In advance of Rare Disease Day 2026, GHMC convened its International Advisory Panel* for a global discussion on the realities of working in rare diseases. With experience spanning dozens of rare conditions across multiple markets, our teams reflected on this year’s theme, “More Than You Can Imagine.”
There are more people with rare diseases than you can imagine. Despite each individual rare disease having a small population, once combined, 1 in every 17 people is thought to have a rare disease. This adds up to millions of people.
Rare diseases also demand more than you can imagine: more empathy, more flexibility, more patience, and more strategic precision. They also offer more than you might expect—deeper partnerships and powerful patient stories that give us the opportunity to create meaningful change for communities that are too often overlooked.
From a global perspective, several themes related to biopharma industry communications seem universal: pricing, regulatory approval, funding, advocacy and other aspects. However, in rare disease these themes are also more nuanced than you can imagine, especially when working across markets and region
1. Emotional sensitivity is not optional
Rare disease patients and families often live with prolonged uncertainty—delayed diagnoses, limited treatment options and difficult access pathways. Communication about clinical trials, particularly ineligibility, requires exceptional care because every interaction has the potential to carry significant weight.
2. Marginalization and stigma are real
In many countries, rare disease communities have limited advocacy infrastructure. Stigma and social isolation can compound medical challenges. Communications must be culturally sensitive, locally informed and rooted in respect.
3. Access pathways are rarely straightforward
Reimbursement and regulatory environments vary dramatically. From alternative funding mechanisms in Singapore to evolving health system integration in Argentina and lengthy processes across parts of Latin America, rare disease innovation often moves through non-traditional and highly scrutinized pathways. Strategy must reflect this complexity.
4. With small patient populations, every story is consequential
Some conditions may affect only a handful of individuals in a given country. When numbers are this small, communication becomes both more personal and more consequential. Ethical storytelling and privacy considerations are paramount.
5. Foundational education is often the first step
Rare diseases frequently lack baseline understanding among policymakers, payers, clinicians and the public. Before driving awareness or action, communicators must often establish the fundamentals—what the disease is, how it presents and why it matters.
6. Pricing and public debate require thoughtful navigation
Therapies—especially those involving children—can attract intense media scrutiny. Public discourse around pricing and reimbursement can become emotional and politicized. Clear, balanced communication grounded in patient need and long-term value is essential.
7. “Rare disease” is not one category
There are thousands of rare diseases, and only a fraction have approved treatments. Many patients lack representation or advocacy support, leading to varied levels of engagement across disease types. Each disease area demands a tailored approach rather than a one-size-fits-all strategy.
8. Depth matters more than breadth
Traditional awareness metrics don’t always apply. Broad reach may be unrealistic and unnecessary. Success in rare disease communications is often measured by depth of engagement, strength of relationship, and trust built within small but highly connected communities.
9. Patients are partners, not spokespeople
Listening to patients’ diagnostic journeys and supporting them in telling their stories—on their own terms—builds authentic advocacy. Empowerment strengthens not only communications outcomes, but also community resilience.
10. Community-building drives impact
Grassroots campaigns have influenced policy decisions and accelerated inclusion of therapies in national programs. Collective advocacy amplifies voices. Visual storytelling and, when appropriate, public figures can help reduce isolation and broaden understanding.
Rare disease work requires more imagination, more patience and more responsibility than many other therapeutic areas. But it also offers the chance to make a disproportionate difference.
Need help interpreting global complexity for your brand? If you’re navigating a rare disease innovation and need experienced, globally informed communications support, we’d welcome the conversation.
* The GHMC International Advisory Panel brings together a curated network of senior healthcare communications experts from across more than 20 key global markets with more than 200 years of combined experience. The panel serves as a real-time sounding board for the most important trends, challenges and developments shaping the international healthcare landscape.
Designed to move beyond theory, the Advisory Panel translates global insight into practical guidance, helping organizations anticipate change, stress-test strategies and stay ahead in a rapidly evolving health ecosystem. We stay connected to share insights and perspectives between different markets, but we all share the same desire to connect medical advances with those who need them.