Without the Orphan Drug Act, there would probably be no Biogen Idec, no Genzyme, Alexion Pharmaceuticals.
FORBES: How The Odd Couple's Jack Klugman Helped Create The Biotech Industry
One idea is to create an incentive for drugmakers comparable to that in the Orphan Drug Act.
The Orphan Drug Act was passed only because of the support of the National Organization of Rare Diseases.
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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.
FORBES: How The Odd Couple's Jack Klugman Helped Create The Biotech Industry
They should start by taking the Orphan Drug Act already over a quarter-century old and using it as a model.
FORBES: To Cure Rare Diseases, Unleash Orphan Drug Innovations
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.
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 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.
FORBES: How The Odd Couple's Jack Klugman Helped Create The Biotech Industry
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
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.
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.
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