Small But Mighty: The Use of Real‐World Evidence to Inform Precision Medicine

The intersection of information technology and biomedical breakthroughs creates new opportunities for real-world evidence (RWE). Initially used in comparative effectiveness and population health research, RWE now can bolster personalized care. Precision medicine segments diseases into small, biologically informed subgroups. For these rare populations, small-cohort RWE can inform clinical, regulatory, and development decision making as well as...