#7 The challenge of rare diseases – Christina Ström Möller

Send us a voicemail!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 drugsHow patient organisations work to support rare disease patients and raise awareness of their conditionWant 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.Got a story to share?We’re always looking for new topics and interesting voices. If you have an idea or any other feedback for the show, get in touch!About UMCUppsala Monitoring Centre promotes safer use of medicines and vaccines for everyone everywhere. Follow us on Facebook, LinkedIn, X, and Bluesky.