Key Takeaways from the World Orphan Drug Congress

The World Orphan Drug Congress brought us together with the top stakeholders in rare and orphan disease. Here are our top three takeaways. 

1. Artificial Intelligence is here to stay.

Whether it was AI platforms to help in rare disease discovery or chatbots to support rare disease diagnoses, everyone was talking about AI. The general consensus is that AI isn’t necessarily close to implementation but that it will be an essential part of the toolbox for future advances in rare and orphan diseases.

2. Data Accessibility continues to be a massive challenge.

With an inherently small patient base, rare disease research and clinical trials have a dire need for data which is often locked in siloes. While the information coming from inside the U.S. is valuable, we must also include and integrate the information from the rest of the globe. Additionally, patient and advocacy groups generate a lot of potentially useful data, and we need to figure out how to incorporate that into our understanding of the disease.

3. FDA Ruling on Omaveloxolone shows a willingness to innovate

The acceptance by FDA of the use of a natural history data set on Freidfreich’s ataxia (FA-COMS) to perform a propensity match-based analysis for the Omaveloxolone clinical trial is a major step forward. This validates the work and utility of the natural history data being collected by rare disease patients and their advocates and sets the path for how it can contribute to future clinical trials.

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