The Global Socioeconomic Impact of Rare Diseases: A Call for Action

May 22, 2024

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Open to all ISPOR Members and Non-members

Title: The Global Socioeconomic Impact of Rare Diseases: A Call for Action

Wednesday, May 22, 2024
10:00AM EDT | 2:00PM UTC | 4:00PM CEST

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The webinar will begin with a (5-minute) introduction of the participants of the study to the ISPOR Rare Disease (RD) SIG, followed by an overview of the study objectives, its methodology and findings (25-minute). This will be followed by a (30-minute) Q&A moderated discussion. 

The novel study explored the burden of different RDs across a range of countries, including low-and-middle-income countries (LMICs). The diseases and countries were chosen to allow for a diverse and relevant representation of RD burden globally, aiming to capture cross-disease and cross-country unmet needs, and consequent areas for policy intervention. 

The RDs were selected based on key commonalities (i.e., prevalence distribution across countries, not being ultra-rare, having defined diagnostic protocols, and treatment availability). Countries were selected to give distribution across global regions not covered in previous studies (Latin America, Africa, Eastern Mediterranean Region, Asia, and Oceania), income levels and healthcare system characteristics. 

The heterogeneity in diseases (e.g., diagnostic and treatment pathways, prognosis, onset) and countries (e.g., universal healthcare coverage; availability of national RD plan, RD specialists, patient advocacy) allowed for the exploration of factors that could help explain differences in burden and impact. The data for this study was derived from literature searches, and input from patient experts, a health economist and industry representatives to provide context and validation to the findings and interpretations. 

Following the presentation, the multi-stakeholder panel will reflect on the findings and the implications from the perspectives of a health economist, the RD patient community and the pharmaceutical industry. The panel will aim to cover the following topics: 

  • Comparison with studies conducted in High-Income Countries (HICs), and the impact of data scarcity in LMICs on the socioeconomic estimates; 
  • The consequences of low investment in RDs on the overall burden; 
  • The value of investing in diagnosis and early interventions; and
  • The ‘best-buys’ to increase investment in RDs.

Learning Objectives

  • Understand the global impact of rare diseases in on patients, caregivers, healthcare systems and society – and the associated socioeconomic burden. 
  • Have a clear idea of data and policy gaps for RD, especially in LMICs, and discuss policy recommendations, with a specific focus on LMICs. 
  • Provide with a basis for future studies on estimating the global socioeconomic impact of RD, also considering the data limitations in LMICs.


Carina Righetti, MSc, Director, Global Market Access & Policy, J&J, Princeton, NJ, USA

Ann-Lise Mikolajczak, PharmD, Manager, Global Health and Access, IFPMA, Geneva, Switzerland


Durhane Wong-Rieger, PhD, Chair, Rare Diseases International (RDI), Toronto, ON, Canada

Clara Zacharko, Consulting Associate, Charles Rives Associates (CRA), London, England, UK 

Steven Simoens, PhD, Professor of Health Economics, KU Leuven, Leuven, Belgium

Brought to you by: The ISPOR Rare Disease Special Interest Group

Please note:
 On the day of the scheduled webinar, the first 1000 registered participants will be accepted into the webinar. For those who are unable to attend, or would like to review the webinar at a later date, the full-length webinar recording will be made available at the ISPOR Educational Webinar Series webpage approximately 2 days after the scheduled Webinar.

Reservations are on a first-come, first-served basis.

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