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Finding time and money for the orphans
July 2006
by Chris Anderson  |  Email the author

Most people have a soft spot for orphans. These unfortunate souls, often depicted with unusual­ly large and needy eyes, tug at the heartstrings.
Such has not always been the case in the phar­maceutical industry, where an orphan is a treat­ment for a rare disease that occurs in fewer than 200,000 people in the United States, as defined by the FDA's Orphan Drug Act, passed in 1983.
Do a little number crunching and it is easy to see why big pharma hasn't been exactly enam­ored of bringing orphan drugs to market. Pick whatever number you feel like around $1 billion that studies indicate it costs to bring a drug to market and it's easy to see why corporate share­holders, and Wall St., whose mantra is usu­ally "What have you done for me this quarter?" would have a hard time swallowing R&D funds from a large, public pharma company being fired at such a small market.
That said, there are indications that the Orphan Drug Act has had the desired affect of stimulating research and in bringing drugs to market to treat a wide range of diseases such as cystic fibrosis, multiple scle­rosis and even some rare forms of cancer.
In all, more than 100 drugs have been brought to market since the inception of the Orphan Drug Act, not too shabby a yearly average, espe­cially if viewed through the lens of recent years and the overall decline of new drugs coming to market.
One would think that with pharmaceutical companies staring directly at some significant patent expirations, as well as a rising tide of generics, that work on orphan diseases might decline, as they scramble for the next Lipitor.
But you might be wrong.
According Cynthia Joyce, executive director of the Spinal Muscular Atrophy Foundation (see E070618), the tide may be turning even more in favor of companies conducting research in rare dis­eases. "I think there is a growing recognition among pharmaceuti­cal and biotech compa­nies, that even though rare diseases are much smaller markets, there is still plenty of oppor­tunity there," she says.
Orphan drugs are more likely to be the playing field of smaller, up-and-coming companies lured by significant R&D tax breaks, grant money and exclusive rights. But there are still a handful of larger companies still willing to stick their necks out in this market.
One notable company in that regard is Genzyme, a company that virtually cuts its teeth on rare diseases long before it became what it is today. Yet, like a real-life orphan, now adopted and successful who goes back to help today's children, Genzyme still finds time and money in its business to pursue orphan drug opportunities.
Most recently, it won FDA approval to mar­ket its drug Myozyme to treat progressive mus­cle disorder Pompe disease, even though it only affects 5,000 people in the United States.
As Dan Quinn, a spokesman for Genzyme commented refreshingly in a recent story post­ed online at The "The way we look at that is it's not so much the size of the market but the therapeutic value of the drug." No fur­ther sermonizing necessary from this scribe.
But rare disease research in the coming years may also be on the upswing thanks to the May announcement that the NIH-sponsored Rare Disease Clinical Research Network (RDCRN) was launching its first clinical studies. Over the next few months, researchers at more than 50 sites will begin clinical work on 20 rare dis­eases. The $71 million that has been directed at these studies may seem like small potatoes to some, but it is surely music to the ears of any of the 25 million Americans who suffer from any of the 6,000 identified rare diseases.
And while shareholders might demand a broader customer base, it is nice to know that there still exist those willing to help a wide-eyed orphan.



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