Johnson & Johnson’s Imbruvica is one example of a rare-disease drug facing competition. Imbruvica’s competitors include Genentech’s Gazyva and Gilead Science’s Zydelig — among others.

Susan Stein, president and CEO, Connexion Healthcare:

Commercialization of orphan-disease products is more strategic and targeted than traditional products. It requires in-depth knowledge of HCPs, patients, and caregivers as well as policy and patient advocacy groups. Increased physician education about rare-disease recognition is needed since many patients go from doctor to doctor for many years without obtaining an accurate diagnosis ― and, as a result, they go without treatment. Patient centricity is also critical because patients and advocacy groups are very involved. Some patients may be more educated about their disease than the doctors who treat them. Education about orphan-drug development and commercialization that addresses these complex issues is also needed. Marketers should choose partners wisely and work with companies specializing in orphan-product launches to obtain the most effective guidance in navigating all aspects of the treatment landscape.

Deborah Lotterman, chief creative officer, precisioneffect:

The rules of rare-disease therapies are radically different. Patients and their families expect engagement. They may have spent years pursuing a diagnosis. Now they’re educated, passionate, vocal. They’re embedded in their communities and online 24/7. Their relationships — with HCPs, with brands — can be deep and enduring. Expectations are high and their memories are long. Similarly, physicians who treat these patients are highly specialized and well-versed in the data. Those are the broad strokes. But each rare disease is unique. The primary challenge is to deeply understand patient/caregiver/HCP needs and aspirations to develop the brand’s content and support that fills genuine gaps.

When competing for a handful of patients, each of whom represents long-term brand loyalty, healthcare brands must deliver the type of service that we see with high-end consumer purchases. These brands are a terrific model as they invest heavily in developing customer insights with highly qualitative research, from ethnographies to long-running smart communities that can help shape messaging and service offerings.

Christopher Tobias, president, Dudnyk:

In the past, developing a treatment for a rare disease usually came with an extended period of time with little to no product competition. But that is no longer the norm, and the rare-disease landscape is moving toward something more familiar: a competitive market. It is not uncommon now for a rare-disease population to have three actively promoted products, along with two more in the pipeline. Great advancements in orphan-product development are taking place in drug delivery, targeted therapies, and even gene therapy and biologics.

Marketers today may overlook a very important component of rare-disease marketing in this new landscape. The relationship a manufacturer builds through support programs with a patient who has a rare disease is even more important today than ever. Rare-disease patients are tenacious, empowered, and almost warrior-like in their abilities to advocate for treatment and understanding. And now they have many choices. They may be loyal to a manufacturer that has held their hand, supported them and their family, and provided a solution to their unique challenge. Yes, the landscape of competition in rare diseases is changing, but it will only drive greater support for the patients, who so desperately deserve these solutions.

Leon Behar, CEO, 81qd:

We believe the more pressing challenge for marketers of drugs for rare diseases, and all stakeholders in the rare-disease space for that matter, is still an earlier and accurate diagnosis. This is the critical, rate-limiting step to getting patients the care they so desperately need. It is in the best interest of every stakeholder in the rare-disease space to get the right patient on the right therapy as early as possible. This challenge serves as a catalyst for the use of data analytics to facilitate the identification of both misdiagnosed and yet-to-be diagnosed patients with rare diseases and the specific clinicians who are currently managing those very patients for other conditions.