What is an orphan drug?


About the research

Do you remember baby Pia? She suffered from the rare muscle disease AMS and the medicine to save her cost a whopping €1.9 million. If you were the Minister of Health, would you pay for Pia's medicine? Even if that meant there was no budget left for another medicine that might save 100 people? Alessandra Blonda (KU Leuven) tells you more about so-called orphan drugs, medicines for rare diseases.

Alessandra Blona
KU Leuven

After graduating in Drug Development at KU Leuven, Alessandra Blonda worked in the pharmaceutical industry for a couple of years. As her last position focused on market access & public affairs for biosimilars and generics, she became more aware of the issues regarding patient access to medicines, especially those for rare disease patients. For this reason, she went back to KU Leuven to start a PhD. Through her research, she aims to increase access to orphan drugs by optimizing existing reimbursement processes.

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