Worldwide, it is estimated that over 300 million people are living with one or more of the 6,000 to 8,000 identified rare conditions, yet the impact of rare diseases is poorly understood in lower-middle and upper-middle income countries (collectively referred to here as middle-income countries). In this report, on behalf of IFPMA, CRA’s Life Sciences consultants examine the potential influence of factors such as country income level, standard of care, and health system financing on the socioeconomic impact of rare diseases on individuals, families, and communities around the world – an area few studies have considered.
Overall, the socioeconomic impact of rare diseases in middle-income countries is significant. The authors propose five policy recommendations:
- Prioritization of rare disease intervention is vital to address the significant impact identified, with specific actions to consider national contexts
- Establish programs to improve screening and early diagnosis, as they have important clinical benefits and can reduce the socioeconomic impact of rare disease on persons living with a rare disease, caregivers, and societies
- Improve the collection of data to ensure the complete socioeconomic impact of rare disease is understood and that policies can be designed appropriately
- Invest in the training of specialists, improve healthcare provider awareness, and establish national reference centers to expand access to dedicated care
- Ensure access to effective treatments for all patients to reduce the impact on persons living with a rare disease, their caregivers, and on other parts of the healthcare system
Read the full report here
Read the infographic here
This report was developed with contributions from CRA colleagues Tim Wilsdon, Michele Pistollato, Clara Zacharko, Elaine Damato, and Lucinda Douse, along with support from IFPMA, Rare Diseases International, and Professor Steven Simoens (Professor of Health Economics at KU Leuven).
