applications for drug to treat rare diseases. An oversight report found problems with how agency is handling them.

Health & Safety

FDA Faulted For Lapses In Orphan Drug Program

4:07pm November 30, 2018

The Government Accountability Office found breakdowns in the way the Food and Drug Administration evaluates drugs for rare diseases. The analysis came after an investigation by Kaiser Health News.

Dr. Scott Gottlieb, Food and Drug Administration commissioner, told Kaiser Health News the incentives intended to spur development of drugs for rare diseases deserve a fresh look.

Health & Safety

FDA Commissioner: Are The Incentives Right For Orphan Drugs?

1:58pm December 22, 2017

by Sarah Jane Tribble

Food and Drug Commissioner Scott Gottlieb says he wants to know why drugmakers churn out medicines for some rare diseases but not others.

Peter Saltonstall, president of the National Organization of Rare Disorders, speaks at a rally Tuesday in support of tax credits for companies that develop drugs for rare diseases.

Health & Safety

Advocates For Patients With Rare Diseases Defend Tax Credits For Orphan Drugs

6:13pm November 29, 2017

by Sarah Jane Tribble

The Republican tax plan could reduce or eliminate federal tax credits that reward companies for developing drugs to treat rare diseases. People who benefit from the medicines have objected.

Speaker of the House Paul Ryan, center, and other lawmakers have a plan to overhaul the tax code that includes a provision that would repeal a tax credit for makers of drugs for rare diseases.

Health & Safety

House Republicans Aim To Eliminate Tax Credits For Orphan Drugs

2:55pm November 03, 2017

by Sarah Jane Tribble

For more than three decades, pharmaceutical companies have claimed a 50 percent tax credit for the cost of clinical trials of drugs for rare diseases. The credit is now in jeopardy.

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 dev

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

11:27pm October 04, 2017

by Anna Gorman

There's a genetic test for ALD, the inherited disorder portrayed in the movie Lorenzo's Oil, and the federal government recommends it for all newborns. But only a handful of states offer it routinely.

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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,

Rural Hospitals Struggle To Stock Expensive Drugs That Could Save Lives

4:48pm September 15, 2017

by Sarah Jane Tribble

A federal drug program blocks rural hospitals from getting discounts on rare-disease drugs, forcing staff to cut back on supplies of lifesaving medicines.

FDA Commissioner Scott Gottlieb says the agency will close a loophole that lets drug manufacturers skip pediatric testing in some drugs intended for children.

FDA Moves To Rein In Drugmakers' Abuse Of Orphan Drug Law

5:02pm September 13, 2017

by Sarah Jane Tribble

Following an investigation by Kaiser Health News and NPR, the Food and Drug Administration has moved to close a loophole that lets drugmakers skip pediatric testing of drugs to treat rare diseases.

The Government Accountability Office will investigate potential abuses of the Orphan Drug Act.

GAO Will Investigate Skyrocketing Prices For Orphan Drugs

4:20pm March 22, 2017

by Sarah Jane Tribble

The Orphan Drug Act was created to help patients with rare diseases get life-saving medications. But soaring prices suggest the law is being manipulated to increase profits.