The Role of Literature-Derived RWE in Rare or Complex Indications
In this white paper, we reveal how literature-derived real-world evidence (RWE) can fill critical evidence gaps in rare and complex indications:
- For NPM1-mutated AML, literature-derived RWE workflow surfaced >1,000 patients where an EHR dataset provided only a few dozen.
- In PRKAG2 syndrome, it enabled detection of 2.7x more patients than prior reviews, with richer phenotyping and longitudinal follow-up.
- In pediatric ABCC6 deficiency, it uncovered a heterogeneous, multi-organ phenotype in a previously uncharacterized subpopulation.
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