Drug Safety Matters

#7 The challenge of rare diseases – Christina Ström Möller

February 26, 2021 Uppsala Monitoring Centre
Drug Safety Matters
#7 The challenge of rare diseases – Christina Ström Möller
Show Notes

By definition, a rare disease is one that only affects a handful of people in the world. But with more than 300 million people collectively affected by these conditions and only few approved treatments available, rare diseases constitute a major unmet medical need. Christina Ström Möller from Swedish biopharmaceutical company Sobi walks us through the scientific, commercial and human challenges of the rare disease landscape.

Tune in to find out:

  • What drug development, testing and distribution look like when patients are few and far apart 
  • Why patient narratives are especially important when monitoring the safety of rare disease drugs
  • How patient organisations work to support rare disease patients and raise awareness of their condition

Want to know more?

Read the stories of patients living with rare diseases on the Sobi website.

Show your support for Rare Disease Day on 28 February by sharing campaign materials online with the hashtag #RareDiseaseDay or joining one of the many events planned around the world.

Rare Disease Day is organised by EURORDIS-Rare Diseases Europe, a non-profit alliance of rare disease patient organisations from 73 countries.

When assessing the safety of rare disease medicines, also known as orphan drugs, it is important to include all available sources of data.

A recent review of orphan drug policies found that several low- and middle-income countries had developed such policies in the last decade ­– although disparities between countries with different income levels still remain.

A survey conducted by EURORDIS in 2020 found that the COVID-19 pandemic exacerbated the many challenges that people living with a rare disease face.

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