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Population health science: unlocking insights for rare diseases

Population health science can give rare disease communities powerful tools to describe their needs, highlight inequalities, and advocate for better care. This course introduces the key ideas, like health equity, burden of disease, and epidemiology, in a clear and practical way, and explains why rare conditions are often overlooked in traditional public health systems.

You’ll learn how to use population-level data to highlight unmet needs, demonstrate the true impact of your condition, and strengthen your advocacy. The course also shows you how public health institutions work, where rare diseases fit in national strategies, and how patient groups can influence decisions about care, research, and policy.

By the end, you’ll have the language, tools, and confidence to ensure your community is seen, counted, and included in population health conversations – locally, nationally, and globally.

With thanks to:

We’d like to extend our thanks to Divya Karan, who volunteered with Beacon throughout 2025 and 2026 in the development of this guide. See Divya’s bio below!

Divya Karan is a Senior Consultant at Dalberg Advisors and a population health researcher, with experience working across global health, data, and health systems strengthening.

She has supported organizations such as UNICEF, philanthropic foundations, and global health partners on initiatives spanning health system resilience, access to new treatments, health data innovations, and vaccine uptake.

Divya holds an MPhil in Population Health Sciences from the University of Cambridge, where she specialised in Health Data Science, and a BSc in Economics from the London School of Economics. Through her work with Beacon for Rare Diseases, she is passionate about translating complex health and data concepts into clear, practical learning resources that empower communities navigating rare diseases.

Resource type

Course

Skill level

Beginner/Intermediate

Duration

Est. 1.5h

Last updated

March 2026

Resource type

Course

Skill level

Beginner/Intermediate

Duration

Est. 1.5h

Last updated

March 2026

What will you learn?

  • Different public health concepts, including population health, health equity, epidemiology, and burden of disease, and how they relate to rare conditions

 

  • How different frameworks are used to assess disease burden, such as the Global Burden of Disease and Orphanet approaches, and what their strengths and limitations are for rare diseases
    • How to use population-level data to support your advocacy, from making the case for funding and research to highlighting unmet needs and influencing policy decisions

     

    • How to work effectively with public health institutions, contribute to national and international rare disease strategies, and collaborate with researchers and decision-makers to improve outcomes for your community

    Who is this course for?

    This guide is designed to help patient organisations understand how population health science applies to rare diseases and how they can use this knowledge to advocate, engage in research, and influence health policy.

    Resource Content

    INTRODUCTION
    Understanding population health sciences
    UNDERSTANDING INDICATORS
    Mortality and morbidity indicators
    Health system and equity indicators
    UNDERSTANDING WHY RARE DISEASES MATTER
    Why population health science matters for rare diseases
    FRAMEWORKS ASSESSING DISEASE BURDEN
    Frameworks assessing burden of disease
    THE INTERSECTION OF RARE DISEASE AND POPULATION HEALTH
    Introduction to Public Health Institutions and case study
    Public Health Institutions: their roles
    HOW ENGLAND EVALUATES ULTRA-RARE DISEASE TREATMENTS
    Spotlight: How England Evaluates Treatments for Ultra-Rare Diseases
    DATA AND EVIDENCE IN RARE DISEASES
    Introduction to data and evidence
    Challenges in data collection and the importance of data
    CASE STUDIES
    The Rare Disease Registries (RDRs) and the RDR Patient Council
    POLICY
    Public health policy
    England Rare Diseases Action Plan 2025
    France embedding rare diseases in its National Health Strategy
    PATIENT ORGANISATIONS USING POPULATION HEALTH APPROACHES
    Duchenne Muscular Dystrophy (DMD) case study
    SUMMARY
    Summary
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