Main Menu

From Information Scarcity to Support

What Rare Disease Patients Need
& How Marketers Can Help

Rare disease patients are redefining how they seek, trust, and engage with health information.

Rare diseases represents a significant and often overlooked healthcare imperative in the U.S. with tens of millions of Americans living with a rare disease (one that affects fewer than 200,000 people). Today, more than 7,000 rare diseases have been identified, yet over 90% lack an FDA‑approved treatment*. Even when treatments do exist, patients, caregivers, and physicians frequently encounter long, complex healthcare journeys marked by limited disease‑specific information. These information gaps can delay diagnosis and underscore the urgent need for personalized education and support.

Healthcare marketers are uniquely positioned to help address these challenges. Our current MARS Consumer Health Study, covering 26 conditions with a prevalence of 1% or less among the U.S. adult population, highlights personalized insights on sources they trust for health information, support they need and channels they utilize to stay informed.

Rare disease patients^ value doctors less, self‑research more

Rare disease patients^ are less likely than the average patient to rely on their doctors as their primary source of health information and are more likely to conduct their own health research. While 90% of patients overall value their doctor for health information, that figure drops to 76% among rare disease patients. As a result, many adopt a more proactive role: 66% research treatment options independently and then bring their findings and questions to discussions with their physicians. Despite this high level of engagement and self-advocacy, rare disease patients^ still have significant gaps in health information.

17% less likely than avg. patients to:
Value doctors for health information

16% more likely than avg. patients to agree:
“I research treatment options on my own and then ask my doctor about them”

Supporting rare disease patients^ where they struggle & where they engage

Rare disease patients often face a high daily burden that shapes how they engage. Compared with average patients, they are more likely to report difficulty completing everyday tasks and are less likely to feel they can fully promote their own health and wellness.


At the same time, these patients are significantly more engaged with digital tools, using patient portals at higher rates to access records, review results, and communicate with HCPs. Together, this points to a need for empathetic, practical support that reduces day‑to‑day strain while leveraging digital channels to deliver accessible, personalized, and ongoing care.

Graphic featuring three patient insight statistics with circular lifestyle photos. Top section: “18% less likely than avg. patients to agree: ‘I do everything I can to promote and maintain my personal health and wellness’” alongside an image of a man jogging outdoors. Middle section: “13% more likely than avg. patients to agree: ‘My condition makes it difficult to do / complete day-to-day tasks’” next to an image of a person sitting outside a building. Bottom section: “29% more likely than avg. patients to use a patient portal to access health records, view test results, communicate with an HCP” paired with an image of a woman working on a laptop at home.
Bar chart titled “Most Valued Sources of Health Information (Very much/somewhat)” comparing Rare Disease Patients (blue bars) with Average Patients (gray bars). Condition or health-related events: 77% rare disease patients vs. 69% average patients. Drug company/brand websites: 68% vs. 59%. Online communities or support groups: 67% vs. 58%. Email newsletters: 56% vs. 47%. Social networking sites: 52% vs. 46%. Social media health influencers: 52% vs. 43%. Across all listed sources, rare disease patients report higher value than average patients.

Beyond patient portals, this population places greater importance on digital, social, and peer‑driven education than the average patient. They actively seek opportunities to connect with others who share similar experiences, whether in person at disease‑related events or online through social networks, influencers, and condition‑specific communities.

 

This behavior underscores the need for personalized, condition‑focused messaging and reaching them in areas that are dedicated to their condition. Pharmaceutical company websites also play a role in making sure patients are educated on the latest treatment information that they will likely bring to their next doctor appointment.

Ultimately, rare disease patients need more than just access to information, they need education and support that recognize the daily burden of their condition, meets them in the digital and community spaces they already use, and respects their role as active participants in their care. Clear, condition‑specific education can help support them in navigating complex care journeys.

While these initial insights scratch the surface of understanding what rare disease patients need and how to reach them, we understand marketers’ need deeper insights to better personalize marketing plans. To meet this need, M3 MI is launching a new syndicated rare disease solution featuring dedicated studies for Patients & Caregivers and for Physicians (PCPs and Specialists). Available in Q3 2026, these studies will provide essential insights into the rare disease information journey, supporting more personalized, patient‑centric engagement while helping to close information gaps and reduce diagnostic delays.

Sources: Rare Disease Facts & Statistics M3 MI's 2025 MARS Consumer Health Study ^ Rare conditions include Sickle Cell Disease, Hemophilia, Lupus, Narcolepsy and rare cancers (Ovarian, Liver, Multiple Myeloma, Lung (SCLC or NSCLC), Pancreatic)

Information

For more information on detailed study results from M3 MI, including at the specialty level, contact us at
info@M3-MI.com.

Get in Touch