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
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