The Orphan Opportunity

This year marks the 25th anniversary of the Orphan Drug Act, a landmark piece of legislation that has altered the fate of patients living with rare diseases in this country. According to FDA, one in 10 Americans is diagnosed each year with a rare disease, and there are 6,000–7,000 rare conditions and diseases worldwide.

It's important to appreciate the impact the Orphan Drug Act has had over time. The legislation provides incentives for pharmaceutical companies to invest in orphan drug development. (Drugs receive orphan drug designation when they treat conditions affecting fewer than 200,000 Americans.) Prior to the Act's passage, only 47 rare-disease drugs were approved in the US—and of those, only 10 had been developed by the US pharmaceutical industry. But since the Orphan Drug Act's inception, more than 1,800 treatments have been granted orphan drug designation, and 326 orphan drugs have been approved by FDA. These drugs have made an impact on the lives of more than 25 million Americans living with rare diseases.

Rare diseases require special attention for drug development. An estimated 85 to 90 percent of these diseases are serious or life threatening—and patients affected by these conditions very often have no treatment options. And while there is momentum for developing orphan drugs in the US, much more has to be done to help the vast number of patients still waiting for life-altering treatments.

Incentives for pharmaceutical companies—ranging from tax relief to marketing exclusivity—have encouraged orphan drug development. But as powerful new research tools help identify new medicines for specific genetic disorders in small patient populations, more companies that have not previously pursued orphan drug development may see the benefit of building this segment into their growth strategies.

It's important to recognize that even if a product for a rare disease receives FDA approval, there is still difficulty in getting these new drugs to patients. Standard industry marketing practices may not suffice. However, there are many similarities among patients affected by rare diseases, and these can provide important case studies for the industry.

For example, Ovation Pharmaceuticals markets Panhematin (hemin for injection)—which was, in fact, the first drug approved under the Orphan Drug Act. The product treats acute porphyrias, a group of rare genetic disorders that affect a small patient population (about five to 10 out of every 100,000 people). If left untreated, the condition can lead to long term or permanent paralysis, coma, or even death. Panhematin remains the only FDA-approved therapy for patients suffering from this disease.

With three orphan drugs on the market, three more in the pipeline, and still other products for small patient populations, Ovation has developed a unique set of marketing skills to bring these specialized therapies to market. Below, we include the success factors that are instrumental in commercializing and effectively delivering orphan drugs to patients who need them.

Create meaningful relationships with product champions To connect the right products with the right patients, it's important to establish close, collaborative relationships with advocacy organizations and physicians who champion rare diseases. These collaborations are mutually beneficial. Such relationships ensure that physicians and patients are well informed, and have access to disease-state information and products they need to manage the patient's condition. In turn, pharma companies benefit from an educated marketplace.

So how can companies create meaningful relationships? The key to building a valuable relationship begins with developing a strong understanding of the advocate's needs and perspectives, and ends with helping them accomplish their goals. In other words, by directly addressing a doctor's objectives, you can 1) make authentic connections with physicians who care for patients with rare diseases, 2) provide important patient and professional education, and 3) demonstrate the value of your product. Ultimately, advocates and physicians are looking to find a cure for a disease and are hoping that the pharma industry can offer a means to achieve this goal.

Be creative with data sources when traditional audit tools come up short New orphan drugs are very often the first available therapy for a specific disease, and as such, marketers must often start from scratch to identify and define new market segments and key physicians. The challenge for companies is to find both the small patient population affected by the condition as well as the appropriate physicians without the benefit of preexisting data.

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.

How does this education translate into better use of orphan drugs? As an example, patients who historically presented with acute porphyrias often jumped through hoops due to misdiagnosis—some going so far as to have unnecessary surgeries—when in the end, a simple urine test would have helped uncover the enzyme deficiency associated with this condition. Given Ovation's targeted education efforts with physicians, now enhanced with Tablet PC technology, an increasing number of physicians are able to better understand the disease, its signs, and its symptoms, and identify new patients in need of treatment. In fact, over the past five years, the number of new accounts (physicians and institutions) prescribing Panhematin has increased by nearly 30 percent.

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.