Because the development of such products often would be prohibitively expensive and unprofitable under normal circumstances, the United States adopted a law to encourage work in this important arena. According to the Orphan Drug Act (ODA), which was passed in January of 1983, an orphan drug is one that is used to treat "any disease or condition which (a) affects less than 200,000 persons in the U.S. or (b) affects more than 200,000 persons in the U.S. but for which there is no reasonable expectation that the cost of developing and making available in the U.S. a drug for such disease or condition will be recovered from sales in the U.S. of such drug."

Because researching and developing these types of drugs is a financial burden and often untenable, the companies that do so receive tax reductions and marketing exclusivity on that drug for an extended time – seven years after government approval. The hope is that these rewards will inspire more companies to fund research.

Neil Berliner: How do representatives for your company differ from representatives who work for more traditional pharmaceutical companies, if at all?

Paul Stickler: Our representatives are very dedicated to patients with rare and often complicated medical problems. They tend to have larger territories than representatives from other companies, partly because there are fewer patients per capita who have these uncommon conditions. They also tend to be very strongly based in science.

Do you, therefore, avoid hiring people directly out of college?

Yes. Our representatives are all very experienced; we hire account managers who have demonstrated consistent, superior sales results, and outstanding planning and organizational skills, and are also looking for the gratification that comes with working with products for smaller patient populations where there is a significant need for treatment. In fact, our representatives often see, firsthand, the results that our products can bring to patients.

Do you think that your representatives have easier access to doctors than other representatives?

Our representatives are often, in fact, sought out by physicians. They enjoy good relationships and are given ample time by many doctors because of the special needs that they and our products meet.

I see that you also have some relatively well-known products in your portfolio, such as Cogentin and Tranxene. Do companies like yours need a mix of products to help stay financially viable?

We have a diversified portfolio with about 20 products. Our strong commercial base combined with our strong development pipeline enables us to bring forward products that may not be "blockbuster" products by today's standards, but they take on blockbuster significance for certain patient populations where treatment options are limited but essential.

How do potential products for small markets come to your attention?

Interestingly, besides doing our own research, physicians often bring medications to our attention. If a doctor is treating a rare condition, he or she may become aware of a medication in use outside the United States and can inform us of that medication's potential.

Could you give us an example of a rare condition that is treated by one of your products?

Yes. One example is acute intermittent porphyria, a rare metabolic disorder in the production of heme, the oxygen-binding prosthetic group of hemoglobin. Our product, Panhematin, is an enzyme inhibitor derived from processed red blood cells that's used to treat this serious condition.