Without the Orphan Drug Act, there would probably be no Biogen Idec, no Genzyme, Alexion Pharmaceuticals.
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One idea is to create an incentive for drugmakers comparable to that in the Orphan Drug Act.
AZT, began as an orphan drug for what was then an obscure disease of the immune system.
The Orphan Drug Act was passed only because of the support of the National Organization of Rare Diseases.
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Fortunately, policymakers have found ways to improve the incentives for pharmaceutical firms to invest in orphan drug research.
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Combined with the increasing number of orphan drug approvals, the data suggests compounded annual growth will outpace other drugs.
Kynamro is an orphan drug approval, meaning it was developed to treat a disorder affecting fewer than 200, 000 people.
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But while the Orphan Drug Act increased the amount of research into rare diseases, the field is still vastly underfunded.
Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act of 1983.
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They should start by taking the Orphan Drug Act already over a quarter-century old and using it as a model.
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The Orphan Drug Act, enacted in 1982, provides incentives--including tax credits and exemption from user fees--for the development of drugs for rare diseases.
At the time this process was initiated, neither orphan drug status or even the more limited Hatch Waxman period of exclusivity was guaranteed.
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In a course he teaches at Boston University, Astrue teaches students six ways of getting a drug approved when another treatment has received orphan drug status.
In fact, the Cowen analysts say, the last stock to outperform after its FDA launch was Alexion, the maker of the high-priced orphan drug Soliris, in 2007.
Thirty years ago, the Orphan Drug Act provided financial incentives to pharmaceutical companies to develop treatments for rare diseases -- diseases that afflict fewer than 200, 000 people.
As KV Pharma followers may recall, Makena was originally projected to become a big moneymaker after the FDA approved the treatment early last year under the Orphan Drug Act.
The compound annual growth rate of the orphan drug market between 2001 and 2010 was 25.8 percent, compared with 20.1 percent for a matched control group of non-orphan drugs.
The passage of the Orphan Drug Act in 1983 made it financially worthwhile for drug companies to chase treatments for disorders like multiple sclerosis, rare cancers, even rare genetic disease.
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The orphan drug act only gives them eight years of exclusivity, but it does set a precedent for giving companies an extension of their monopoly that is not based on patent protection.
Consider the Orphan Drug Act, passed in 1983.
FORBES: To Cure Rare Diseases, Unleash Orphan Drug Innovations
Analysts have interpreted that as giving Genzyme's treatment, Fabryzyme, the inside track toward getting a six-year term of exclusivity under orphan drug laws, which is meant to encourage companies to develop treatments for rare diseases.
If Pfizer were to gain approval of such an orphan drug, for example, the company might be granted more time to market its blockbuster cholesterol-lowering drug Lipitor before any manufacturer was permitted to produce a generic version of that drug.
America's drug industry also enjoys one of the lowest effective tax rates of any industry because of the extraordinary concessions made in order to persuade companies to develop drugs that might not be profitable through the orphan drug tax credit, for example.
Of course, the big show will be the suit against the FDA. The drugmaker has accused the agency of messing with the incentives provided under the Orphan Drug Act and selectively applying policy when it comes to enforcing the law concerning compounding.
Pfizer ( PFE - news - people ) were to gain approval of such an orphan drug, for example, the company might be granted more time to market its blockbuster cholesterol-lowering drug Lipitor before any manufacturer was permitted to produce a generic version of that drug.
The typical clinical trial model may not be best suited for generating evidence that a drug is safe and effective against diseases where patient enrollment can be difficult and diseases progress slowly and unevenly over a long period of time, according to Steven Grossman, who worked on the original Orphan Drug Act in 1983 and is now a policy and regulatory consultant.
Without orphan-drug privileges at home, the richer European firms head to America to get them.
This has become such a profitable pursuit that most drugmakers are pursuing at least one "orphan" drug.
Orphan-drug status, for example, can help a biotech firm raise capital on the strength of its latest product.
But an alliance of 100 rare-disease support groups from 12 European countries has coalesced around orphan-drug legislation to form Eurordis.
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