Unlike the introduction of a new diabetes drug, for example, where data already exists listing the prescribing physicians, orphan drug marketers must pursue more creative strategies. The use of prescription data wouldn't necessarily help identify physicians who treat a rare disorder where there are no current drug therapies on the market. Yet marketers can combine prescription data with medical claims or hospital discharge data to gain insight into symptoms being treated, which can help them identify physicians who have already diagnosed a specific rare disease, even in the absence of an approved drug therapy.

Develop a consultative sales force When it comes to rare diseases, physicians often face the daunting task of identifying and properly diagnosing conditions they may never have seen or heard of. It's common for rare diseases to go misdiagnosed for years, either because they mimic symptoms common to other conditions, or simply because physicians don't know about the disease. It is thus imperative that pharmaceutical companies arm their sales forces with specialized knowledge, enabling them to take on a consultative role and educate doctors not only on drug indications, but also on the diseases and conditions they treat.

We've done this by equipping our sales reps with Tablet PCs and closed-loop marketing software that brings into focus the rare diseases our drugs treat through animation, videos, and easily accessible, peer-reviewed data. Tablet PCs essentially allow sales reps to customize their meetings to address the individual needs and interests of each physician, thereby enhancing disease-state education.

Scale infrastructure to match the market you're pursuing Small markets that characterize orphan drugs possess inherent efficiencies, and to be successful in this space, companies must maximize them. Enlisting fewer sales reps to support a number of drugs helps maintain a leaner SG&A spend. However, even with a smaller scale sales force, at Ovation we maintain incredible coverage because we reach a small but influential base of prescribing physicians. In addition, our reps—like other specialty reps—take a portfolio approach with physicians, gaining greater efficiencies by detailing a portfolio of brands in a single category.

Opportunity Remains

The Orphan Drug Act is perhaps one of the best examples of the government and industry coming together to make a significant difference in people's lives. As an industry, we should take great pride in our contributions to advancing the availability of orphan drugs. While celebrating our success, let's also remember that we have only scratched the surface.

Currently, more than 300 orphan drugs are in human clinical trials or awaiting FDA approval. While it's encouraging to see the strong appreciation for orphan drugs, bringing them to market—and realizing the legislation's true vision of bringing hope to those in this country with unmet medical needs—is an endless pursuit.

Looking back over the last 25 years, the Orphan Drug Act has achieved its intended goal: opening doors that have encouraged pharmaceutical companies to produce lifesaving medications. We must all recognize, however, that it's not enough to develop and obtain approval for these drugs alone. Equally vital to realizing the Act's mission is that we do whatever we can to ensure that patients with rare diseases who depend on us for innovation actually benefit from our investments.

Jeffrey S. Aronin is CEO of Ovation Pharmaceuticals. He can be reached at (847) 282-1000.