In the U.S., rare diseases are classified as any disease that affects fewer than 200,000 Americans. Many of these conditions are also considered “orphan diseases,” for which there is minimal research and therapeutic development. This is partly due to the limited market potential for drugs in the space, but also due to the sheer volume of rare diseases: approximately 6,000–8,000 have been identified. As the saying goes, they are individually rare but collectively common. Many people, cumulatively, are affected. This fuels an enormous need for effective patient communication and education as new therapies move through the biopharma pipeline.
Marketing to rare disease patient populations is equal parts opportunity and responsibility. Below we share some strategies for mutually beneficial engagement and education, specifically for U.S. audiences (direct-to-patient advertising rules are fundamentally different overseas). The overarching theme: As with rare disease drug development, precision and personalization are key.
First: It’s Not an Audience, It’s a Person
While the number of people affected by a given rare disease may be small, finding an effective treatment is enormously important to those individuals and their loved ones. No marketer or communicator trying to engage this audience should ever lose sight of that lived experience. With this in mind, our efforts to reach these people should focus on providing answers, information, and hope—with all the necessary caveats.
Those of us in the biopharma industry know how fraught drug development can be. We have a responsibility to communicate the uncertainty of the journey to patients—likewise, the limitations of the therapy and the eligibility requirements for the clinical trials. Does it help everyone with this disease or a subset who carry a specific mutation? Can it reverse health issues, or will it be indicated for newly diagnosed patients to slow disease progression? These small nuances are important to individual patients and their families, who may be in the most challenging moments of their lives. Humanity, compassion, and empathy should form our constellation of north stars.
Regulatory Compliance and Ethical Considerations
It may sound like an obvious statement, but to engage patient audiences as a commercial entity, someone needs to keep a regulatory eye on the program. It’s not just right from a legal perspective; it’s ethically important that guardrails are in place to keep everything on the straight and narrow. This will foster trust and transparency while keeping you on the right side of the U.S. Food & Drug Administration (FDA). It will also help patients set appropriate expectations around safety, efficacy, and their potential eligibility for your investigational drug.
To do this consistently, biopharma companies need to stay abreast of regulatory guidelines and compliance requirements related to pre-approval healthcare marketing, education, and patient privacy. While most FDA regulations are largely fixed, guidelines on how to interpret those regulations continue to evolve. As do the tools and channels we use to communicate our messages (for example, dynamic payper-click ads).
We always recommend companies work closely with their legal and regulatory teams to ensure that marketing materials adhere to industry standards and ethical guidelines. If that team is not yet in place, consider hiring a regulatory consultant who will advise on what can and can’t be done. It’s better to pay a good one than to receive a letter from the FDA.
The Right People, in the Right Places, at the Right Time
Without question, the marketing landscape for treatments of rare and ultra-rare diseases can confuse and befuddle even experienced healthcare marketers. It requires a highly customized, informed, and inspirational approach.
Unlike broader campaigns, you’re speaking to a very finite audience. However, they’re also individuals who differ based on demographics, disease subtype, treatment history, geographic location, and more. This is where omnichannel marketing can be particularly helpful, using data to personalize each person’s marketing experience so it’s not redundant or repetitive across channels or over time.
Determining what channels to activate is another important decision. A strategy for marketing to rare disease patients should be comprehensive and dynamic, uniting both traditional funnel-based marketing and digital media strategies into an omnichannel program. While there are too many to list, below are some of the channels and approaches we have found particularly successful.
Educating Through Resources
Education is a critical component of engaging any patient population, no matter how large or small. Therefore, the backbone of the program should be comprehensive educational materials that explain the science behind precision medicine and its potential impact on treating a rare disease. This is best achieved by creating a dedicated education website that uses symptombased keyword searches, patient impact studies, and advice hotlines.
Partnering with Patient Groups
Patient advocacy groups (PAGs) hold the keys to both access and credibility. They don’t give away those keys lightly—and rightly so. They’re critical gatekeepers for these patient communities. With this in mind, relationships with PAGs should always focus on adding value and contributing to the community instead of leading with a request or expecting immediate access. Start early, pay it forward, and then evolve the relationship as your program moves through the clinical trial phases. As you approach commercialization, they can serve as a partner for financial support and patient assistance programs, which can be co-developed to ensure medication accessibility and affordability.
Utilizing Digital Media for Scalability
Digital media should be highly customized pre- and postcommercialization. These efforts and messages can be scaled to the next patient group after commercialization. There are many digital channels through which to serve content, and educational channels targeting doctors and patients are used to spread awareness about the new drug.
Leveraging Online Platforms for Patient Identification
Undiagnosed patients often seek solutions on online chat forums and social media, providing valuable clues through their search behavior and discussions. Keywords and behavioral patterns can be analyzed to identify potential patient groups and feed the top of the marketing funnel. We can’t make assumptions about a person’s disease status and serve them content accordingly. We can, however, position ourselves as a highly visible resource for key topics in this area.
Educating Healthcare Providers (HCPs)
Through Social Media Sponsored ads on social media apps can be used to promote peer-reviewed medical research and clinical studies. Some closed networks and forums also allow for highly targeted advertising based on geographic, socioeconomic, and clinical interests. While HCPs are a distinct audience, they ultimately help patient communities make sense of the information you put out there. They need to be armed with the knowledge to do so effectively.
Machine Learning and Look-a-Like Patient Targeting
AI and machine learning are increasingly used to identify audiences with similar characteristics to existing patients. Tracking and analyzing such data enables targeted digital advertising. Personalizing the Messages Reaching a patient or caregiver through the right channel does very little (or could even backfire) if the message is not personalized to them and their experience. To prevent their message from falling flat, companies need insight into the patient journey and those guiding these programs need empathy to understand them. This comes through direct interactions and working closely with patient advocacy and other groups.
Rely on patient insights and the patient voice to guide the tailoring of messages that align with the preferences and needs of different patient segments. Then, utilize data and marketing automation to deliver personalized content based on recipient’s interests and engagement history.
Allow this to be a conversation, make efforts to participate in the patient community, and take feedback where necessary to hone the messages and stories with those learnings. Finally, highlighting and amplifying the personal stories of people living with rare diseases can only improve awareness, understanding, and empathy for the condition and potential treatments. Instill confidence and inspiration by enabling people to see themselves in the struggles and triumphs of others.
A Final Word
Marketing to small patient populations in the era of precision medicine requires a multifaceted approach that prioritizes education, community engagement, and ethical considerations. By leveraging segmentation, educational content, digital marketing strategies, personalization, and regulatory compliance, healthcare marketers can create impactful campaigns that resonate with individuals affected by rare and orphan diseases.
As precision medicine continues to advance, embracing these best practices will be essential in driving awareness, access, and adoption of tailored treatments for small patient populations worldwide. If we are sophisticated with our methods but open with our hearts, we can support these small patient populations and pave the way for a future where the benefits of precision medicine are available to all who stand to benefit.
