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Posted by on Jan 3, 2008 in Health, Society | 6 comments

Overlooked but Not Forgotten

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In the hooplah surrounding today’s caucuses in Iowa and the anticipation of New Hampshire’s primaries next Tuesday, here’s a calendar entry that will probably be overlooked by many: tomorrow is the 25th anniversary of the signing of the Orphan Drug Act (ODA).

Who cares? Well, our family does, along with millions of others. We care because we all know someone who lives with one of 7,000 rare diseases — ailments that individually affect fewer than 200,000 Americans but in aggregate touch the lives of an estimated 25 million.

Given the relatively small population affected by each of these disorders, research into the causes and the development of treatments and/or cures has been limited, although such R&D is certainly more expansive today than it was on Jan. 4, 1983 when the ODA became law.

According to the press release distributed by the National Organization for Rare Diseases (NORD):

In the decade before the Orphan Drug Act was passed by Congress and signed on January 4, 1983, by President Ronald Reagan, only 10 treatments had been developed for rare diseases by the pharmaceutical industry. In the 25 years since then, more than 1,100 treatments for rare diseases have entered the research pipeline and more than 300 have been approved by the U.S. Food and Drug Administration for marketing. In addition, the Act has proven to be a potent catalyst to the growth of the pharmaceutical and biotechnology industries in the United States.

… In the past, these diseases of low prevalence were overlooked by drug and medical device developers. The Orphan Drug Act provides financial incentives that help companies recover the cost of developing a drug for small patient populations.

This case is one of those where my small-government, federalist principles take a holiday, while I sing the praises of the reach and effectiveness (yes, I said effectiveness) of the Washington establishment; where I recognize all too well that the states acting alone could not have devised or supported such breakthroughs.

Twenty five years ago, I was preparing for my final semester of high school and looking forward to college and career and whatever the future might bring — arrogantly, blindly believing I would be a self-made man and thus never require the beneficent hand of government.

Today, my son is preparing for his final semester of high school and looking forward to college and career and whatever the future might bring — knowing full well that it was the beneficent hand of government two-and-half decades earlier that set up a system whereby drugs could be created that would help him manage his Tourette Syndrome and thus contribute to a life filled with hope and promise.

And that, my friends, is progress.

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  • superdestroyer

    I believe one of the reasons that companies did not bother with low occurrence diseases is that the cost of getting the drug approved by the government and marketed were so high that the companies could never recover the costs.

    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.

  • Once again SD is wrong. The pharmaceutical companies are interested in profit. Orphan drugs are for conditions that just don’t offer the scale for enough profit for a modern public corporation without any costs “caused” by government. Would he and others who agree with his statement rather just have drugs released with no testing? And since when is marketing a regulatory cost?

  • superdestroyer

    Jim,

    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.

  • DLS

    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)

  • DLS

    I found it — an article I read some time ago.

    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)

  • DLS

    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.

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