The pharmaceutical companies are interested in profit.

Definitely in the case of EPO and related anemia drugs (the use of which isn’t restricted to athletes cheating to improve their chances at success), which have been associated with kickbacks “rebates” to doctors and dialysis clinics.

Over-prescription of these drugs was in the news recently and these drugs are in the news once more, today. (here)

Enjoy your drug reading.

With no cap on prices and patients with few options, companies found they could profit in small markets — charging as much as $600,000 a year per patient for drugs that people would need their entire lives. …

The cost of such drugs has grown so dramatically that employers and insurers are now pushing back. Some health plans are excluding coverage of certain orphan drugs. Others are requiring employees to pay as much as half of the cost of the pricey medicines. All this makes it tougher for patients to get the very drugs that the Orphan Drug Act helped make possible. …

“Maybe the medicine is not working,” Dr. Vermani says he told Mr. Ash.

“I can’t afford the prescribed dose,” Mr. Ash replied. “I’m taking half.”

How Drugs for Rare Diseases Became Lifeline for Companies (Federal Law Gives Monopoly or Seven Years, Fueling urge in Biotech Profits; A Teen’s $360,000 Treatment)

And there’s more:

Orphan Drugs, Intellectual Property, and Social Welfare–Posner

Comment on Orphan Drugs and Intellectual Property-BECKER

Orphan Drugs–Posner’s Response to Comments

As biotech drug prices rise, U.S. hunts for solution

Employers and the government, which bear most of the cost for these medicines for diseases like cancer, rheumatoid arthritis and multiple sclerosis, are struggling to pay. They’re increasingly making patients pay up to 50 percent of the costs of treatments that can run several thousand dollars to $600,000 per patient annually. These “blunt instrument” approaches are spreading and will ultimately take these life-saving medicines out of reach of the average American, says Scott Howell, an internist who serves as vice president of pharmacy affairs at Highmark Inc., a Blue Cross and Blue Shield company in Pittsburgh.

As Costs Rise, New Medicines Face Pushback (Insurers Limit Coverage To FDA-Approved Uses; $300,000 Drug Denied)

the states acting alone could not have devised or supported such breakthroughs

They could have — at least a few states, California first and foremost. All it would take is one state.

* * *

the cost of getting the drug approved by the government and marketed were so high that the companies could never recover the costs

What’s been sought are “blockbuster” drugs, which can be patented and allow recovery of some of the costs. (Having a patent in the case of special drugs is also a license to steal, but overall the industry is suffering, not thriving.)

The following two articles (from last month; they are up to date) illustrate what “Big Pharma” will be facing.

Big Pharma Faces Grim Prognosis (Industry Fails to Find New Drugs to Replace Wonders Like Lipitor)

As Drug Industry Struggles, Chemists Face Layoff Wave (Lipitor Pioneer Is Out At Doomed Pfizer Lab; A Blockbuster Drought)

The regulatory compliance and the marketing are two different costs. Both costs are very high and do not scale for low market.

Achieving statistical significance in a clinical trial requires the same number of patients no matter the disease. Multi-clinic trials are harder to perform because researchers are actually going to have to get a larger percentage of the people with a disease to volunteer for a clinical trial but there are fewer physicians interested in acting as an investigator.

The government was/is basically offer a subsidy to offset the regulatory compliance costs.

One of the odd things these days is how it is to do research to prove a drug is effective. Bring a children’s medicine to market is also cost prohibitive but in today’s climate the companies just do not the research and let the physicians take the risks by using a drug off-label.