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WHO Adds Rare Disease Treatments to the Essential Medicines List
Salome Mekhuzla/LinkedIn
Oct 9, 2025, 12:54

WHO Adds Rare Disease Treatments to the Essential Medicines List

Salome Mekhuzla, Director of Global Development at the World Federation of Hemophilia, reposted from Rare Diseases International on LinkedIn:

”Thrilled to have contributed to this project and congratulations to RDI team on all the work accomplished.”

Quoting Rare Diseases International‘s post:

”New Report Published!

With the publication of the updated WHO Model List of Essential Medicines (EML) and the Model List for Children (EMLc), important progress has been made: for the first time, treatments for several rare diseases have been included. This achievement highlights how advocacy and active engagement are vital to ensure that more therapies for rare diseases are recognized as essential in the future. In this context, RDI is proud to share the publication of the Essential Medicines Working Group (EMWG) Report.
​​
This report, developed by RDI’s Essential Medicines Working Group, explores:​

  • Successful and unsuccessful applications for rare disease medicines to the EML, identifying challenges, success factors, and opportunities to strengthen the evaluation process for essential RD medicines.
  • Concrete recommendations and a Call-to-Action to strengthen the EML’s impact for people living with rare diseases globally.

This work would not have been possible without the invaluable contributions of the members of the RDI Essential Medicines Working Group and the organizations that generously shared their experiences. Special thanks to all contributors for their dedication and insights. This project was developed by RDI Global Programme: Chiuhui Mary Wang, Chiara Tovani and Sara Brambilla.

Read the full report here․

For more information, check our website․”

Rare Disease

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Chiara Tovani Chiuhui Mary Wang EMWG Essential Medicines Working Group Health Hematology Hemophilia Hemostasis Today Medicine Rare Diseases International RDI Salome Mekhuzla Sara Brambilla WHO
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