Data on a Mission: Removing Barriers to Understanding of Rare Disease
Research and drug development for rare and genetic diseases is challenging without insight into history and clinical manifestations. Missing efficacy [...]
ICER and Other Stakeholders address High Cost of Rare Disease Therapy
At the AMCP Nexus 2020 Virtual meeting, two major themes emerged from the specialty pharmacy practice sessions: new methods for [...]
[Podcast] How To Best Launch a Therapy For an Ultra-rare Disease?
In a special episode of Beyond the Molecule, Fishawack Health explores the rare disease space to reveal the perspectives of two [...]
A Magnet for the Haystack: Using AI to Find Rare Disease Patients
Correctly identifying a patient with a rare disease can be like looking for a needle in a haystack: by definition, each rare disease has fewer than 200,000 patients out of more than 300M people in the US.
Tackling Rare Diseases with an Arsenal of Real-World Data
Technological innovations have fostered the age of big data in healthcare – and the huge populations amassed afford the ability to mine for key subpopulations -- getting down to ever more precise groups of likely responders to therapy, high-risk patients and other high-value subphenotypes.
