Treating rare disorders was once considered a "good-will science endeavor," but market forces have encouraged companies to revisit how these small molecules can be big business, and researchers are finding that therapeutic applications for one rare disease area often holds promise in others. Gleevec and Enbrel are members of the billion dollar club once reserved for allergy drugs and antidepressants.
Orphan-drug companies face a Herculean task: raising visibility among needle-in-a-haystack groups. Orphan drug populations typically number less than 200,000, while an “ultra-orphan” drug can treat fewer than 10,000. Reaching out requires transcending the bias that these are minor illnesses—they're colossal to patients.
Small patient populations have their own patient groups and advocacy programs, where communities learn about treatments, interact with members and attend virtual meetings. Groups offer information on experimental therapies and provide R&D updates.
New media also plays a big role in outreach efforts. While blogs can be an important target alongside traditional media, they create compliance challenges. Orphan communities change the rules for adverse-event reporting. Some manufacturers may not want to take on the responsibility. Patients exchanging personal views online may not want opinions shared with a company. Snooping in sensitive areas can damage relationships.
Social networking offers opportunities to interact directly with focused audiences. Web video offers an intimacy unmatched by other communication channels. For patients whose are very motivated to find information, an aggressive, Web 2.0-focused campaign may be appropriate, integrating viral tactics with traditional PR.
Gil Bashe is EVP, health practice, Makovsky + Co.