Orphan Drugs

President Bush today vetoed legislation designed to boost wider development of so-called "orphan drugs" that treat rare diseases and dilute monopoly opportunities for drug firms. Bush said in a statement that he had "serious concerns" that the measure actually would remove incentives for drug companies to develop new treatments, having the opposite effect of its purpose.

It didn't used to be in Sandy Binder's job description to be a drug sleuth. As director of oncology community practices at UCLA, she didn't have to spend two hours a day checking on the supply of crucial cancer drugs at her six clinics and dickering with suppliers to make sure her doctors could get their hands on what their patients needed, even if at a markup that tripled the normal cost. Those were the old days, before the crisis of drug shortages in the United States spiraled out of control.

Nexell Therapeutics Inc. shares more than tripled Wednesday after the Irvine drug maker said it will have seven years of exclusive U.S. marketing rights for an experimental drug to treat a rare genetic blood ailment. The stock, which had lost 75% of its value this year through Tuesday, climbed $1.65 to $2.40 on Nasdaq. Nearly 8 million shares changed hands, compared with average daily volume of about 84,500 shares over the last three months.

Nexell Therapeutics Inc. shares more than tripled Wednesday after the Irvine drug maker said it will have seven years of exclusive U.S. marketing rights for an experimental drug to treat a rare genetic blood ailment. The stock, which had lost 75% of its value this year through Tuesday, climbed $1.65 to $2.40 on Nasdaq. Nearly 8 million shares changed hands, compared with average daily volume of about 84,500 shares over the last three months.

The Orphan Drug Act was created to motivate drug companies to develop treatments for an estimated 5,000 rare diseases, some of which afflict only a few hundred people. To compensate for the extremely small markets, the law provides a 7-year monopoly to the first firm that discovers and manufactures a so-called orphan drug.

President Bush Friday vetoed legislation intended to prevent drug manufacturers from realizing unintended windfall profits from drugs developed under a law designed to stimulate research into therapies for rare disorders. The legislation, approved unanimously by both houses of Congress, would have amended the 1983 Orphan Drug Act, which gives companies exclusive marketing rights and tax incentives to develop drugs for diseases that afflict fewer than 200,000 people.

There's an effort in Congress to curtail the market monopolies awarded to some drugs under the Orphan Drug Act, a federal law whose beneficiaries so far include Amgen Inc. and its blockbuster drug Epogen. But even if the proposed changes in the law are passed and survive President Bush's threatened veto, don't look for Thousand Oaks-based Amgen to get hurt. That's because Amgen's patents on Epogen give the drug a second coat of armor against competition.

The federal government has awarded Genentech a seven-year monopoly in the sale of a genetically engineered growth hormone, allowing the South San Francisco firm to rack up what is believed to be another monumental first for itself and the fledgling biotechnology industry. The Food and Drug Administration on Monday gave Genentech's drug, called Protropin, status as an "orphan drug" for the treatment of growth hormone deficiency and Turner's Syndrome, a chromosomal disorder in female children.

It was a law with a curious name and grand intentions: to provide financial incentives that would motivate drug companies to develop treatments for an estimated 5,000 rare diseases. Without such incentives, the reasoning went, drug companies would be unlikely to do the research and production on their own. Some of the diseases afflicted only a few hundred people nationwide, meaning it would have cost the companies more to produce the drugs than they could hope to earn from sales.

When Julie, the daughter of a State Department development official, packed for an overseas post with her parents in 1989, part of her luggage was a small ice cooler carrying $20,000 worth of hGH, a drug designed to aid the growth of children who lack certain hormones. Julie, a 14-year-old with the stature of an 8-year-old, is one of perhaps 15,000 children who suffer from a rare disorder, an abnormally low growth rate, and who use the recently developed biosynthetic hGH to treat their disease.

Orphan drugs are medications that have not yet been approved by the federal Food and Drug Administration and are of benefit to relatively few people. This designation qualifies the manufacturer to receive certain benefits from the government in exchange for developing the drug. The drug company can pull this drug off the market at any time even if it has been used by patients effectively for 20 years and will wreak havoc with lives.

The U.S. Food and Drug Administration on Tuesday approved the nation's first drug to treat kidney cancer that has spread beyond that organ, a disease that typically claims its victims within a year of diagnosis. The approval of Proleukin--a genetically engineered "orphan drug" developed by biotechnology pioneer Cetus Corp., now part of Chiron Corp.--should buoy the hopes of the nearly 10,000 patients diagnosed each year with kidney cancer.

There's an effort in Congress to curtail the market monopolies awarded to some drugs under the Orphan Drug Act, a federal law whose beneficiaries so far include Amgen Inc. and its blockbuster drug Epogen. But even if the proposed changes in the law are passed and survive President Bush's threatened veto, don't look for Thousand Oaks-based Amgen to get hurt. That's because Amgen's patents on Epogen give the drug a second coat of armor against competition.

It was a law with a curious name and grand intentions: to provide financial incentives that would motivate drug companies to develop treatments for an estimated 5,000 rare diseases. Without such incentives, the reasoning went, drug companies would be unlikely to do the research and production on their own. Some of the diseases afflicted only a few hundred people nationwide, meaning it would have cost the companies more to produce the drugs than they could hope to earn from sales.

The White House made a big mistake Friday on a bill intended to trim huge profits from some drugs that are produced under federal protection to treat rare diseases. It should erase the error. The mistake was an announcement that President Bush would let the bill die. That baffled Washington, where decisions often hinge on relative levels of pain inflicted by lobbyists. This bill was painless. The Senate and the House approved the bill unanimously.

President Bush Friday vetoed legislation intended to prevent drug manufacturers from realizing unintended windfall profits from drugs developed under a law designed to stimulate research into therapies for rare disorders. The legislation, approved unanimously by both houses of Congress, would have amended the 1983 Orphan Drug Act, which gives companies exclusive marketing rights and tax incentives to develop drugs for diseases that afflict fewer than 200,000 people.

Orphan drugs are medications that have not yet been approved by the federal Food and Drug Administration and are of benefit to relatively few people. This designation qualifies the manufacturer to receive certain benefits from the government in exchange for developing the drug. The drug company can pull this drug off the market at any time even if it has been used by patients effectively for 20 years and will wreak havoc with lives.

It didn't used to be in Sandy Binder's job description to be a drug sleuth. As director of oncology community practices at UCLA, she didn't have to spend two hours a day checking on the supply of crucial cancer drugs at her six clinics and dickering with suppliers to make sure her doctors could get their hands on what their patients needed, even if at a markup that tripled the normal cost. Those were the old days, before the crisis of drug shortages in the United States spiraled out of control.

President Bush today vetoed legislation designed to boost wider development of so-called "orphan drugs" that treat rare diseases and dilute monopoly opportunities for drug firms. Bush said in a statement that he had "serious concerns" that the measure actually would remove incentives for drug companies to develop new treatments, having the opposite effect of its purpose.

Larry Wright wanted a drum set, but his mother had money only for drugs. So one day he took the subway down to Times Square, flipped over a plastic bucket and began pounding on it with battered drumsticks. He hoped for coins, maybe $10 or $20 worth. But as strangers watched this sinewy youth play--sweat pouring from his shirtless torso, tiny bits of wood and plastic flying--they tipped him dollars instead of quarters.