Drugs for rare diseases // Drug Repurposing

Drugs for rare diseases

Pharma's Windfall

Thirty years ago, Congress acted to spur research on rare diseases. Today, we have hundreds of new drugs — along with runaway pricing and market manipulation, as drugmakers turn a law with good intentions into a profit engine.

By and

Her vision failed first.

Then she fell asleep at school from inexplicable fatigue. Even walking proved difficult, often impossible, as she knocked into furniture and walls. It was like an electrical switch in her body toggled without warning. Some days she was in control, most she was not.

Specialists were stumped. Some wondered if 10-year-old Violet O’Dell exaggerated her symptoms for attention. A teacher warned that it was adolescent rebellion.

It took three months before a general practitioner detected the shadowy, cancerous tumor that clung to Violet’s brain.

“How much would you pay to have extra time with your dying child? We don’t make a lot of money. We knew we’d lose our home, everything we own.”

In the fall of 2011, a cancer physician at Seattle Children’s hospital met with Violet. He crouched, to talk eye to eye. And he told her: The rare, inoperable malignancy struck just a few hundred children each year. Most died within a year. None survived.

But doctors could offer this: An “orphan drug” — one approved for a rare disease — might slow the cancer’s growth. With the drug, Avastin, Violet might live an extra month, maybe longer.

The drug cost up to $50, 000.

“How much would you pay to have extra time with your dying child?” Violet’s mother, Jessica O’Dell, asks. “We don’t make a lot of money. We knew we’d lose our home, everything we own.”

For the O’Dell family, the drug’s cost exceeded their annual income. For Genentech, the San Francisco-based company that manufactures Avastin, the drug brings in $3 billion a year.

The O’Dells’ conundrum — a lot of money for a little time — highlights the pharmaceutical industry’s unchecked profiteering from rare diseases.

Revolutionary drug transforms orphan industry

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How Obama Would Stifle Drug Innovation

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If you want cutting-edge health care, don't make it a cost-controlled commodity.
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Pfizer recently said it's exiting the development of drugs for common conditions like heart disease. This is part of a shift underway in the pharmaceutical industry to give up on routine medical problems in favor of discovering "specialty" drugs for rare diseases and unmet medical needs like cancer.
The shift is driven in part by the industry's critics in Washington, who have long maligned drug companies for targeting too many routine medical problems with drugs that were "merely" tweaks on existing medicines

This year, the Food and Drug Administration

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Rejected the only medicine capable of treating the rare and fatal lung disease known as idiopathic pulmonary fibrosis. Pirfenidone, which has been available in Japan since 2008 and was just approved in Europe, was spurned by the FDA because the drug only showed efficacy in a single big trial—not the two large studies the FDA now requires. The decision to ban the drug is one of a rash of recent decisions that shows the FDA is making it more and more difficult for promising drugs to reach severely ill patients
Rare diseases make finding large patient pools to conduct clinical trials VERY difficult, if not impossible

It's for the children

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In an unintended consequence of the new health care law, drug companies have begun notifying children’s hospitals around the country that they no longer qualify for large discounts on drugs used to treat rare medical conditions…
Congress, in revising the drug discount program as part of the new health care law, blocked these hospitals from continuing to receive price cuts on orphan drugs intended for treatment of diseases affecting fewer than 200,000 people in the United States… The discounts typically range from 30 percent to 50 percent, and children’s hospitals say the change is costing them hundreds of millions of dollars

The costliness depends on what type of drug

by --

Treatment is being developed and sometimes the drugs shelf-life (flu vaccines). The only truely costly ones are for very rare diseases. They have little market value. The more prevelant an illness the easier to recoup costs and to make a huge profit.
"since people are buying and, literally, eating this stuff up anyway. And, of ALL their drugs, the psyche drugs are the MAJOR class, and growing by leaps and bounds". Mental illness is considered a chronic condition unlike a bacterial infection where 7-10days of a medication will usually cure the infection.
Rarely, once prescribed, are psychiatric drugs discontinued

Shire Says Holders Deserve Higher as AbbVie Eyes Offer  — Businessweek
Product sales will increase at a double-digit rate as Shire expands in drugs for rare diseases and eye maladies, rising to $6.5 billion by 2016, and the board needs to keep that in mind as it considers the company's future, CEO Flemming Ornskov said.