Data, Evidence, Education: Tackling the Global Multi‑Trillion‑Dollar Rare Disease Burden
February 25, 2026 | Wold Health Assembly, World Economic Forum, rare disease
Rare diseases are individually uncommon, yet together they affect an estimated 300 million+ people worldwide and touch the lives of over one billion when families and caregivers are included. For the >7,000 distinct rare conditions, around 70% of which begin in childhood, many patients and families face common challenges including delayed diagnosis, fragmented care, and significant social and financial burdens. A recent World Economic Forum white paper, “Making Rare Diseases Count: How Better Data Can Unlock a Multi‑Trillion‑Dollar Opportunity,” argues that these conditions represent not only a profound human challenge, but also one of the largest untapped opportunities in global health and economic resilience.
The paper highlights the massive but largely invisible cost of rare diseases, estimated at between USD 7.2 and 8.6 trillion each year, and calls for stronger data systems to make this burden visible and actionable. Its roadmap centres on five priorities: defining a minimum dataset across countries, strengthening patient registries, expanding screening and diagnostic capacity, enabling trusted data sharing, and using AI and digital tools to close evidence gaps.
This agenda builds on growing policy momentum, including the pivotal 2025 World Health Assembly resolution on rare diseases, sponsored by Egypt and Spain and co‑sponsored by 39 other UN Member States, which includes a call to member states to support education for healthcare providers on rare diseases. Shortening the diagnostic odyssey through education is crucial: today, only 19% of physicians feel confident diagnosing rare diseases, and many patients wait years for an accurate diagnosis.
Medscape, as a contributor to the WEF paper, brings evidence that targeted professional education can help close this gap. Its rare disease programmes, informed by global survey data and real‑world outcomes, have shown that clinicians who participate in accredited training as compared with similar cohorts who do not, order significantly more genetic tests and code more often for rare diseases. This suggests that education may help shorten the diagnostic journey in real world practice. Medscape data also reveal striking gaps in physician awareness of rare disease prevalence, with a majority reporting that they never or rarely (defined as 1-2x per year), see rare disease patients despite evidence suggesting they should be seeing several per week.
With a community of >13 million healthcare professionals and the largest dedicated library of rare disease education, Medscape is positioned to translate the WEF’s data‑driven roadmap into everyday clinical practice. By aligning better data with focused education, the rare‑disease community can turn global recognition into earlier diagnoses, better access to care, and measurable value for patients, health systems, and societies worldwide.
To learn more about how Medscape Education is tackling the rare disease burden through precision education, please contact S Christy Rohani‑Montez.
Sign up to Medscape Education’s Rare Disease Newsletter here.
About Medscape Education
Medscape Education (medscape.org) is the leading destination for continuous professional development, consisting of more than 30 specialty-focused destinations offering thousands of free CME and CE courses for physicians, nurses, and other healthcare professional
This content was provided by Medscape Education
Company Details
Latest Content from Medscape Education
Showing Deep Expertise at ONS
The Medscape Oncology team will be connecting with the oncology nursing community this week in Washington, DC for the ONS Congress 2024. Medscape Oncology has deep expertise in the oncology...
MEDSCAPE’S NEW AGE OF CERTAINTY FOR CME
The CME industry is changing. More than ever, activities need to be prioritized and measured to increase their impact on patient outcomes. Improving patient outcomes is the core objective for...
Getting Past the Old-Fashioned View of Patient Advocacy
Patient advocacy organizations today are data-driven, doing amazing work — and have the hard evidence to support their impact on patient care. The challenge is getting past the perception of...
Mile High Impact at AAN 2024
The Medscape team will be joining our neurology colleagues in Denver, CO for this year’s AAN Annual Meeting. Impact will take center stage at this year’s event as Medscape presents...
Everything Was Peachy at ACC 2024 in Atlanta, GA
The annual ACC meeting has officially wrapped up in Atlanta, bringing a successful conference to a close for another year. During the conference, members of the Medscape team hosted a...
A Woman Should Not Be Treated As a Small Man
What should the future of women’s healthcare look like? Two renowned physicians say it involves a multidisciplinary, holistic approach, where a group of different specialists work together to provide a...
Sharing Four Times the Impact at ACC 2024
ACC is kicking off next week in Atlanta, GA, and Medscape is heading south to join our cardiology colleagues. The Medscape team will be hosting booth #3431 on the exhibit...
Transforming San Diego for AAD 2024
The 2024 meeting of the American Academy of Dermatology has wrapped up, and Medscape has returned from a series of successful days at their booth, symposia, and offsite event. To...
The Power Dynamics of Women and Cancer
Women, Power, and Cancer: a Lancet commission, studied the interaction of women and cancer through a social justice and equity lens. What does ‘power’ mean when it comes to cancer...
Evaluating the Forces Shaping Women’s Healthcare
Gaps in women’s healthcare go beyond access and cost. Do clinicians have the tools they need to understand women’s health?