Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example

Andrea M. Gross

2021Current Problems in Cancer21 citationsDOI

Topics & Concepts

MedicineRare diseaseClinical trialIntensive care medicineDiseasePopulationEnvironmental healthPathologyNeurofibromatosis and Schwannoma CasesHereditary Neurological DisordersNeuroblastoma Research and Treatments

Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations &amp; a case example

Topics & Concepts

Using real world data to support regulatory approval of drugs in rare diseases: A review of opportunities, limitations & a case example