A New Era of Individualized Care:
A Lesson From Orphan Drug Marketing

 Michael Marino

Michael Marino / Founder & CEO


From health coverage to cost to documenting outcomes, the perceived chaos and general unknowns surrounding healthcare is only worsening among patients, physicians, and payors. With impending pay-for-performance measures—and the inevitable avoidance of high-risk patients by providers—more and more patients have no choice but to reluctantly assume responsibility for their own health.

Across all disease categories, the impact of these uncertainties falls upon patients and physicians alike. They are desperate for support, clarity, and a new level of medical attention that goes beyond simple receipt and administration of therapeutics.

This ever-expanding gap in healthcare delivery represents a key opportunity for biotech and pharmaceutical marketers to step in with a level of highly personalized attention that helps anchor patients’ own care and wellness. By taking a more individualized approach to marketing and communication, firms have the capability of building equities with greater depth and breadth among their constituents—equities that can be immediately accrued to the brand.

Such personalized marketing and relationship-building strategies have historically been employed by highly successful orphan drug manufacturers/marketers like Genzyme, Sigma Tau, and others. Genzyme brands effectively engage with some of the world’s smallest patient and physician populations, and have established bonds with constituents that have withstood the test of time, competition, and even product unavailability. Companies like Raptor Pharmaceuticals employ patient access managers to connect patients and their families to a wide-range of both internal and external resources.

However, it is clear that this method can be applied even across large, mainstream indications by larger, less specialized manufacturers. Pharmaceutical products of all types, serving all size patient populations, can successfully deploy many of the rare drug marketers’ most targeted strategies to seize upon the current care gap in the market and thereby return value for their brands.


Why should a brand look to orphan drug strategies?

The rare disease therapeutics category began a generation ago with the US government commitment to underwrite development costs in order to incentivize manufacturers to market “orphan drugs” for disease states with very small patient populations. Sanofi Aventis’ purchase of Genzyme for $20 billion, as well as pharma and biotech M&As like Roche and Genentech, and Lilly and ImClone—along with Pfizer’s creation of an Orphan and Genetic Diseases Unit—demonstrate that the marketing of therapies for rare diseases has been recognized as a lucrative business opportunity.

Rare disease drug marketing is notable for its level of personalization—small patient populations and small cohorts of practicing physicians make one-to-one marketing a very effective option. This has created audience and brand relationships that in some cases proceed on a first-name basis.

It is this type of relationship that can serve to bridge the widening gap in care, and deliver on unmet patient and physician needs. For a given indication, a single brand has the opportunity to act as a patient-centered personal-care resource—whether by helping a patient track therapeutic goals and delivering lifestyle-specific exercises or wellness solutions, for example, or assisting a physician in aggregating his or her own practice data to better assess patient progress.

Advances in IT now allow for the hyper-segmentation of both professional and patient targets to create markets of one, and advances in digital marketing channels enhance brand experiences of all kinds, especially virtual one-on-one conversations with customized content, channels, and engagement strategies.


Big Arrow Group: bringing orphan drug strategies to larger markets

Big Arrow Group, Agency of Record for Genzyme, has helped develop not only the concept of personalized genetic health but the execution of personalized genetic marketing as well. Big Arrow focuses on delivering value-added communications and tactics to hyper-segmented audience—with relevant marketing, branding, and communication strategies to help pharmaceutical marketers succeed, no matter what size patient population they serve.

Big Arrow has guided Genzyme’s global LSD group in analyzing and addressing patient segments and the physicians who treat them, asking, where are our patients on the spectrum of symptoms? Diagnosis? Condition knowledge? Treatment initiation? Therapeutic goals? What are the physical and emotional barriers to advancement along this spectrum? And perhaps most importantly, where are patients on the spectrum of their own return to “normalcy”?

Because for many orphan diseases, successful treatment hinges on early initiation and diligent, lifelong adherence to challenging therapeutic regimens, Raptor Pharmaceuticals—whose product PROCYSBI for nephropathic cystinosis was launched globally with Big Arrow—invests heavily on facilitating and assisting adherence through a variety of personal and online programs, for both patients and their caregivers and families.

As for physicians, where does each stand on condition familiarity and understanding? Treatment choices? Treatment preferences and protocols? How do they perceive their practice-based body of data for the condition? And are they true champions of the patient? The disease? Its treatment?


Adopting orphan drug strategies for mainstream markets

Addressing hyper-segmented audiences demands rigorous research and planning, as well as a proven infrastructure for analysis, assessment, and application.  When this information is plotted against business and behavioral objectives, the brand platform begins to take shape.  And with highly targeted tactics, a powerful marketing effort takes form—one that opens doors to one-on-one relationship-building with patients and physicians as important individuals, rather than members of a generalized target.

Part and parcel of this effort, we carefully and thoughtfully craft the brand—its image, its voice, its identity, its connection to patients, physicians, and other stakeholders—to establish “irreplicable” bonds and equities that help insulate the brand, establish clear leadership positions, and avoid commoditization.

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