Building bridges to rare disease patients

Pharmaceutical CommercePharmaceutical Commerce - September/October 2012

Orphan drug development and marketing have important distinct requirements for R&D, regulatory and marketing

Have you noticed that interest in rare disease product development isn’t as “rare” as it used to be? Currently about one-third of the drug development pipeline is targeting rare (or “orphan”—the two are used interchangeably) diseases, which are defined as those that affect fewer than 200,000 people in the US. Approximately 10% of the US population has one of the orphan or rare diseases. Congress, with the encouragement of the FDA and patient advocates, created the Orphan Drug Act of 1983 to help motivate and incentivize pharmaceutical companies to invest in developing treatments to better meet the needs of rare disorder patients.

Rare disease patients are interested in being collaborative stakeholders in drug development, and patient communities are a potent factor in

Logo of the Global Rare Disease Day, emphasizing a collective holding of hands to address rare disease needs. Credit: Eurodis

conducting research and clinical trials, and partnering with pharma for a drug’s commercialization. Patient insight, feedback, and data relative to science, medicine and quality of life are critical to identifying gaps and opportunities that are unique to the rare disorder patient perspective on the disease. The FDA Rare Disease Unit already involves patients directly through its Patient Representative Program, in which patients work with the FDA and sponsors throughout the product development process. I believe that this patient-centric model is the future of healthcare as we move toward personalized medicine that is customized to patient populations based on their genetic information.

The long road to diagnosis

The conventional expectation is for a patient to rely on a physician for an accurate diagnosis of a common disease and an effective treatment. Often neither of these presumptions applies to a patient with a rare disorder. Given the small universe of patients, a typical physician might never encounter a rare disorder patient over the course of his or her entire career. Physicians may not have the expertise to recognize or diagnose a rare disorder. A biotech or pharmaceutical company can play an important role in education—not only for patients but also for physicians.

It can take several years for a patient to receive a correct rare disorder diagnosis. Misdiagnosis often results in poor quality of life for the patient and, potentially, side effects from medication that’s incorrectly prescribed. Even when the correct diagnosis is made, the majority of patients are faced with the reality that there is no treatment. An FDA-approved therapy is available for only about 5% of the 7,000 rare disorders and diseases.

Facing endless healthcare questions without many answers, rare disorder patients often take the lead in driving and managing their own diagnosis and treatment. Understandably, they rely heavily on the Internet for information that may not be available anywhere else. “Dr. Google” is frequently consulted when searching symptoms or treatment options, verifying a physician’s diagnosis, or finding information about a prescription drug. For rare disease-focused pharmaceutical companies, there’s a need for digital expertise rather than a reliance on sales representatives and advertising. Digital connections and communications specific to the rare space are required to effectively connect with key opinion leaders (KOLs), patient advocates and patients to provide information and resources for diagnosis, available treatments and, potentially, enrollment in ongoing clinical trials leading to new therapies.

For a newly diagnosed rare disorder patient, the Internet and social media provide a necessary bridge to connecting with others in the same situation. Treatment information and advice from fellow patients can be critical to adopting the most effective disease management regimen. Supportive relationships formed between rare disease patients have a positive impact socially, emotionally and psychologically—especially when managing chronic care, which is the case for most rare disorders.

Rare disorder patients (and their caregivers) want pharmaceutical companies to engage with them. If there is no pharmaceutical company investing in their disease, and the information or resources needed aren’t available, they’ll move forward independently. They can be extremely motivated knowing the critical gaps that need to be filled. Patient advocates have been known to fund research, initiate clinical trials, buy promising drug therapies and start their own pharmaceutical companies.

Patient advocacy

There usually isn’t a single unified voice from a patient advocacy organization that represents the entire patient community for a particular rare disease. Rather, there are many voices—often with conflicting messages. It’s messy. So it can be challenging for a biopharmaceutical company to identify an appropriate patient advocate or patient advocacy group (PAG) for collaboration with the brand.

The Internet is like a giant digital soapbox that can give every patient visibility and an amplified voice. Sometimes a patient or caregiver who doesn’t agree with the larger PAG will start a new organization for the same disease. Even sophisticated patient advocacy organizations, such as the Cystic Fibrosis Foundation, have competitors that have demonstrated dissension from the ranks. PAGs vary, too, in their level of professionalism.

The National Organization for Rare Disorders (NORD) works on behalf of all rare disorder patient advocacy organizations, patients and caregivers. As a NORD board member and the mother of a daughter with a rare disorder, I appreciate how NORD represents the collective interests of patients and caregivers, as they did recently with helping to shape the Safety and Innovation Act to include provisions for many issues specific to rare disorders.

Henri Termeer, former chairman of Genzyme, established that investment in rare disorder therapies can be a viable business model for biopharmaceutical companies. The burden of illness for a rare disorder, the cost of not treating a rare disorder, the cost for misdiagnosis and the impact on a patient’s quality of life, as well as the cost of clinical development and patient support programs, are all taken into consideration when investing in development and pricing a therapy for a rare disease. Value-based pricing enables a biopharmaceutical company to earn a return on a risky investment while fulfilling the critical need of providing better health outcomes for the thousands of rare disorder patients awaiting life-saving therapies.

By providing relevant information and resources that address the unmet needs of patients as quickly as possible, a brand can become a consolidator and centralized resource for education and access. A company entering this space should consider itself to be an active partner with the PAG. Alignment with a PAG should be built on the intent to do the right thing with an assurance of transparency in all interactions. Ultimately, it’s possible to create a good business with good outcomes for patients.

As always, compliance is mandatory and must be standard practice. Working in the highly regulated pharmaceutical industry, at Siren Interactive we always recognize the responsibility we have to our clients, and have made compliance with all current promotional regulations and guidelines that govern the biopharmaceutical industry an essential element of our approach to our work. All Siren Interactive staff have been certified (by the Center for Communication Compliance) in regulatory compliance for Internet Promotion/Social Media and in Patient Relationship Marketing (PRM). As such, I take great pride in the fact that Siren Interactive is the nation’s only company to receive this dual certification in regulatory compliance.

Relationship marketing

By focusing on a relationship marketing approach of educating patients, caregivers and HCPs before selling the brand, pharma earns acceptance and trust in patient communities. This is critical to initiating new patient starts and maintaining adherence to therapies.

Effectively engaging patients requires the ability to identify where patients are online and understand what they need. We do this through Siren Interactive’s MicroTargetingSM, our proprietary approach for finding rare disorder patients and caregivers online, and reaching them with relevant information. It enables a connection between patients and online content through search engine marketing and online advertising.

Another proprietary service, Siren Interactive’s Rare Online IntelligenceSM, identifies the patients’ unmet needs, and their conversations about how they refer to themselves, the disease and treatments. Results are often different from what might be expected. Although pharma marketers typically can’t engage in the online conversation, they can identify keywords that align with the most relevant issues and topics. This insight is integral to developing search and social media strategies with relevant messages and compelling opt-in content that delivers value to patients and caregivers.

The marketing challenge

Using the insights from Siren Interactive’s Rare Online IntelligenceSM we develop a strategy to achieve the client objectives through measurable tactics which can include:

  • Pay-per-click advertising on popular search engines and/or on Facebook
  • Unbranded websites with disease awareness information to help with diagnosis
  • Websites for a commercial therapy brand, which may have dual navigation for information targeting patients, caregivers and healthcare providers
  • Video, a powerful medium, combined with YouTube—particularly well suited to pharma since comments can be turned off

The pace of progress

Fig. 2. A campaign by Siren Interactive, for Lundbeck’s Panhematin product, guides sufferers of the rare disease porphyria to resources.

Over the past 12 years, Siren Interactive has pioneered the niche industry category of relationship marketing for rare disorder therapies. We’ve found that there’s no typical timetable for the commercialization of an orphan drug. But there is an optimal path that pharmaceutical companies can follow with patient collaboration at every step; and involving patients from the beginning of the drug development process can expedite the timeline. Desperate for an FDA-approved therapy, rare disease patients push for clinical trials and are eager to participate. Often, a drug being developed for a rare disorder is the patient’s only potential hope for survival. Once a therapy is approved for a rare disorder, the collaboration with patients and patient advocates that has been developing from the point of the initial research provides the basis for authentic and transparent communication. This is necessary to help establish trust to engage new patients and maintain adherence to the therapy.

Developing rare disorder therapies is about recognizing opportunities and providing solutions. From a business standpoint, the time is right for additional biopharmaceutical companies to look to rare disorder therapies as an opportunity to maintain growth and to serve patients. Doing so applies a company’s scientific expertise to develop life-saving treatments for patients suffering with a rare disorder. It’s good for patients, and it’s good business for pharmaceutical companies.


Wendy White is founder and president of Siren Interactive ( The agency specializes in digital relationship marketing expertise for rare disorder therapies. Siren Interactive received the 2011 Manny Heart Award for philanthropic work in rare diseases, is recognized on the Inc. 5000 list of the fastest-growing privately held companies, and is the nation’s first and only marketing agency with dual regulatory compliance certification for Patient Relationship Marketing and Internet Promotion/Social Media. Wendy is the mother of a child with a rare disorder, an author and a board member of the Healthcare Businesswomen’s Association and the National Organization for Rare Disorders (NORD).

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