The Rare Disease Boom Is Here: Are We Educating for It?

What you’ll learn:

Rare diseases present a paradox in modern healthcare. Collectively, they affect hundreds of millions of people worldwide. Yet, each individual disease is so uncommon that many healthcare professionals (HCPs) will encounter only a handful of patients in their careers.¹ Limited clinical exposure, overlapping symptoms, and sparse treatment guidelines often result in delayed diagnosis and uncertainty around management.² This paradox also exposes a fundamental gap in how clinicians are educated about rare disease.

In this environment, pharmaceutical manufacturer–supported medical communications, across both medical affairs and commercial functions, play a uniquely critical role.

Rare Disease Innovation Has Reached an Inflection Point

Over the past decade, rare diseases have moved from the periphery of pharmaceutical development to its center.

Since 2015, therapies for rare and orphan conditions have steadily increased as a share of US Food and Drug Administration (FDA) approvals, culminating in a clear inflection point. From 2022 through 2024, more than half of all novel FDA drug approvals in the US were for rare or orphan diseases.^3-5

Rare disease therapies now account for the majority of novel FDA drug approvals.

This shift reflects both scientific advancement and strategic prioritization. Breakthroughs in genomics, biomarkers, and precision medicine have unlocked new targets across previously untreatable conditions⁶. At the same time, manufacturers increasingly recognize rare diseases as areas of high unmet need and long-term value.

This investment in rare disease therapies is translating into meaningful commercial impact. Orphan drugs are projected to represent approximately 20% of global prescription drug sales by 2030,⁷ with revenues expected to grow from ~$185 billion in 2024 to ~$270 billion by 2028.⁸ As rare disease portfolios expand, the question is no longer whether innovation will arrive, but whether the healthcare system is prepared to absorb it.

Rare Diseases Expose the Limits of Traditional Education

The education challenge in rare diseases is not only about what HCPs need to learn, but also about how they need to learn it.

Many clinicians have never diagnosed or managed a patient with a given rare disease. Evidence bases are often evolving, real-world experience is limited, and clinical decisions may carry significant consequences stemming from delayed or incorrect diagnoses and suboptimal treatments.² Asking HCPs to build confidence through one-way, lecture-style education is increasingly misaligned with both the complexity of the science and the realities of modern clinical practice.

 In rare diseases, education must build confidence… not just awareness.

Rare diseases demand education that enables dialogue, exploration, and application. HCPs need opportunities to engage with data, discuss real-world scenarios, and learn from peers navigating similar uncertainty. When education lacks interaction, knowledge may be conveyed, but clinical readiness often is not.

Why Rare Disease Education Must Be Designed, Not Just Delivered

As rare disease pipelines expand, education must be intentionally designed around how HCPs actually learn.

Today’s clinicians are time-constrained and highly selective. Generic content, static formats, and one-size-fits-all programs struggle to earn attention.⁹ In rare diseases, relevance and personalization are prerequisites. Education must either motivate infrequent treaters to engage at all, or provide specialists—who may themselves encounter certain conditions only rarely—with the depth and peer exchange needed to navigate clinical uncertainty. For specialists, that value comes not from repetition of known information, but from opportunities to engage with peers and experts, explore real-world cases, and refine decision-making in the absence of extensive clinical experience. For broader HCP audiences, such as primary care, education must be immediately relevant and practical, focusing on recognizing when a rare disease should be suspected and when to refer or co-manage, rather than on mastering treatment details.

In rare diseases, education must be designed for how—and how often—HCPs actually encounter patients.

For rare diseases, this approach is especially powerful, helping HCPs move from awareness to understanding and from understanding to action.

Early, Sustained, and Connected Engagement Is Essential

In rare diseases, education cannot begin or end at launch.

Early engagement builds foundational disease understanding well before a therapy becomes available, preparing HCPs to recognize appropriate patients and interpret new evidence as it emerges.¹⁰ After launch, sustained education remains critical. Even when effective therapies exist, many patients remain undiagnosed or untreated due to persistent awareness gaps.¹¹

Addressing this requires more than isolated programs. It requires connected learning journeys that reinforce key concepts over time and meet HCPs across multiple touchpoints. When education is treated as an ongoing ecosystem rather than a series of standalone events, it is far more likely to influence clinical behavior.

Alignment across medical affairs and commercial education is equally important. When scientific exchange and promotional education reinforce one another, each within its appropriate boundaries, HCPs receive a clearer, more cohesive narrative that builds both credibility and confidence.

The Bottom Line

Rare diseases now represent a growing share of pharmaceutical innovation, investment, and launches. As this shift accelerates, medical communications must evolve accordingly.

Success in rare diseases will not be driven solely by reach. It will be driven by education that reflects clinical reality and is designed for complexity, uncertainty, and infrequent exposure. That means motivating engagement among broad HCP audiences, such as primary care, who may never encounter a given condition, while also enabling deep, peer-driven scientific exchange for specialists navigating limited evidence and high-stakes decisions.

Meeting this challenge requires more than updating legacy programs or scaling traditional formats. It demands a fundamentally different approach to medical education, one that is grounded in deep scientific fluency, intentionally designed around how clinicians learn, and capable of operating seamlessly across medical affairs and commercial contexts.

This is where Klick brings a distinct point of view and capability. Our medical communications work sits at the intersection of medical and scientific leadership, experience design, and adult learning principles, allowing us to help manufacturers rethink not just what they communicate, but how education is experienced. By integrating strategy, content, design, and execution across functions, we help transform education from a series of standalone programs into connected learning journeys that evolve.

In the rare disease space—where exposure is limited and confidence is often the biggest barrier to action—this approach is essential. It is how Klick helps ensure that scientific breakthroughs are not only disseminated but also understood, trusted, and applied in practice, translating innovation into meaningful outcomes for patients who cannot afford to be missed.

References

Klick Health is the world’s largest independent commercialization partner for life sciences and a leading full-service pharma marketing partner, serving as agency of record for leading pharma, biotech, and healthcare brands. Klick’s specialized offerings are rooted in deep medical and scientific understanding, including market insights, award-winning creative, and proprietary AI and data models to craft impactful brand narratives and seamless customer journeys. Backed by nearly 250 medical experts and advanced healthcare analytics, Klick delivers integrated marketing strategy and communications, from new product launch strategy to MLR review with real-world evidence, helping brands thrive in today’s complex healthcare landscape. Learn more at Klick.com.